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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
- ClinicalTrials.gov ID
- NCT05676034
- Acronym / sponsor ID
- A35-008
- Conditions
- Wolfram Syndrome
- Keywords
- Wolfram, Wolfram syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Amylyx Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- 3 Mar 2023 (actual)
- Primary completion
- 23 Jul 2024 (actual)
- Study completion
- 31 May 2028 (estimated)
- First posted
- 9 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A 208 week open-label treatment period of AMX0035 administered twice daily
| Arm | Type | Receives |
|---|---|---|
| AMX0035 | Experimental | Drug: AMX0035AMX0035 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 |
Who may qualify
- Ages
- 17 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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 (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AMX0035Also: Proprietary formulation of taurursodiol and sodium phenylbutyrateAMX0035 | Drug | AMX0035 |
Primary outcome
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)
Time frame: 24 weeks
* 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
To assess the safety and tolerability of AMX0035 administered orally for up to 208 weeks in adult participants with Wolfram syndrome
Time frame: 208 weeks
* Incidence and severity of Adverse Events and Serious Adverse Events * Incidence of abnormalities in clinical laboratory assessments
5 secondary outcomes
To evaluate the effect of AMX0035 during a 0-240 minute MMTT
Time frame: 48 weeks
To assess the effect of AMX0035 on visual acuity
Time frame: 48 weeks
To evaluate the effect of AMX0035 on total daily insulin dose
Time frame: 24 weeks
To evaluate the effect of AMX0035 on glucose range
Time frame: 24 weeks
To evaluate the effect of AMX0035 on HbA1c levels
Time frame: 24 weeks and 48 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2026.
- Individual participant dataSharing planned: Yes
Related trials for Wolfram Syndrome
Active, not recruitingObservational study
Looking for VUS to Confirm Dominant Wolfram-like Syndrome Instead of Recessive Wolfram Syndrome
Optical Coherence Tomography (OCT) of the Retina, Mutation
Not yet recruitingObservational study
Does Recessive Optic Atrophy Due to WFS1 Exist?
Wolfram Syndrome 1, Optic Atrophies, Hereditary
Not yet recruitingObservational study
Mechanisms of Fecal Incontinence in Wolfram Syndrome
Wolfram Syndrome, Fecal Incontinence
RecruitingObservational study
Wolfram Syndrome and WFS1-related Disorders International Registry and Clinical Study
Wolfram Syndrome, Diabetes Mellitus
RecruitingObservational study
Monogenic Diabetes Misdiagnosed as Type 1
Diabetes Mellitus, Type 1, Monogenic Diabetes
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Camille Bedrosian, MD study director
Amylyx Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AMX0035 in people with Wolfram Syndrome, run by Amylyx Pharmaceuticals Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 14 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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) (time frame: 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Apr 2026.