Wolfram SyndromePhase 2NCT05676034
AMX0035 in Adult Patients With Wolfram Syndrome
A Phase 2 study testing AMX0035 in people with Wolfram Syndrome, run by Amylyx Pharmaceuticals Inc. at 1 site in United States.
Trial summary
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome. Read more about this study
- Who is it for?
- Ages 17 years and older
- Conditions the study is for
- Wolfram Syndrome
- What is being tested?
- AMX0035
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Mar 2023to 31 May 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 17 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
Read the 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.
- Conditions
- Wolfram Syndrome
- Being tested
- AMX0035
- Keywords
- Wolfram, Wolfram syndrome
- Medical subject headings
- Wolfram Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AMX0035Drug
Also called Proprietary formulation of taurursodiol and sodium phenylbutyrate
AMX0035
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: AMX0035 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 208 weeks
Secondary outcomes (5)
To evaluate the effect of AMX0035 during a 0-240 minute MMTT
C-peptide AUC response to a 240-minute MMTT at Week 48
Measured: 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
Measured: 48 weeks
To evaluate the effect of AMX0035 on total daily insulin dose
Change from Baseline of exogenous insulin dose to Week 24
Measured: 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
Measured: 24 weeks
To evaluate the effect of AMX0035 on HbA1c levels
Change from Baseline to Week 24 and Week 48 in HbA1c level
Measured: 24 weeks and 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2023
- Study start3 Mar 2023
- Primary completion23 Jul 2024
- Study completion31 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wolfram SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A 208 week open-label treatment period of AMX0035 administered twice daily
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amylyx Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Camille Bedrosian, MD, Study director, Amylyx Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05676034
- Sponsor's protocol ID
- A35-008
- Status last verified
- Apr 2026
- First submitted
- 22 Dec 2022
- Last updated
- 14 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes