AMX0035 in Adult Patients With Wolfram Syndrome

NCT05676034 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: A35-008
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Wolfram Syndrome

Keywords

Wolfram; Wolfram syndrome

Outcome Measures

Primary Outcomes (2)

• 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)

  * 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

  24 weeks

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

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

  208 weeks

Secondary Outcomes (5)

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

  48 weeks

• To assess the effect of AMX0035 on visual acuity

  48 weeks

• To evaluate the effect of AMX0035 on total daily insulin dose

  24 weeks

• To evaluate the effect of AMX0035 on glucose range

  24 weeks

• To evaluate the effect of AMX0035 on HbA1c levels

  24 weeks and 48 weeks

Study Arms (1)

AMX0035

EXPERIMENTAL

AMX0035 administered by mouth for 208 weeks: Once daily for first 3 weeks and then twice daily for the remainder of the study if tolerated by participant

Drug: AMX0035

Interventions

AMX0035 DRUG

AMX0035

Also known as: Proprietary formulation of taurursodiol and sodium phenylbutyrate

AMX0035

Eligibility Criteria

• Age: 17 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Amylyx Pharmaceuticals Inc.: lead

Study Sites (1)

Washington University

St Louis, Missouri, 63110, United States

Location

MeSH Terms

Conditions

Wolfram Syndrome

Interventions

sodium phenylbutyrate and taurursodiol; 4-phenylbutyric acid

Condition Hierarchy (Ancestors)

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

Study Officials

• Camille Bedrosian, MD

  Amylyx Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: A 208 week open-label treatment period of AMX0035 administered twice daily

Study Record Dates

• First Submitted: December 22, 2022
• First Posted: January 9, 2023
• Study Start: March 3, 2023
• Primary Completion: July 23, 2024
• Study Completion (Estimated): May 31, 2028
• Last Updated: April 14, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

US (1)