AMX0035 in Adult Patients With Wolfram Syndrome

Wolfram Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05676034
• Other study ID #: A35-008
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: March 3, 2023
• Est. completion date: January 30, 2026

Study information

• Verified date: April 2024
• Source: Amylyx Pharmaceuticals Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 12
• Est. completion date: January 30, 2026
• Est. primary completion date: July 17, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 17 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Syndrome
• Wolfram Syndrome

Intervention

Drug:
• AMX0035
AMX0035

Locations

Country	Name	City	State
United States	Washington University	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Amylyx Pharmaceuticals Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate the effect of AMX0035 during a 0-240 minutes mixed-meal tolerance test (MMTT)	C-peptide AUC response at Week 24; Change from Baseline in C-peptide at Week 24	24 weeks
Primary	To assess the safety and tolerability of AMX0035in adult participants with Wolfram syndrome	Incidence and severity of Adverse Events and Serious Adverse Events; Incidence of abnormalities in clinical laboratory assessments	100 weeks
Secondary	To assess the effect size of AMX0035 on visual acuity	Change from Baseline on best-corrected visual acuity (Logmar) at Week 24	24 weeks
Secondary	To evaluate the effect of AMX0035 on total daily insulin dose	Change from Baseline of exogenous insulin dose to Week 24	24 weeks
Secondary	To evaluate the effect of AMX0035 on glucose range	Change from Baseline to Week 24; Time in good range; Time below range; Time above range	24 weeks
Secondary	To evaluate the effect of AMX0035 on HbA1c levels	Change from Baseline to Week 24 in HbA1c level	24 weeks