Ignite Creation Date:
2025-12-24 @ 10:20 PM
Ignite Modification Date:
2025-12-28 @ 5:43 AM
Study NCT ID:
NCT04499235
Status:
TERMINATED
Last Update Posted:
2023-09-14
First Post:
2020-07-31
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study to Assess the Therapeutic Effect and Safety of Adjunctive AKST4290 in Subjects With Bullous Pemphigoid
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D010391', 'term': 'Pemphigoid, Bullous'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D012871', 'term': 'Skin Diseases'}], 'ancestors': [{'id': 'D012872', 'term': 'Skin Diseases, Vesiculobullous'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000068656', 'term': 'Mometasone Furoate'}], 'ancestors': [{'id': 'D011244', 'term': 'Pregnadienediols'}, {'id': 'D011245', 'term': 'Pregnadienes'}, {'id': 'D011278', 'term': 'Pregnanes'}, {'id': 'D013256', 'term': 'Steroids'}, {'id': 'D000072473', 'term': 'Fused-Ring Compounds'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'info@alkahest.com', 'phone': '(650) 801-0474', 'title': 'Head of Communications', 'organization': 'Alkahest, Inc.'}, 'certainAgreement': {'otherDetails': 'The Clinical Trial Agreement contains language that restricts the PI from discussing or publishing Sponsor confidential and/or proprietary information. The embargo period may be extended by mutual agreement of the Sponsor and PI.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': '5 weeks', 'eventGroups': [{'id': 'EG000', 'title': 'Mometasone Furoate + AKST4290', 'description': 'Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nAKST4290: Oral AKST4290', 'otherNumAtRisk': 5, 'deathsNumAtRisk': 5, 'otherNumAffected': 2, 'seriousNumAtRisk': 5, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Mometasone Furoate + Placebo', 'description': 'Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nPlacebo: Oral placebo', 'otherNumAtRisk': 1, 'deathsNumAtRisk': 1, 'otherNumAffected': 0, 'seriousNumAtRisk': 1, 'deathsNumAffected': 1, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Blood potassium decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mometasone Furoate + AKST4290', 'description': 'Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nAKST4290: Oral AKST4290'}, {'id': 'OG001', 'title': 'Mometasone Furoate + Placebo', 'description': 'Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nPlacebo: Oral placebo'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline to up to 3 weeks (until disease control)', 'description': 'Disease control is defined as ≤ 3 new blisters/eczematous lesions/urticarial plaques/day and healing of existing blisters/eczematous lesions/urticarial plaques without requiring rescue therapy.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants With TEAEs, Assessed by Seriousness and Severity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mometasone Furoate + AKST4290', 'description': 'Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nAKST4290: Oral AKST4290'}, {'id': 'OG001', 'title': 'Mometasone Furoate + Placebo', 'description': 'Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nPlacebo: Oral placebo'}], 'classes': [{'title': 'Subjects Reporting at Least One TEAE', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Subjects Reporting at Least One Serious TEAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'TEAE by Severity: Mild', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'TEAE by Severity: Moderate', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'TEAE by Severity: Severe', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline to 5 weeks', 'description': 'Treatment-emergent AEs summarized by MedDRA coding terms; separate tabulations produced for incidence, seriousness and severity of AEs', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The Safety Evaluable Set includes all randomized subjects who receive at least 1 dose of the study agent (AKST4290 or placebo)'}, {'type': 'SECONDARY', 'title': 'Time to Disease Control', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mometasone Furoate + AKST4290', 'description': 'Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nAKST4290: Oral AKST4290'}, {'id': 'OG001', 'title': 'Mometasone Furoate + Placebo', 'description': 'Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nPlacebo: Oral placebo'}], 'classes': [{'title': 'Time (Days) to Disease Control: 8', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Time (Days) to Disease Control: 11', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Time (Days) to Disease Control: 15', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Time (Days) to Disease Control: 16', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Time (Days) to Disease Control: 22', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline to up to 3 weeks (until disease control)', 'description': 'Time to disease control by treatment day/week. The time to disease control is calculated as the date of disease control minus Date of Visit 2 (Baseline (Day 1)) plus 1.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Analysis population includes subjects who achieved disease control with and without rescue therapy.'}, {'type': 'SECONDARY', 'title': 'Time to Rescue Therapy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mometasone Furoate + AKST4290', 'description': 'Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nAKST4290: Oral AKST4290'}, {'id': 'OG001', 'title': 'Mometasone Furoate + Placebo', 'description': 'Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nPlacebo: Oral placebo'}], 'classes': [{'title': 'Time (Days) to Rescue Therapy: 4', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Time (Days) to Rescue Therapy 8', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Time (Days) to Rescue Therapy 9', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.', 'description': 'Time to rescue therapy by treatment day/week. The time to rescue therapy is calculated as the start date of the first rescue therapy minus Date of Visit 2 (Baseline (Day 1)) plus 1.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'The Bullous Pemphigoid Disease Area Index (BPDAI) Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mometasone Furoate + AKST4290', 'description': 'Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nAKST4290: Oral AKST4290'}, {'id': 'OG001', 'title': 'Mometasone Furoate + Placebo', 'description': 'Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nPlacebo: Oral placebo'}], 'classes': [{'title': 'Total BPDAI Skin Blister Score: Baseline', 'categories': [{'measurements': [{'value': '15', 'spread': '12.83', 'groupId': 'OG000'}, {'value': '13.0', 'groupId': 'OG001'}]}]}, {'title': 'Total BPDAI Skin Blister Score: EOT', 'categories': [{'measurements': [{'value': '7.4', 'spread': '7.02', 'groupId': 'OG000'}, {'value': '8.0', 'groupId': 'OG001'}]}]}, {'title': 'Total BPDAI Skin Urticarial Score: Baseline', 'categories': [{'measurements': [{'value': '16.4', 'spread': '9.15', 'groupId': 'OG000'}, {'value': '14.0', 'groupId': 'OG001'}]}]}, {'title': 'Total BPDAI Skin Urticarial Score: EOT', 'categories': [{'measurements': [{'value': '9.2', 'spread': '6.38', 'groupId': 'OG000'}, {'value': '12.0', 'groupId': 'OG001'}]}]}, {'title': 'Total BPDAI Mucosal Activity Score: Baseline', 'categories': [{'measurements': [{'value': '3.4', 'spread': '7.60', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Total BPDAI Mucosal Activity Score: EOT', 'categories': [{'measurements': [{'value': '1.6', 'spread': '3.58', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Total BPDAI Damage Score: Baseline', 'categories': [{'measurements': [{'value': '5.0', 'spread': '4.18', 'groupId': 'OG000'}, {'value': '9.0', 'groupId': 'OG001'}]}]}, {'title': 'Total BPDAI Damage Score: EOT', 'categories': [{'measurements': [{'value': '5.0', 'spread': '3.81', 'groupId': 'OG000'}, {'value': '8.0', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.', 'description': 'Change from baseline in BPDAI score at End of Treatment (EOT). Subscales for the BPDAI include the skin blister score (range 0-120), skin urticarial score (range 0-120), mucosal activity score (range 0-120), and damage score (range 0-12). Higher scores indicate greater disease activity or damage.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'The Bullous Pemphigoid Disease Area Index Visual Analog Scale (BPDAI-VAS)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mometasone Furoate + AKST4290', 'description': 'Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nAKST4290: Oral AKST4290'}, {'id': 'OG001', 'title': 'Mometasone Furoate + Placebo', 'description': 'Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nPlacebo: Oral placebo'}], 'classes': [{'title': 'BPDAI-VAS Baseline', 'categories': [{'measurements': [{'value': '19.4', 'spread': '5.68', 'groupId': 'OG000'}, {'value': '15.0', 'groupId': 'OG001'}]}]}, {'title': 'BPDAI-VAS EOT', 'categories': [{'measurements': [{'value': '9.2', 'spread': '3.42', 'groupId': 'OG000'}, {'value': '4.0', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.', 'description': 'Change from baseline in pruritus as evaluated by the BPDAI-VAS at End of Treatment (EOT). EOT occurs at disease control (up to 3 weeks) or at Week 3 when the subject is discontinued from treatment due to not reaching disease control. Scores for the BPDAI-VAS can range from 0 to 30, with higher scores indicating a worse condition.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Total Cumulative Steroid Exposure', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mometasone Furoate + AKST4290', 'description': 'Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nAKST4290: Oral AKST4290'}, {'id': 'OG001', 'title': 'Mometasone Furoate + Placebo', 'description': 'Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nPlacebo: Oral placebo'}], 'classes': [{'categories': [{'measurements': [{'value': '260', 'spread': '210', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.', 'description': 'Total cumulative steroid exposure (cortisol equivalent/kg) by treatment group', 'unitOfMeasure': 'mg cortisol equivalent/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Total cumulative steroid exposure is calculated based on oral route treatment from Day 1 to EOT/disease control. Subjects with no oral route treatment were excluded from analysis.'}, {'type': 'SECONDARY', 'title': 'Maximum Daily Steroid Dose', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mometasone Furoate + AKST4290', 'description': 'Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nAKST4290: Oral AKST4290'}, {'id': 'OG001', 'title': 'Mometasone Furoate + Placebo', 'description': 'Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nPlacebo: Oral placebo'}], 'classes': [{'title': 'Maximum Daily Steroid Dose Occurred: Day 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '175', 'groupId': 'OG000'}]}]}, {'title': 'Maximum Daily Steroid Dose Occurred: Day 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '150', 'groupId': 'OG000'}]}]}, {'title': 'Maximum Daily Steroid Dose Occurred: Day 11', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '150', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.', 'description': 'Evaluation of maximum daily steroid dose at baseline, by treatment week, and at disease control. Study Day 1 is defined as the initiation of study treatment. 1 mg/kg prednisolon(e) = 5 mg/kg cortisone.', 'unitOfMeasure': 'mg cortisol equivalent/kg', 'reportingStatus': 'POSTED', 'populationDescription': 'The analysis population includes subjects who received systemic steroids. In the AKST4290 group, 3 subjects received systemic steroids. In the Placebo group, the single subject did not have systemic steroid exposure.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Mometasone Furoate + AKST4290', 'description': 'Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nAKST4290: Oral AKST4290'}, {'id': 'FG001', 'title': 'Mometasone Furoate + Placebo', 'description': 'Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nPlacebo: Oral placebo'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'preAssignmentDetails': 'Enrolled participants per the protocol are participants who are randomized to mometasone furoate + AKST4290 or mometasone furoate + placebo. Subjects screened but not randomized are not reflected in Participant Flow. The study enrolled 6 subjects of the planned 30 subjects before study prematurely terminated by the Sponsor.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Mometasone Furoate + AKST4290', 'description': 'Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nAKST4290: Oral AKST4290'}, {'id': 'BG001', 'title': 'Mometasone Furoate + Placebo', 'description': 'Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.\n\nMometasone furoate: Topical mometasone furoate\n\nPlacebo: Oral placebo'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '66', 'spread': '7.78', 'groupId': 'BG000'}, {'value': '60', 'groupId': 'BG001'}, {'value': '65', 'spread': '7.38', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2020-05-15', 'size': 4922193, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2022-03-29T21:30', 'hasProtocol': True}, {'date': '2021-08-10', 'size': 1026358, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2022-03-29T21:31', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 6}}, 'statusModule': {'whyStopped': 'The study was prematurely terminated by the Sponsor due to operational challenges stemming from the coronavirus disease 2019 (COVID-19) pandemic, treatment limitations, rarity of the disease, and drug supply considerations.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2020-01-30', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-09', 'completionDateStruct': {'date': '2021-04-14', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-09-01', 'studyFirstSubmitDate': '2020-07-31', 'resultsFirstSubmitDate': '2022-03-30', 'studyFirstSubmitQcDate': '2020-07-31', 'lastUpdatePostDateStruct': {'date': '2023-09-14', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-09-01', 'studyFirstPostDateStruct': {'date': '2020-08-05', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-09-14', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-03-29', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy', 'timeFrame': 'Baseline to up to 3 weeks (until disease control)', 'description': 'Disease control is defined as ≤ 3 new blisters/eczematous lesions/urticarial plaques/day and healing of existing blisters/eczematous lesions/urticarial plaques without requiring rescue therapy.'}], 'secondaryOutcomes': [{'measure': 'Number of Participants With TEAEs, Assessed by Seriousness and Severity', 'timeFrame': 'Baseline to 5 weeks', 'description': 'Treatment-emergent AEs summarized by MedDRA coding terms; separate tabulations produced for incidence, seriousness and severity of AEs'}, {'measure': 'Time to Disease Control', 'timeFrame': 'Baseline to up to 3 weeks (until disease control)', 'description': 'Time to disease control by treatment day/week. The time to disease control is calculated as the date of disease control minus Date of Visit 2 (Baseline (Day 1)) plus 1.'}, {'measure': 'Time to Rescue Therapy', 'timeFrame': 'Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.', 'description': 'Time to rescue therapy by treatment day/week. The time to rescue therapy is calculated as the start date of the first rescue therapy minus Date of Visit 2 (Baseline (Day 1)) plus 1.'}, {'measure': 'The Bullous Pemphigoid Disease Area Index (BPDAI) Score', 'timeFrame': 'Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.', 'description': 'Change from baseline in BPDAI score at End of Treatment (EOT). Subscales for the BPDAI include the skin blister score (range 0-120), skin urticarial score (range 0-120), mucosal activity score (range 0-120), and damage score (range 0-12). Higher scores indicate greater disease activity or damage.'}, {'measure': 'The Bullous Pemphigoid Disease Area Index Visual Analog Scale (BPDAI-VAS)', 'timeFrame': 'Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.', 'description': 'Change from baseline in pruritus as evaluated by the BPDAI-VAS at End of Treatment (EOT). EOT occurs at disease control (up to 3 weeks) or at Week 3 when the subject is discontinued from treatment due to not reaching disease control. Scores for the BPDAI-VAS can range from 0 to 30, with higher scores indicating a worse condition.'}, {'measure': 'Total Cumulative Steroid Exposure', 'timeFrame': 'Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.', 'description': 'Total cumulative steroid exposure (cortisol equivalent/kg) by treatment group'}, {'measure': 'Maximum Daily Steroid Dose', 'timeFrame': 'Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.', 'description': 'Evaluation of maximum daily steroid dose at baseline, by treatment week, and at disease control. Study Day 1 is defined as the initiation of study treatment. 1 mg/kg prednisolon(e) = 5 mg/kg cortisone.'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Autoimmune disease', 'Skin disease', 'Vesiculobullous'], 'conditions': ['Pemphigoid, Bullous']}, 'descriptionModule': {'briefSummary': 'This study will evaluate the therapeutic effect and safety of adjunctive AKST4290 in subjects with bullous pemphigoid (BP).', 'detailedDescription': 'This is a randomized, double-blind, placebo-controlled study to assess the therapeutic effect and safety of adjunctive AKST4290 in subjects with bullous pemphigoid (BP). Subjects will receive topical mometasone furoate cream (MFC) therapy concurrently with study agent (placebo or AKST4290) in an inpatient setting until disease control is reached (duration of inpatient stay is dependent upon individual disease course, but is estimated between 1-3 weeks).\n\nSubjects will receive rescue therapy at any time if their clinical condition worsens or if their clinical condition fails to improve by the completion of Week 1 on study treatment, as assessed by the investigator. Rescue therapy will consist of whole-body clobetasol propionate cream (CPC) (15-50g) and/or oral prednisone (0.5 mg/kg per day), as determined by the investigator. Subjects who receive rescue therapy will remain in the study until disease control, unless they are withdrawn or withdraw from participation.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '95 Years', 'minimumAge': '60 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Clinical diagnosis of mild to moderate BP at screening.\n* Treatment naïve or initiation of whole-body high potency topical steroid treatment ≤ 7 days of screening (lesion-only treatment for any amount of time with any topical steroids prior to screening is allowed without restriction).\n* Provide a signed and dated informed consent form in accordance with local regulations and/or IRB/IEC guidelines.\n\nExclusion Criteria:\n\n* Severe BP.\n* Initiation of gliptins and other treatments (e.g., etanercept, sulfasalazine, furosemide, penicillin) that can trigger BP if this treatment was started within 4 weeks prior to screening and is considered possibly related to the onset of BP.\n* Any concomitant medications in the last 3 months prior to screening and assessed by the investigator as possibly related to the development of BP.\n* Planned use of intravenous immunoglobulin or other concomitant treatments for BP (i.e., doxycycline, dapsone) during the study period.\n* Use of systemic immunosuppressants (i.e., mycophenolate, azathioprine, methotrexate) within 4 weeks prior to screening.\n* Treatment with rituximab within 1 year prior to screening.\n* Subjects taking warfarin.\n* Use of systemic steroids (\\>10 mg prednisone or equivalent/day) within 14 days of first dose of study agent or known diseases (other than BP) that could require the use of systemic steroids within the study period.\n* Clinically relevant abnormal laboratory value at screening, including hematology, blood chemistry, or urinalysis (laboratory testing may be repeated once during the screening phase).\n* Participation in studies of investigational drugs must have been discontinued within 30 days or 5 half lives of the drug (whichever was longer) prior to screening.'}, 'identificationModule': {'nctId': 'NCT04499235', 'briefTitle': 'A Study to Assess the Therapeutic Effect and Safety of Adjunctive AKST4290 in Subjects With Bullous Pemphigoid', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alkahest, Inc.'}, 'officialTitle': 'Double-Blind, Randomized, Placebo-Controlled Trial of AKST4290 for Adjunctive Treatment of Mild to Moderate Bullous Pemphigoid', 'orgStudyIdInfo': {'id': 'AKST4290-221'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Mometasone furoate + AKST4290', 'description': 'Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.', 'interventionNames': ['Drug: Mometasone furoate', 'Drug: AKST4290']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Mometasone furoate + Placebo', 'description': 'Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.', 'interventionNames': ['Drug: Mometasone furoate', 'Drug: Placebo']}], 'interventions': [{'name': 'Mometasone furoate', 'type': 'DRUG', 'description': 'Topical mometasone furoate', 'armGroupLabels': ['Mometasone furoate + AKST4290', 'Mometasone furoate + Placebo']}, {'name': 'AKST4290', 'type': 'DRUG', 'description': 'Oral AKST4290', 'armGroupLabels': ['Mometasone furoate + AKST4290']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Oral placebo', 'armGroupLabels': ['Mometasone furoate + Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '01307', 'city': 'Dresden', 'country': 'Germany', 'facility': 'Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie', 'geoPoint': {'lat': 51.05089, 'lon': 13.73832}}, {'zip': '40225', 'city': 'Düsseldorf', 'country': 'Germany', 'facility': 'Universitätsklinikum Düsseldorf Klinik für Dermatologie', 'geoPoint': {'lat': 51.22172, 'lon': 6.77616}}, {'zip': '91054', 'city': 'Erlangen', 'country': 'Germany', 'facility': 'Universitätsklinikum Erlangen - Hautklinik', 'geoPoint': {'lat': 49.59099, 'lon': 11.00783}}, {'zip': '79104', 'city': 'Freiburg im Breisgau', 'country': 'Germany', 'facility': 'Universitätsklinikum Freiburg Klinik für Dermatologie und Venerologie', 'geoPoint': {'lat': 47.9959, 'lon': 7.85222}}, {'zip': '23538', 'city': 'Lübeck', 'country': 'Germany', 'facility': 'Universitätsklinikum Schleswig-Holstein Klinik für Dermatologie, Allergologie und Venerologie (Hautklinik) Exzellenzzentrum Entzündungsmedizin', 'geoPoint': {'lat': 53.86893, 'lon': 10.68729}}, {'zip': '39120', 'city': 'Magdeburg', 'country': 'Germany', 'facility': 'Universitätsklinikum Magdeburg A.ö.R. Universitätshautklinik', 'geoPoint': {'lat': 52.13129, 'lon': 11.63189}}, {'zip': '55131', 'city': 'Mainz', 'country': 'Germany', 'facility': 'Universitätsmedizin der Johannes Gutenberg-Universität Mainz Hautklinik Clinical Research Center (CRC)', 'geoPoint': {'lat': 49.98185, 'lon': 8.28008}}, {'zip': '97080', 'city': 'Würzburg', 'country': 'Germany', 'facility': 'Universitätsklinikum Würzburg Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie', 'geoPoint': {'lat': 49.79391, 'lon': 9.95121}}], 'overallOfficials': [{'name': 'Alkahest Medical Monitor', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Alkahest, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alkahest, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}