Your session is about to expire
← Back to Search
Monosaccharide
ManNAc for GNE Myopathy (MAGiNE Trial)
Phase 2
Waitlist Available
Led By Francis Rossignol, MD
Research Sponsored by Leadiant Biosciences, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Subjects must have 10.00-65.99% of predicted muscle strength measured by QMA at screening in at least one of the selected muscle groups (ankle dorsiflexion, knee flexion, grip, shoulder abduction and elbow flexion)
Subject has a diagnosis of GNE myopathy based upon a consistent clinical course and biallelic GNE gene mutations that classify as pathogenic or likely pathogenic according to American College of Medical Genetics and Genomics (ACMG) guidelines
Must not have
The presence of persistent diarrhea or malabsorption that could interfere with the subject's ability to absorb drugs or to tolerate ManNAc therapy
Subject with existing renal dysfunction, as defined by glomerular filtration rate (GFR) less than 60 mL/min/1.73 m2 at screening
Timeline
Screening 3 weeks
Treatment Varies
Follow Up minimum 2 years, until 24 months from randomization of last subject
Awards & highlights
Summary
This trial tests ManNAc, a sugar molecule, in patients with GNE myopathy, a rare muscle disease. The goal is to see if ManNAc can help produce more Neu5Ac, improving muscle strength and slowing down muscle weakening. ManNAc has been previously tested for safety in subjects with GNE myopathy.
Who is the study for?
This trial is for adults aged 18-70 with GNE myopathy, a muscle-weakening disease. Participants must have certain levels of muscle strength and be able to travel to the study site without assistance. Women who can get pregnant and men should use birth control during the trial. People with recent severe illness, other conditions affecting physical function, or those on certain medications recently cannot join.
What is being tested?
The study tests ManNAc, a drug in development for treating GNE myopathy against a placebo (a substance with no therapeutic effect). It's randomized and double-blind, meaning neither participants nor researchers know who gets the real drug versus placebo. The goal is to see if ManNAc improves muscle function safely over time.
What are the potential side effects?
Potential side effects are not detailed here but may include reactions related to hypersensitivity to ManNAc or its components like erythritol. Since it's under investigation, part of this study's purpose is also to monitor any adverse effects that could arise from taking ManNAc.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My muscle strength in one of the specified areas falls within 10-66% of the expected level.
Select...
I have GNE myopathy confirmed by genetic testing.
Select...
I am between 18 and 70 years old.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I have ongoing diarrhea or issues absorbing food that could affect my medication intake.
Select...
My kidney function is reduced, with a GFR under 60 mL/min.
Select...
I do not have a mental health or neurological condition that would prevent me from following the study's requirements.
Select...
I have not been hospitalized or needed IV antibiotics in the last 30 days.
Select...
I have a serious health condition that needs urgent surgery or affects my heart, lungs, liver, kidneys, blood, metabolism, or digestive system.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ minimum 2 years, until 24 months from randomization of last subject
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~minimum 2 years, until 24 months from randomization of last subject
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Muscle strength of ankle dorsiflexion, knee flexion, knee extension, shoulder abduction, elbow flexion and grip measured by fixed-frame Quantitative Muscle Assessment (QMA)
Secondary study objectives
Inclusion Body Myositis Functional Rating Scale (IBMFRS)
Other study objectives
Activities-specific Balance Confidence (ABC) scale
Adult Myopathy Assessment Tool
Adverse Events
+7 moreTrial Design
2Treatment groups
Active Control
Placebo Group
Group I: ManNAcActive Control1 Intervention
Oral ManNAc will be administered at a dose of 4 grams three times daily (total of 12 grams daily).
Group II: PlaceboPlacebo Group1 Intervention
Oral Placebo will be administered three times daily.
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
N-Acetylmannosamine (ManNAc) is a treatment under investigation for GNE Myopathy, a genetic muscle disease characterized by progressive muscle atrophy and weakness. ManNAc acts as a precursor in the biosynthesis of N-acetylneuraminic acid (Neu5Ac), a crucial component in glycan sialylation.
This process is essential because mutations in the gene responsible for Neu5Ac biosynthesis lead to the muscle degeneration seen in GNE Myopathy. By supplementing with ManNAc, the aim is to restore normal Neu5Ac levels, thereby improving muscle function and slowing disease progression.
This approach is significant for patients as it targets the underlying biochemical defect, offering a potential disease-modifying therapy.
Abnormal Calcium Handling in Duchenne Muscular Dystrophy: Mechanisms and Potential Therapies.Nitric Oxide (NO) and Duchenne Muscular Dystrophy: NO Way to Go?Galectin-3 and N-acetylglucosamine promote myogenesis and improve skeletal muscle function in the mdx model of Duchenne muscular dystrophy.
Abnormal Calcium Handling in Duchenne Muscular Dystrophy: Mechanisms and Potential Therapies.Nitric Oxide (NO) and Duchenne Muscular Dystrophy: NO Way to Go?Galectin-3 and N-acetylglucosamine promote myogenesis and improve skeletal muscle function in the mdx model of Duchenne muscular dystrophy.
Find a Location
Who is running the clinical trial?
Brigham and Women's HospitalOTHER
1,655 Previous Clinical Trials
11,499,556 Total Patients Enrolled
National Institute of Neurological Disorders and Stroke (NINDS)NIH
1,368 Previous Clinical Trials
652,201 Total Patients Enrolled
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NIH
494 Previous Clinical Trials
1,089,388 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have ongoing diarrhea or issues absorbing food that could affect my medication intake.My kidney function is reduced, with a GFR under 60 mL/min.I haven't used any experimental drugs or devices in the last 90 days.My muscle strength in one of the specified areas falls within 10-66% of the expected level.I can travel to the clinic for my appointments.I have GNE myopathy confirmed by genetic testing.I haven't taken ManNAc, sialic acid, IVIG, or similar compounds in the last 6 months.I do not have a mental health or neurological condition that would prevent me from following the study's requirements.I have another health condition that affects my physical ability.Your blood tests show that you have low red blood cells, platelets, or white blood cells.I am willing to use birth control during the trial.You are allergic to ManNAc or erythritol, or the doctor thinks you might have a condition that could make the treatment risky for you.I can follow the study's requirements for tests, taking medication, and strength assessments.I have not been hospitalized or needed IV antibiotics in the last 30 days.Your liver test results are three times higher than the normal range.I have a serious health condition that needs urgent surgery or affects my heart, lungs, liver, kidneys, blood, metabolism, or digestive system.I have had stem cell or gene therapy in the last year.I am between 18 and 70 years old.
Research Study Groups:
This trial has the following groups:- Group 1: Placebo
- Group 2: ManNAc
Awards:
This trial has 0 awards, including: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.
Share this study with friends
Copy Link
Messenger