Ignite Creation Date:
2025-12-25 @ 3:18 AM
Ignite Modification Date:
2025-12-26 @ 3:55 PM
Study NCT ID:
NCT01048905
Status:
COMPLETED
Last Update Posted:
2021-06-10
First Post:
2010-01-12
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006976', 'term': 'Hypertension, Pulmonary'}, {'id': 'D000755', 'term': 'Anemia, Sickle Cell'}, {'id': 'D013789', 'term': 'Thalassemia'}], 'ancestors': [{'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D006973', 'term': 'Hypertension'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D005973', 'term': 'Glutamine'}], 'ancestors': [{'id': 'D024361', 'term': 'Amino Acids, Basic'}, {'id': 'D000596', 'term': 'Amino Acids'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D000599', 'term': 'Amino Acids, Diamino'}, {'id': 'D021542', 'term': 'Amino Acids, Neutral'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'claudia.r.morris@emory.edu', 'phone': '404-727-5500', 'title': 'Claudia R. Morris MD, FAAP', 'organization': 'Emory University School of Medicine'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': '8 weeks', 'eventGroups': [{'id': 'EG000', 'title': 'Treatment: L-glutamine', 'description': 'Patients will receive an 8-week course of oral L-glutamine 10 grams TID\n\nL-Glutamine: Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children \\< 15 years of age.', 'otherNumAtRisk': 13, 'deathsNumAtRisk': 13, 'otherNumAffected': 1, 'seriousNumAtRisk': 13, 'deathsNumAffected': 1, 'seriousNumAffected': 4}], 'otherEvents': [{'term': 'Sexually Transmitted Disease Related Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders'}], 'seriousEvents': [{'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'General disorders'}, {'term': 'Acute Chest Syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'General disorders'}, {'term': 'Sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Erythrocyte Glutamine/Glutamate Ratio at 8 Weeks', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment: L-glutamine', 'description': 'Patients will receive an 8-week course of oral L-glutamine 10 grams TID\n\nL-Glutamine: Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children \\< 15 years of age.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.40', 'spread': '1.6', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '8 weeks', 'description': 'Erythrocyte Glutamine/Glutamate Ratio: a novel biomarker of oxidative stress', 'unitOfMeasure': 'Ratio', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Blood work only available for N=6 participants, as reported here.'}, {'type': 'SECONDARY', 'title': 'Plasma Glutamine', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment: L-glutamine', 'description': 'Patients will receive an 8-week course of oral L-glutamine 10 grams TID\n\nL-Glutamine: Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children \\< 15 years of age.'}], 'classes': [{'categories': [{'measurements': [{'value': '685.8', 'spread': '139', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '8 weeks', 'unitOfMeasure': 'µmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Tricuspid Regurgitant Jet Velocity on Doppler Echocardiography', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment: L-glutamine', 'description': 'Patients will receive an 8-week course of oral L-glutamine 10 grams TID\n\nL-Glutamine: Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children \\< 15 years of age.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.8', 'spread': '0.5', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '8 week', 'description': 'Tricuspid Regurgitant Jet Velocity was measured using Doppler Echocardiography in meters per second.', 'unitOfMeasure': 'Meters per second', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Data available for 11 participants only because one patient missed their appointment for their week 8 echocardiogram and was lost to follow up, and another patient died of sepsis before week 8 visit.'}, {'type': 'SECONDARY', 'title': '6 Minute Walk Distance', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment: L-glutamine', 'description': 'Patients will receive an 8-week course of oral L-glutamine 10 grams TID\n\nL-Glutamine: Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children \\< 15 years of age.'}], 'classes': [{'categories': [{'measurements': [{'value': '366', 'spread': '109', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '8 weeks', 'description': 'The six-minute walk test (6MWT) measures the distance in meters an individual is able to walk over a total of six minutes on a hard, flat surface.', 'unitOfMeasure': 'Meters', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Liver Function Tests', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment: L-glutamine', 'description': 'Patients will receive an 8-week course of oral L-glutamine 10 grams TID\n\nL-Glutamine: Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children \\< 15 years of age.'}], 'classes': [{'title': 'ALT', 'categories': [{'measurements': [{'value': '38', 'spread': '27', 'groupId': 'OG000'}]}]}, {'title': 'AST', 'categories': [{'measurements': [{'value': '63', 'spread': '26', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '8 weeks', 'description': 'Alanine aminotransferase (ALT) Aspartate aminotransferase (AST)', 'unitOfMeasure': 'U/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Renal Function Tests', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment: L-glutamine', 'description': 'Patients will receive an 8-week course of oral L-glutamine 10 grams TID\n\nL-Glutamine: Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children \\< 15 years of age.'}], 'classes': [{'title': 'Creatinine', 'categories': [{'measurements': [{'value': '0.9', 'spread': '0.3', 'groupId': 'OG000'}]}]}, {'title': 'BUN', 'categories': [{'measurements': [{'value': '17', 'spread': '7', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '8 weeks', 'description': 'Creatinine Blood urea nitrogen (BUN)', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Treatment: L-glutamine', 'description': 'Patients will receive an 8-week course of oral L-glutamine 10 grams TID\n\nL-Glutamine: Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children \\< 15 years of age.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '12'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Treatment: L-glutamine', 'description': 'Patients will receive an 8-week course of oral L-glutamine 10 grams TID\n\nL-Glutamine: Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children \\< 15 years of age.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '45.8', 'spread': '13.8', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Erythrocyte Glutamine/Glutamate Ratio', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '1.75', 'spread': '0.6', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Ratio', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'Blood work only available for N=6 participants, as reported here.'}, {'title': 'Plasma Glutamine', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '704.5', 'spread': '137', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'µmol/L', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'Blood work only available for N=6 participants, as reported here.'}, {'title': 'Tricuspid Regurgitant Jet Velocity on Doppler Echocardiography', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '3.0', 'spread': '0.6', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'Tricuspid Regurgitant Jet Velocity was measured using Doppler Echocardiography in meters per second.', 'unitOfMeasure': 'Meters per second', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': '6 Minute Walk Distance', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '386', 'spread': '94', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'The six-minute walk test (6MWT) measures the distance in meters an individual is able to walk over a total of six minutes on a hard, flat surface.', 'unitOfMeasure': 'Meters', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Liver Function Tests', 'classes': [{'title': 'Alanine aminotransferase (ALT)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '40', 'spread': '42', 'groupId': 'BG000'}]}]}, {'title': 'Aspartate aminotransferase (AST)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '73', 'spread': '40', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'U/L', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Renal Function Tests', 'classes': [{'title': 'Creatinine', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '0.9', 'spread': '0.4', 'groupId': 'BG000'}]}]}, {'title': 'Blood urea nitrogen (BUN)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '14', 'spread': '10', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 13}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-03'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-06', 'completionDateStruct': {'date': '2014-03', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-06-08', 'studyFirstSubmitDate': '2010-01-12', 'resultsFirstSubmitDate': '2021-04-20', 'studyFirstSubmitQcDate': '2010-01-13', 'lastUpdatePostDateStruct': {'date': '2021-06-10', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-06-08', 'studyFirstPostDateStruct': {'date': '2010-01-14', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2021-06-10', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2014-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Erythrocyte Glutamine/Glutamate Ratio at 8 Weeks', 'timeFrame': '8 weeks', 'description': 'Erythrocyte Glutamine/Glutamate Ratio: a novel biomarker of oxidative stress'}], 'secondaryOutcomes': [{'measure': 'Plasma Glutamine', 'timeFrame': '8 weeks'}, {'measure': 'Tricuspid Regurgitant Jet Velocity on Doppler Echocardiography', 'timeFrame': '8 week', 'description': 'Tricuspid Regurgitant Jet Velocity was measured using Doppler Echocardiography in meters per second.'}, {'measure': '6 Minute Walk Distance', 'timeFrame': '8 weeks', 'description': 'The six-minute walk test (6MWT) measures the distance in meters an individual is able to walk over a total of six minutes on a hard, flat surface.'}, {'measure': 'Liver Function Tests', 'timeFrame': '8 weeks', 'description': 'Alanine aminotransferase (ALT) Aspartate aminotransferase (AST)'}, {'measure': 'Renal Function Tests', 'timeFrame': '8 weeks', 'description': 'Creatinine Blood urea nitrogen (BUN)'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Pulmonary Hypertension', 'Sickle Cell Disease', 'Thalassemia'], 'conditions': ['Pulmonary Hypertension', 'Sickle Cell Disease', 'Thalassemia']}, 'descriptionModule': {'briefSummary': 'The primary hypothesis of this study is that glutamine supplementation will improve the erythrocyte glutamine/glutamate ratio, a biomarker of oxidative stress, hemolysis and pulmonary hypertension (PH) in sickle cell disease (SCD) and thalassemia (Thal) patients with PH. PH is defined as a tricuspid regurgitant jet velocity (TRV) on Doppler echocardiography \\> 2.5 m/s. We also predict that glutamine therapy will increase arginine bioavailability and subsequently alter sickle red cell endothelial interaction that can be identified using endo-PAT technology through nitric oxide (NO) generation, leading to changes in biological markers, and clinical outcome. Specifically our second hypothesis is that oral glutamine will decrease biomarkers of hemolysis and adhesion molecules, and improve the imbalanced arginine-to-ornithine ratio that occurs in hemolytic anemias, leading to improved arginine bioavailability and clinical endpoints of endothelial dysfunction and PH in patients with SCD and Thal.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '4 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Established diagnosis of SCD (Hb SS, SC or SBeta- thalassemia) or Thal\n* PH documented by echocardiography, defined as at TRV greater than 2.5 m/s\n* Age greater than or equal to 4 years\n\nExclusion Criteria:\n\n* Inability to take or tolerate oral medication\n* Acute crisis or hospitalization within 1 month of enrollment\n* Hepatic dysfunction (SGPT greater than 3X normal)\n* Renal dysfunction (Creatinine greater than 2X normal)\n* Allergy to glutamine\n* Pregnancy or breastfeeding\n* Patients on sildenafil (Viagra), calcium channel blockers, or amino acid/protein supplements (other therapies acceptable if stable more than 3 months)'}, 'identificationModule': {'nctId': 'NCT01048905', 'briefTitle': 'Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension', 'organization': {'class': 'OTHER', 'fullName': "UCSF Benioff Children's Hospital Oakland"}, 'officialTitle': 'Phase 2 Trial for Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension', 'orgStudyIdInfo': {'id': '1R01FD003531-01', 'link': 'https://reporter.nih.gov/quickSearch/1R01FD003531-01', 'type': 'FDA'}, 'secondaryIdInfos': [{'id': 'IRB 2008-059', 'type': 'OTHER', 'domain': 'Institutional Review Board'}, {'id': '1R01FD003531-01', 'link': 'https://reporter.nih.gov/quickSearch/1R01FD003531-01', 'type': 'FDA'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Treatment: L-glutamine', 'description': 'Patients will receive an 8-week course of oral L-glutamine 10 grams TID', 'interventionNames': ['Drug: L-Glutamine']}], 'interventions': [{'name': 'L-Glutamine', 'type': 'DRUG', 'description': 'Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children \\< 15 years of age.', 'armGroupLabels': ['Treatment: L-glutamine']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94608', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital & Research Center Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}], 'overallOfficials': [{'name': 'Claudia Morris, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Emory University'}, {'name': 'Augusta Saulys, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Children's Hosptial & Research Center Oakland"}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': "UCSF Benioff Children's Hospital Oakland", 'class': 'OTHER'}, 'collaborators': [{'name': 'Food and Drug Administration (FDA)', 'class': 'FED'}], 'responsibleParty': {'type': 'SPONSOR'}}}}