Ignite Creation Date:
2025-12-25 @ 12:10 AM
Ignite Modification Date:
2025-12-25 @ 10:10 PM
Study NCT ID:
NCT01255358
Status:
COMPLETED
Last Update Posted:
2024-10-10
First Post:
2010-12-03
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}], 'ancestors': [{'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D003907', 'term': 'Dexamethasone'}, {'id': 'C004180', 'term': 'dexamethasone 21-phosphate'}], 'ancestors': [{'id': 'D011246', 'term': 'Pregnadienetriols'}, {'id': 'D011245', 'term': 'Pregnadienes'}, {'id': 'D011278', 'term': 'Pregnanes'}, {'id': 'D013256', 'term': 'Steroids'}, {'id': 'D000072473', 'term': 'Fused-Ring Compounds'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}, {'id': 'D013259', 'term': 'Steroids, Fluorinated'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'guenter.janhofer@erydel.com', 'phone': '+39 02 36504470', 'title': 'Clinical Study Coordinator', 'organization': 'EryDel spa'}, 'certainAgreement': {'restrictionType': 'LTE60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'The adverse events were monitored and collected during the 6 month study period', 'description': 'Please note that non serious Adverse Events were monitored/assessed without regard to the specific Adverse Event Term but only by SOC.', 'eventGroups': [{'id': 'EG000', 'title': 'Ery-Dex', 'description': 'Patients treated with monthly treatment of Ery-Dex (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)', 'otherNumAtRisk': 22, 'deathsNumAtRisk': 22, 'otherNumAffected': 15, 'seriousNumAtRisk': 22, 'deathsNumAffected': 0, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'General disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (10.0)'}, {'term': 'immune disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (10.0)'}, {'term': 'infections and infestations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numEvents': 17, 'numAffected': 15}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (10.0)'}, {'term': 'respiratory disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (10.0)'}], 'seriousEvents': [{'term': 'Bronchopneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (10.0)'}, {'term': 'Bronchiectasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 22, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (10.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ERY-DEX', 'description': 'Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)\n\nDexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient).\n\nThe treatment has to be repeated at intervals of 30 days (±10 days)'}], 'classes': [{'title': 'V2', 'categories': [{'measurements': [{'value': '-2.2', 'spread': '5.6', 'groupId': 'OG000'}]}]}, {'title': 'V4', 'categories': [{'measurements': [{'value': '-3.4', 'spread': '7.3', 'groupId': 'OG000'}]}]}, {'title': 'V7', 'categories': [{'measurements': [{'value': '-4.0', 'spread': '7.5', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '0.02', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-4.0', 'ciLowerLimit': '-7.1', 'ciUpperLimit': '-0.9', 'dispersionType': 'STANDARD_DEVIATION', 'dispersionValue': '7.5', 'groupDescription': 'The primary analysis on ICARS Total score has been conducted on the ITT Population employing carry-forward and carry-backward procedures for missing data imputation, in order to evaluate all enrolled patients.', 'statisticalMethod': 'Wilcoxon (Mann-Whitney)', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Changes from baseline (V1) at V2, V4 and V7 were analyzed with a RMANOVA design with age and ICARS at baseline as covariates. A Wilcoxon non-parametric test between visits was performed when the overall analysis was significant in order to help determine timing of effects'}], 'paramType': 'MEAN', 'timeFrame': 'At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)', 'description': 'ICARS ia a 100-point semiquantitative scale, which goes from 0 to 100, offering a compartimentalised quantification of 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6) for a possible total score of 100 points. The minimum score is 0 (normal), while the maximum score is 100 and corresponds to the worst status of the patient. This means that for each subscale, higher scores indicate higher levels of impairment.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent to treat population (ITT) patients receiving at least one treatment'}, {'type': 'PRIMARY', 'title': 'Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ERY-DEX', 'description': 'Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)\n\ndexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient).\n\nThe treatment has to be repeated at intervals of 30 days (±10 days)'}], 'classes': [{'title': 'V2', 'categories': [{'measurements': [{'value': '-2.4', 'spread': '4.9', 'groupId': 'OG000'}]}]}, {'title': 'V4', 'categories': [{'measurements': [{'value': '-3.9', 'spread': '7.1', 'groupId': 'OG000'}]}]}, {'title': 'V7', 'categories': [{'measurements': [{'value': '-5.2', 'spread': '7.0', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '0.0031', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-5.2', 'ciLowerLimit': '-8.4', 'ciUpperLimit': '-2', 'dispersionType': 'STANDARD_DEVIATION', 'dispersionValue': '7.0', 'groupDescription': 'Overall analysis on the PP Population', 'statisticalMethod': 'Wilcoxon (Mann-Whitney)', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Changes from baseline (V1) at V2, V4 and V7 were analyzed with a RMANOVA design with age and ICARS at baseline as covariates. A Wilcoxon non-parametric test between visits was performed when the overall analysis was significant in order to help determine timing of effects'}], 'paramType': 'MEAN', 'timeFrame': 'At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)', 'description': 'ICARS ia a 100-point semiquantitative scale, which goes from 0 to 100, offering a compartimentalised quantification of 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6) for a possible total score of 100 points. The minimum score is 0 (normal), while the maximum score is 100 and corresponds to the worst status of the patient. This means that for each subscale, higher scores indicate higher levels of impairment.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The PP Population includes 18 patients who have been treated according to protocol and fulfill the following criteria:\n\n* compliance with all entry criteria\n* absence of major protocol violations\n* adequate compliance with trial medication (at most one missed or out-of-window infusion of the study preparation).'}, {'type': 'SECONDARY', 'title': 'Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ERY-DEX', 'description': 'Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes).\n\nDexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient). "\n\nThe treatment has to be repeated at intervals of 30 days (±10 days)'}], 'classes': [{'categories': [{'measurements': [{'value': '0.2', 'spread': '0.6', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At visit 7 (final visit, day 180)', 'description': 'An evaluation of the global health status of the patients by the IGA was performed. IGA consisted of an evaluation of neurological signs and symptoms as neuromotor disorders, posture and deambulation, disturbances of the oro-pharyngeal apparatus, presence of peripheral neuropathy, neurodevelopmental disturbances, intellectual level, and behavior disturbances based only on a subjective judgment of the Investigator on a single scale: a 5-point qualitative scale as:\n\n* very much improved (1),\n* slightly improved (2),\n* no change (3),\n* slightly worsened (4),\n* very much worsened (5) was used to assess changes from V4 to V7. The higher the score, the worse the outcome.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT Population includes all 22 patients enrolled into the study.'}, {'type': 'SECONDARY', 'title': 'Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ERY-DEX', 'description': 'Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes).\n\nDexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient).\n\nThe treatment has to be repeated at intervals of 30 days (±10 days)'}], 'classes': [{'categories': [{'measurements': [{'value': '0.2', 'spread': '0.6', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At visit 7 (final visit, day 180)', 'description': 'An evaluation of the global health status of the patients by the IGA was performed. IGA consisted of an evaluation of neurological signs and symptoms as neuromotor disorders, posture and deambulation, disturbances of the oro-pharyngeal apparatus, presence of peripheral neuropathy, neurodevelopmental disturbances, intellectual level, and behavior disturbances based only on a subjective judgment of the Investigator on a single scale: a 5-point qualitative scale as:\n\n* very much improved (1),\n* slightly improved (2),\n* no change (3),\n* slightly worsened (4),\n* very much worsened (5) was used to assess changes from V4 to V7. The higher the score, the worse the outcome.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The PP Population includes 18 patients who have been treated according to protocol and fulfill the following criteria:\n\n* compliance with all entry criteria\n* absence of major protocol violations\n* adequate compliance with trial medication (at most one missed or out-of-window infusion of the study preparation).'}, {'type': 'SECONDARY', 'title': 'Mean Change From V4 to V7 in Ocular Motility - ITT Population', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ERY-DEX', 'description': 'Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes).\n\nDexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient). "\n\nThe treatment has to be repeated at intervals of 30 days (±10 days)'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.3', 'spread': '0.5', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At visit 7 (final visit, day 180)', 'description': 'An evaluation of ocular motility measured by an ad hoc form was performed at V4 and V7.\n\nAt V4 and V7, ocular motility was assessed through a 5-point qualitative scale: very much improved (1), slightly improved (2), no change (3), slightly worsened (4), very much worsened (5).', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT Population includes all 22 patients enrolled into the study.'}, {'type': 'SECONDARY', 'title': 'Mean Change From V4 to V7 in Ocular Motility - PP Population', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ERY-DEX', 'description': 'Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes).\n\nDexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient). "\n\nThe treatment has to be repeated at intervals of 30 days (±10 days)'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.3', 'spread': '0.5', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At visit 7 (final visit, day 180)', 'description': 'An evaluation of ocular motility measured by an ad hoc form was performed at V4 and V7.\n\nAt V4 and V7, ocular motility was assessed through a 5-point qualitative scale: very much improved (1), slightly improved (2), no change (3), slightly worsened (4), very much worsened (5).', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The PP Population includes 18 patients who have been treated according to protocol and fulfill the following criteria:\n\n* compliance with all entry criteria\n* absence of major protocol violations\n* adequate compliance with trial medication (at most one missed or out-of-window infusion of the study preparation).'}, {'type': 'SECONDARY', 'title': 'Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - ITT Population', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ERY-DEX', 'description': 'Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)\n\nDexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient). "\n\nThe treatment has to be repeated at intervals of 30 days (±10 days)'}], 'classes': [{'title': 'V4', 'categories': [{'measurements': [{'value': '1.0', 'spread': '0.9', 'groupId': 'OG000'}]}]}, {'title': 'V7', 'categories': [{'measurements': [{'value': '1.3', 'spread': '1.3', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000'], 'paramType': 'Median Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.3', 'ciLowerLimit': '0.8', 'ciUpperLimit': '1.8', 'dispersionType': 'STANDARD_DEVIATION', 'dispersionValue': '1.2', 'groupDescription': 'VABS total score at V7', 'statisticalMethod': 'Wilcoxon (Mann-Whitney)', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'VABS Total score and subscales have been analyzed with a RMANOVA design, using age as covariate (dichotomized as Low- or High-, using median age as threshold).'}], 'paramType': 'MEAN', 'timeFrame': 'At visits 4 (90 days) and 7 (180 days, final visit)', 'description': "VABS aggregate scale is used.\n\nVABS is divided into 4 adaptive domains and 11 subdomains:\n\n1. Communication: Receptive, Expressive, Written;\n2. Daily Living Skills: Personal, Domestic, Community;\n3. Socialisation: Interpersonal Relationships, Play \\& Leisure Time, Coping Skills;\n4. Motor Skills: Gross, Fine.\n\nFor each \\*question\\* in each of the 11 subdomains, score ranges from 0 to 4 (the higher the value the better the outcome):\n\n* 0 = the subject never performs the behaviour without help or reminders;\n* 1 = rarely performs (as above);\n* 2 = sometimes performs (as above);\n* 3 = often performs (as above);\n* 4 = almost always performs (as above); The total subdomain score is the sum of each \\*question\\* scores and the total domain score is the sum of each subdomains' scores.\n\nDomains min-max interval scores:\n\n0-74 Communication, 0-74 Daily Living Skills, 0-62 Socialization, 0-111 Motor Skills, Sum-of-domains score 0-321(the higher the score, the better the outcome)", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT Population includes all 22 patients enrolled into the study.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - PP Population', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ERY-DEX', 'description': 'Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)\n\nDexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient). "\n\nThe treatment has to be repeated at intervals of 30 days (±10 days)'}], 'classes': [{'title': 'V4', 'categories': [{'measurements': [{'value': '1.1', 'spread': '0.9', 'groupId': 'OG000'}]}]}, {'title': 'V7', 'categories': [{'measurements': [{'value': '1.5', 'spread': '1.1', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.5', 'ciLowerLimit': '1', 'ciUpperLimit': '2', 'dispersionType': 'STANDARD_DEVIATION', 'dispersionValue': '1.1', 'statisticalMethod': 'Wilcoxon (Mann-Whitney)', 'nonInferiorityType': 'OTHER'}], 'paramType': 'MEAN', 'timeFrame': 'At visits 4 (90 days) and 7 (180 days, final visit)', 'description': "VABS aggregate scale is used.\n\nVABS is divided into 4 adaptive domains and 11 subdomains:\n\n1. Communication: Receptive, Expressive, Written;\n2. Daily Living Skills: Personal, Domestic, Community;\n3. Socialisation: Interpersonal Relationships, Play \\& Leisure Time, Coping Skills;\n4. Motor Skills: Gross, Fine.\n\nFor each \\*question\\* in each of the 11 subdomains, score ranges from 0 to 4 (the higher the value the better the outcome):\n\n* 0 = the subject never performs the behaviour without help or reminders;\n* 1 = rarely performs (as above);\n* 2 = sometimes performs (as above);\n* 3 = often performs (as above);\n* 4 = almost always performs (as above); The total subdomain score is the sum of each \\*question\\* scores and the total domain score is the sum of each subdomains' scores.\n\nDomains min-max interval scores:\n\n0-74 Communication, 0-74 Daily Living Skills, 0-62 Socialization, 0-111 Motor Skills, Sum-of-domains score 0-321(the higher the score, the better the outcome)", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The PP Population includes 18 patients who have been treated according to protocol and fulfill the following criteria:\n\n* compliance with all entry criteria\n* absence of major protocol violations\n* adequate compliance with trial medication (at most one missed or out-of-window infusion of the study preparation).'}, {'type': 'SECONDARY', 'title': 'Count of Participants on ERY-DEX Showing Treatment Emergent Adverse Events (TEAEs) Including Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'ERY-DEX', 'description': 'Patients treated with monthly treatment of EryDex (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)\n\ndexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient).\n\nThe treatment has to be repeated at intervals of 30 days (±10 days)'}], 'classes': [{'title': 'Participants with at least 1 TEAE', 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}]}]}, {'title': 'Participants with at least 1 serious AE', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Throughout the study, until the end of 6 months of treatment (day 180)', 'description': 'The effect of ERY-DEX on treatment emergent adverse events was expressed as the number of patients showing at least one TEAE or 1 serious AE', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The SP includes the 22 patients who have received at least one dose of planned trial treatment.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'EryDex', 'description': 'Patients treated with monthly treatment of EryDex (dexamethasone sodium phosphate encapsulated in autologous erythrocytes).'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '22'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '18'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Protocol Violation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}]}], 'recruitmentDetails': 'Study was carried out in 2 Italian Hospitals. Recruitment started on February 2011 and LPLV was performed on August 2011.', 'preAssignmentDetails': 'Patients had 30 days of wash-out from steroid assumption before starting the treatment. 26 patients were screened, 4 of them resulted screening failure, due to lymphocytes value below the limit (3) and concomitant disease (1).'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'ERY-DEX', 'description': 'Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '21', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '11.2', 'spread': '3.5', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Italy', 'categories': [{'measurements': [{'value': '22', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'The study enrolled 22 patients, 11 male and 11 female with a mean age of 11.2. All the enrolled patients received a monthly treatment of EryDex for 6 consecutive months.'}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 22}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-07', 'completionDateStruct': {'date': '2011-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-07-12', 'studyFirstSubmitDate': '2010-12-03', 'resultsFirstSubmitDate': '2012-07-02', 'studyFirstSubmitQcDate': '2010-12-06', 'lastUpdatePostDateStruct': {'date': '2024-10-10', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-07-12', 'studyFirstPostDateStruct': {'date': '2010-12-07', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2024-10-10', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2011-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population', 'timeFrame': 'At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)', 'description': 'ICARS ia a 100-point semiquantitative scale, which goes from 0 to 100, offering a compartimentalised quantification of 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6) for a possible total score of 100 points. The minimum score is 0 (normal), while the maximum score is 100 and corresponds to the worst status of the patient. This means that for each subscale, higher scores indicate higher levels of impairment.'}, {'measure': 'Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population', 'timeFrame': 'At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)', 'description': 'ICARS ia a 100-point semiquantitative scale, which goes from 0 to 100, offering a compartimentalised quantification of 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6) for a possible total score of 100 points. The minimum score is 0 (normal), while the maximum score is 100 and corresponds to the worst status of the patient. This means that for each subscale, higher scores indicate higher levels of impairment.'}], 'secondaryOutcomes': [{'measure': 'Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population', 'timeFrame': 'At visit 7 (final visit, day 180)', 'description': 'An evaluation of the global health status of the patients by the IGA was performed. IGA consisted of an evaluation of neurological signs and symptoms as neuromotor disorders, posture and deambulation, disturbances of the oro-pharyngeal apparatus, presence of peripheral neuropathy, neurodevelopmental disturbances, intellectual level, and behavior disturbances based only on a subjective judgment of the Investigator on a single scale: a 5-point qualitative scale as:\n\n* very much improved (1),\n* slightly improved (2),\n* no change (3),\n* slightly worsened (4),\n* very much worsened (5) was used to assess changes from V4 to V7. The higher the score, the worse the outcome.'}, {'measure': 'Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population', 'timeFrame': 'At visit 7 (final visit, day 180)', 'description': 'An evaluation of the global health status of the patients by the IGA was performed. IGA consisted of an evaluation of neurological signs and symptoms as neuromotor disorders, posture and deambulation, disturbances of the oro-pharyngeal apparatus, presence of peripheral neuropathy, neurodevelopmental disturbances, intellectual level, and behavior disturbances based only on a subjective judgment of the Investigator on a single scale: a 5-point qualitative scale as:\n\n* very much improved (1),\n* slightly improved (2),\n* no change (3),\n* slightly worsened (4),\n* very much worsened (5) was used to assess changes from V4 to V7. The higher the score, the worse the outcome.'}, {'measure': 'Mean Change From V4 to V7 in Ocular Motility - ITT Population', 'timeFrame': 'At visit 7 (final visit, day 180)', 'description': 'An evaluation of ocular motility measured by an ad hoc form was performed at V4 and V7.\n\nAt V4 and V7, ocular motility was assessed through a 5-point qualitative scale: very much improved (1), slightly improved (2), no change (3), slightly worsened (4), very much worsened (5).'}, {'measure': 'Mean Change From V4 to V7 in Ocular Motility - PP Population', 'timeFrame': 'At visit 7 (final visit, day 180)', 'description': 'An evaluation of ocular motility measured by an ad hoc form was performed at V4 and V7.\n\nAt V4 and V7, ocular motility was assessed through a 5-point qualitative scale: very much improved (1), slightly improved (2), no change (3), slightly worsened (4), very much worsened (5).'}, {'measure': 'Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - ITT Population', 'timeFrame': 'At visits 4 (90 days) and 7 (180 days, final visit)', 'description': "VABS aggregate scale is used.\n\nVABS is divided into 4 adaptive domains and 11 subdomains:\n\n1. Communication: Receptive, Expressive, Written;\n2. Daily Living Skills: Personal, Domestic, Community;\n3. Socialisation: Interpersonal Relationships, Play \\& Leisure Time, Coping Skills;\n4. Motor Skills: Gross, Fine.\n\nFor each \\*question\\* in each of the 11 subdomains, score ranges from 0 to 4 (the higher the value the better the outcome):\n\n* 0 = the subject never performs the behaviour without help or reminders;\n* 1 = rarely performs (as above);\n* 2 = sometimes performs (as above);\n* 3 = often performs (as above);\n* 4 = almost always performs (as above); The total subdomain score is the sum of each \\*question\\* scores and the total domain score is the sum of each subdomains' scores.\n\nDomains min-max interval scores:\n\n0-74 Communication, 0-74 Daily Living Skills, 0-62 Socialization, 0-111 Motor Skills, Sum-of-domains score 0-321(the higher the score, the better the outcome)"}, {'measure': 'Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - PP Population', 'timeFrame': 'At visits 4 (90 days) and 7 (180 days, final visit)', 'description': "VABS aggregate scale is used.\n\nVABS is divided into 4 adaptive domains and 11 subdomains:\n\n1. Communication: Receptive, Expressive, Written;\n2. Daily Living Skills: Personal, Domestic, Community;\n3. Socialisation: Interpersonal Relationships, Play \\& Leisure Time, Coping Skills;\n4. Motor Skills: Gross, Fine.\n\nFor each \\*question\\* in each of the 11 subdomains, score ranges from 0 to 4 (the higher the value the better the outcome):\n\n* 0 = the subject never performs the behaviour without help or reminders;\n* 1 = rarely performs (as above);\n* 2 = sometimes performs (as above);\n* 3 = often performs (as above);\n* 4 = almost always performs (as above); The total subdomain score is the sum of each \\*question\\* scores and the total domain score is the sum of each subdomains' scores.\n\nDomains min-max interval scores:\n\n0-74 Communication, 0-74 Daily Living Skills, 0-62 Socialization, 0-111 Motor Skills, Sum-of-domains score 0-321(the higher the score, the better the outcome)"}, {'measure': 'Count of Participants on ERY-DEX Showing Treatment Emergent Adverse Events (TEAEs) Including Serious Adverse Events (SAEs)', 'timeFrame': 'Throughout the study, until the end of 6 months of treatment (day 180)', 'description': 'The effect of ERY-DEX on treatment emergent adverse events was expressed as the number of patients showing at least one TEAE or 1 serious AE'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Ataxia Teleangiectasia', 'AT', 'ERY-DEX', 'Dexamethasone', 'Dexamethasone sodium phosphate'], 'conditions': ['Nervous System Disorder', 'Genetic Syndrome']}, 'descriptionModule': {'briefSummary': 'The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).', 'detailedDescription': 'This is a multi-centre, single arm, open label, 6 months, phase II study to evaluate the effect of ERY-DEX in improving Central Nervous System (CNS) symptoms in patients with Ataxia Teleangectasia (AT). The study consists of a screening period (max duration of 30 days) and a treatment period (duration 6 months).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '3 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* neurological signs of AT\n* patients in autonomous gait or helped by a support\n* proven molecular diagnosis of AT\n* Males and females aged \\> 3 years\n* Body weight \\>15 kg\n* Plasma levels of Lymphocytes CD4+/mm3 \\> 500 (for patients aged 3-6 years) or \\> 200 (older than 6 years)\n* written IC to participate.\n\nExclusion Criteria:\n\n* Current or previous neoplastic disease\n* History of severe impairment of the immunological system\n* Chronic conditions representing a contraindication to the use of steroid drugs\n* Non compliance with the study request\n* Any previous steroid assumption within 30 days before starting ERY-DEX\n* Have any other significant disease that in the Investigator's opinion would exclude the patient from the trial\n* Females of childbearing potential who were pregnant, breast-feeding or were not using adequate contraceptive methods"}, 'identificationModule': {'nctId': 'NCT01255358', 'acronym': 'IEDAT-01', 'briefTitle': 'Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Quince Therapeutics S.p.A.'}, 'officialTitle': 'Evaluations of Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Ataxia Teleangiectasia Patients', 'orgStudyIdInfo': {'id': 'IEDAT-01'}, 'secondaryIdInfos': [{'id': '2010-022315-19', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'ERY-DEX', 'description': 'Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)', 'interventionNames': ['Drug: Dexamethasone']}], 'interventions': [{'name': 'Dexamethasone', 'type': 'DRUG', 'otherNames': ['Dexamethasone sodium phosphate', 'Dex 21P'], 'description': 'Dexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient).\n\nThe treatment has to be repeated at intervals of 30 days (±10 days)', 'armGroupLabels': ['ERY-DEX']}]}, 'contactsLocationsModule': {'locations': [{'zip': '25123', 'city': 'Brescia', 'country': 'Italy', 'facility': 'Spedali Civili', 'geoPoint': {'lat': 45.53558, 'lon': 10.21472}}, {'zip': '00185', 'city': 'Rome', 'country': 'Italy', 'facility': 'University La Sapienza', 'geoPoint': {'lat': 41.89193, 'lon': 12.51133}}], 'overallOfficials': [{'name': 'Luciana Chessa, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "A.O. Sant'Andrea Rome Italy"}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Quince Therapeutics S.p.A.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}