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NCT Number: NCT05676034

AMX0035 in Adult Patients With Wolfram Syndrome

This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Wolfram Syndrome Abnormalities, Multiple Blindness Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cranial Nerve Diseases Deaf-Blind Disorders Deafness Diabetes Insipidus Diabetes Mellitus Diabetes Mellitus, Type 1 Ear Diseases Endocrine System Diseases Eye Diseases Eye Diseases, Hereditary Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Glucose Metabolism Disorders Hearing Disorders Hearing Loss Heredodegenerative Disorders, Nervous System Kidney Diseases Male Urogenital Diseases Metabolic Diseases Nervous System Diseases Neurodegenerative Diseases Neurologic Manifestations Nutritional and Metabolic Diseases Optic Atrophies, Hereditary Optic Atrophy Optic Nerve Diseases Otorhinolaryngologic Diseases Pituitary Diseases Sensation Disorders Urogenital Diseases Urologic Diseases Vision Disorders

Age range

17 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Washington University

St Louis, Missouri, 63110, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • Definitive diagnosis of Wolfram syndrome
  • Insulin dependent diabetes mellitus due to Wolfram syndrome
  • At least 17 years of age
  • Participant must be willing to wear a CGM device for the duration of the study

Key Exclusion Criteria:

  • Presence of pathologies that can alter the enterohepatic circulation of bile acids (e.g., ileal resection and stoma, regional ileitis)
  • Any history of heart failure per New York Heart Association (NYHA)
  • History of or family history of breast and/or ovarian cancer
  • Participant under severe salt restriction where the added salt intake due to treatment would put the patient at risk, in the Investigator's judgment
  • 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
  • Previous treatment with gene or cellular therapy

Treatment and study plan

AMX0035

Drug

AMX0035

Other names: Proprietary formulation of taurursodiol and sodium phenylbutyrate

Primary outcomes

  1. 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)

    Time frame: 24 weeks

    • C-peptide area under the curve (AUC) response at Week 24 using a 0-240 minute MMTT
    • Change from Baseline in area under the curve (AUC) in delta C-peptide at Week 24 using a 0-240 minute MMTT

  2. To assess the safety and tolerability of AMX0035 administered orally for up to 208 weeks in adult participants with Wolfram syndrome

    Time frame: 208 weeks

    • Incidence and severity of Adverse Events and Serious Adverse Events
    • Incidence of abnormalities in clinical laboratory assessments

Secondary outcomes

  1. To evaluate the effect of AMX0035 during a 0-240 minute MMTT

    Time frame: 48 weeks

    C-peptide AUC response to a 240-minute MMTT at Week 48

  2. To assess the effect of AMX0035 on visual acuity

    Time frame: 48 weeks

    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

  3. To evaluate the effect of AMX0035 on total daily insulin dose

    Time frame: 24 weeks

    Change from Baseline of exogenous insulin dose to Week 24

  4. To evaluate the effect of AMX0035 on glucose range

    Time frame: 24 weeks

    Change from Baseline to Week 24

    • Time in good range
    • Time below range
    • Time above range

  5. To evaluate the effect of AMX0035 on HbA1c levels

    Time frame: 24 weeks and 48 weeks

    Change from Baseline to Week 24 and Week 48 in HbA1c level

Sponsors and collaborators

Lead sponsor

Amylyx Pharmaceuticals Inc.

Industry

Registry information

Official study title

A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome

Important dates

Study start
2023
Primary completion
2024
Study completion
2028
First posted
Jan 9, 2023
Registry last updated
Apr 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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