AMX0035 in Adult Patients With Wolfram Syndrome

Phase 2Active Not RecruitingNCT05676034

Amylyx Pharmaceuticals Inc.12 enrolled

Overview

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

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Wolfram Syndrome

Interventions

AMX0035DRUG

AMX0035

Eligibility

Sex: ALLMin age: 17 Years

Medical Language ↔ Plain English

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

Locations (1)

Washington University

St Louis, Missouri, United States

Outcomes

Primary Outcomes

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

Time frame: 24 weeks

To assess the safety and tolerability of AMX0035 administered orally for up to 144 weeks in adult participants with Wolfram syndrome

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

Time frame: 148 weeks

Secondary Outcomes

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

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

Time frame: 48 weeks

To assess the effect of AMX0035 on visual acuity

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

Time frame: 48 weeks

To evaluate the effect of AMX0035 on total daily insulin dose

Change from Baseline of exogenous insulin dose to Week 24

Time frame: 24 weeks

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

Time frame: 24 weeks

To evaluate the effect of AMX0035 on HbA1c levels

Data from ClinicalTrials.gov

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