AMX0035 in Adult Patients With Wolfram Syndrome

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-03-03

Study Completion Date

2026-12-31

Brief Summary

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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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Detailed Description

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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.

Conditions

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Wolfram Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

A 144 week open-label treatment period of AMX0035 administered twice daily

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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AMX0035

AMX0035 administered by mouth for 144 weeks: Once daily for first 3 weeks and then twice daily for the remainder of the study if tolerated by participant

Group Type EXPERIMENTAL

AMX0035

Intervention Type DRUG

AMX0035

Interventions

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AMX0035

Intervention Type DRUG

Other Intervention Names

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Proprietary formulation of taurursodiol and sodium phenylbutyrate

Eligibility Criteria

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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

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

Minimum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No