Ignite Creation Date:
2025-12-25 @ 2:22 AM
Ignite Modification Date:
2025-12-27 @ 11:55 PM
Study NCT ID:
NCT05676034
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-04-02
First Post:
2022-12-22
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
AMX0035 in Adult Patients With Wolfram Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D014929', 'term': 'Wolfram Syndrome'}], 'ancestors': [{'id': 'D054062', 'term': 'Deaf-Blind Disorders'}, {'id': 'D003638', 'term': 'Deafness'}, {'id': 'D034381', 'term': 'Hearing Loss'}, {'id': 'D006311', 'term': 'Hearing Disorders'}, {'id': 'D004427', 'term': 'Ear Diseases'}, {'id': 'D010038', 'term': 'Otorhinolaryngologic Diseases'}, {'id': 'D015418', 'term': 'Optic Atrophies, Hereditary'}, {'id': 'D009896', 'term': 'Optic Atrophy'}, {'id': 'D009901', 'term': 'Optic Nerve Diseases'}, {'id': 'D003389', 'term': 'Cranial Nerve Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D012678', 'term': 'Sensation Disorders'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D001766', 'term': 'Blindness'}, {'id': 'D014786', 'term': 'Vision Disorders'}, {'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D003919', 'term': 'Diabetes Insipidus'}, {'id': 'D007674', 'term': 'Kidney Diseases'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D003922', 'term': 'Diabetes Mellitus, Type 1'}, {'id': 'D003920', 'term': 'Diabetes Mellitus'}, {'id': 'D044882', 'term': 'Glucose Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D010900', 'term': 'Pituitary Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000723627', 'term': 'sodium phenylbutyrate and taurursodiol'}, {'id': 'C075773', 'term': '4-phenylbutyric acid'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'A 144 week open-label treatment period of AMX0035 administered twice daily'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 12}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2023-03-03', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-03', 'completionDateStruct': {'date': '2026-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-03-28', 'studyFirstSubmitDate': '2022-12-22', 'studyFirstSubmitQcDate': '2022-12-22', 'lastUpdatePostDateStruct': {'date': '2025-04-02', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-01-09', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-07-23', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To evaluate the effect of AMX0035 on residual beta-cell function by monitoring C-peptide levels during a 0-240 minutes mixed-meal tolerance test (MMTT)', 'timeFrame': '24 weeks', 'description': '* C-peptide area under the curve (AUC) response at Week 24 using a 0-240 minute MMTT\n* Change from Baseline in area under the curve (AUC) in delta C-peptide at Week 24 using a 0-240 minute MMTT'}, {'measure': 'To assess the safety and tolerability of AMX0035 administered orally for up to 144 weeks in adult participants with Wolfram syndrome', 'timeFrame': '148 weeks', 'description': '* Incidence and severity of Adverse Events and Serious Adverse Events\n* Incidence of abnormalities in clinical laboratory assessments'}], 'secondaryOutcomes': [{'measure': 'To evaluate the effect of AMX0035 during a 0-240 minute MMTT', 'timeFrame': '48 weeks', 'description': 'C-peptide AUC response to a 240-minute MMTT at Week 48'}, {'measure': 'To assess the effect of AMX0035 on visual acuity', 'timeFrame': '48 weeks', 'description': 'Change from Baseline on best-corrected visual acuity for both eyes measured on the LogMAR scale by sight tests using Snellen chart at Week 48'}, {'measure': 'To evaluate the effect of AMX0035 on total daily insulin dose', 'timeFrame': '24 weeks', 'description': 'Change from Baseline of exogenous insulin dose to Week 24'}, {'measure': 'To evaluate the effect of AMX0035 on glucose range', 'timeFrame': '24 weeks', 'description': 'Change from Baseline to Week 24\n\n* Time in good range\n* Time below range\n* Time above range'}, {'measure': 'To evaluate the effect of AMX0035 on HbA1c levels', 'timeFrame': '24 weeks and 48 weeks', 'description': 'Change from Baseline to Week 24 and Week 48 in HbA1c level'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Wolfram', 'Wolfram syndrome'], 'conditions': ['Wolfram Syndrome']}, 'descriptionModule': {'briefSummary': 'This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.', 'detailedDescription': 'AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and to evaluate the effect of AMX0035 on residual beta cell functions by monitoring c-peptide levels during a 0-240 minute mixed-meal tolerance test. The trial will also assess the effects of AMX0035 on changes to diabetic measurements including daily insulin dose, time in good glucose range, and HbA1c levels. Effect on best-corrected visual acuity in both eyes will also be evaluated.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '17 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Key Inclusion Criteria:\n\n* Definitive diagnosis of Wolfram syndrome\n* Insulin dependent diabetes mellitus due to Wolfram syndrome\n* At least 17 years of age\n* Participant must be willing to wear a CGM device for the duration of the study\n\nKey Exclusion Criteria:\n\n* Presence of pathologies that can alter the enterohepatic circulation of bile acids (e.g., ileal resection and stoma, regional ileitis)\n* Any history of heart failure per New York Heart Association (NYHA)\n* History of or family history of breast and/or ovarian cancer\n* Participant under severe salt restriction where the added salt intake due to treatment would put the patient at risk, in the Investigator's judgment\n* Received treatment with any investigational drug or device within the 30 days (or 5 half-lives, whichever is longer) prior to first dose at Day 1\n* Previous treatment with gene or cellular therapy"}, 'identificationModule': {'nctId': 'NCT05676034', 'briefTitle': 'AMX0035 in Adult Patients With Wolfram Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'Amylyx Pharmaceuticals Inc.'}, 'officialTitle': 'A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome', 'orgStudyIdInfo': {'id': 'A35-008'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'AMX0035', 'description': 'AMX0035 administered by mouth for 144 weeks: Once daily for first 3 weeks and then twice daily for the remainder of the study if tolerated by participant', 'interventionNames': ['Drug: AMX0035']}], 'interventions': [{'name': 'AMX0035', 'type': 'DRUG', 'otherNames': ['Proprietary formulation of taurursodiol and sodium phenylbutyrate'], 'description': 'AMX0035', 'armGroupLabels': ['AMX0035']}]}, 'contactsLocationsModule': {'locations': [{'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}], 'overallOfficials': [{'name': 'Camille Bedrosian, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Amylyx Pharmaceuticals'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Amylyx Pharmaceuticals Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}