Ignite Creation Date:
2025-12-25 @ 2:22 AM
Ignite Modification Date:
2025-12-26 @ 12:55 AM
Study NCT ID:
NCT05676034
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-04-02
First Post:
2022-12-22
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
AMX0035 in Adult Patients With Wolfram Syndrome
Sponsor:
Amylyx Pharmaceuticals Inc.
Organization:
Study Overview
Official Title:
A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
Detailed 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.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: