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Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Definitive diagnosis of Wolfram syndrome
Insulin dependent diabetes mellitus due to Wolfram syndrome
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights
Study Summary
This trial tests a new drug to see if it can help adults with Wolfram Syndrome.
Who is the study for?
Adults with Wolfram Syndrome can join this trial. They must be at least 17 years old, have a definitive diagnosis, and need insulin for diabetes caused by the syndrome. Participants should also agree to wear a continuous glucose monitoring (CGM) device throughout the study.Check my eligibility
What is being tested?
The trial is testing AMX0035's safety and effectiveness in adults with Wolfram Syndrome. It's an open label Phase II study, which means everyone knows they're getting the treatment and there’s no placebo group.See study design
What are the potential side effects?
While specific side effects of AMX0035 are not listed here, common ones may include digestive issues, headaches, fatigue or allergic reactions. The exact side effects will be monitored closely during the trial.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with Wolfram syndrome.
Select...
I have diabetes that requires insulin because of Wolfram syndrome.
Select...
I am 17 years old or older.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 52 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
To assess the safety and tolerability of AMX0035in adult participants with Wolfram syndrome
To evaluate the effect of AMX0035 during a 0-240 minutes mixed-meal tolerance test (MMTT)
Secondary outcome measures
To assess the effect size of AMX0035 on visual acuity
To evaluate the effect of AMX0035 on HbA1c levels
To evaluate the effect of AMX0035 on glucose range
+1 moreSide effects data
From 2019 Phase 2 trial • 137 Patients • NCT0312751428%
Fall
21%
Diarrhoea
20%
Muscular weakness
18%
Nausea
15%
Headache
13%
Constipation
11%
Salivary hypersecretion
11%
Viral Upper Respiratory Tract Infection
10%
Dizziness
10%
Dyspnoea
8%
Abdominal pain
8%
Decreased appetite
8%
Fatigue
7%
Laceration
6%
Back pain
6%
Abdominal discomfort
6%
Muscle spasms
6%
Arthralgia
6%
Musculoskeletal pain
6%
Contusion
6%
Urinary Tract Infection
6%
Cough
6%
Weight decreased
6%
Asthenia
6%
Rash
4%
Aspartate aminotransferase increased
4%
Musculoskeletal chest pain
4%
Abdominal pain upper
4%
Upper Respiratory Tract Infection
4%
Alanine aminotransferase increased
3%
Dysphagia
3%
Dry mouth
3%
Insomnia
2%
Respiratory failure
2%
Neck pain
2%
Oedema peripheral
1%
Pneumoperitoneum
1%
Skull fracture
1%
Bacteraemia
1%
Cellulitis
1%
Pneumonia
1%
Diverticulitis
1%
Nephrolithiasis
1%
Respiratory arrest
1%
Vision blurred
1%
Subdural haematoma
1%
Stoma site haemorrhage
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
AMX0035
Trial Design
1Treatment groups
Experimental Treatment
Group I: AMX0035Experimental Treatment1 Intervention
AMX0035 administered by mouth for 48 weeks: Once daily for first 3 weeks and then twice daily for the remainder of the study if tolerated by participant
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AMX0035
2018
Completed Phase 3
~360
Find a Location
Who is running the clinical trial?
Amylyx Pharmaceuticals Inc.Lead Sponsor
9 Previous Clinical Trials
2,169 Total Patients Enrolled
Mathias Leinders, Dr.Study DirectorAmylyx Pharmaceuticals
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have been diagnosed with Wolfram syndrome.I have diabetes that requires insulin because of Wolfram syndrome.I haven't taken any experimental drugs or used experimental devices recently.I have a history of heart failure.I have conditions affecting bile acid movement due to gut surgery or inflammation.I am 17 years old or older.I have had gene or cellular therapy before.I am on a strict low-salt diet and additional salt could harm me.I or someone in my family has had breast or ovarian cancer.
Research Study Groups:
This trial has the following groups:- Group 1: AMX0035
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any unfilled vacancies for subjects in this investigation?
"All indications from clinicaltrials.gov suggest that this trial is actively recruiting patients; the study was first posted on March 1st, 2023 and updated for the last time on March 14th of that same year."
Answered by AI
Has AMX0035 been granted permission by the FDA for public consumption?
"Our assessment of the safety of AMX0035 rates it a 2, as there is clinical evidence validating its security but none yet to prove its efficacy."
Answered by AI
How many participants are included in the current clinical experiment?
"Affirmative. According to clinicaltrials.gov, this medical experiment is presently open for recruitment and was first listed on March 1st of 2023 with the last update occurring two weeks later on March 14th. A total of 12 participants are being recruited from a single site."
Answered by AI
What objectives is this clinical experiment aiming to fulfill?
"As per the research sponsor, Amylyx Pharmaceuticals Inc., this 28 weeks long clinical trial will primarily examine the safety and tolerability of AMX0035 in adults with Wolfram Syndrome. Subsequently, secondary objectives, such as assessing changes to total daily insulin dose (from Baseline to Week 24), glucose range fluctuations (Time in good range , Time below range, Time above range) and HbA1c levels modifications (Change from Baseline to Week 24), will be monitored."
Answered by AI
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