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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 below
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My muscle strength in one of the specified areas falls within 10-66% of the expected level.
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I have GNE myopathy confirmed by genetic testing.
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I am between 18 and 70 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have ongoing diarrhea or issues absorbing food that could affect my medication intake.
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My kidney function is reduced, with a GFR under 60 mL/min.
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I do not have a mental health or neurological condition that would prevent me from following the study's requirements.
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I have not been hospitalized or needed IV antibiotics in the last 30 days.
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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 weeks
Treatment ~ Varies
Follow Up ~minimum 2 years, until 24 months from randomization of last subject
This trial's timeline: 3 weeks for screening, Varies for treatment, and minimum 2 years, until 24 months from randomization of last subject for reporting.

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 more

Trial 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.

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

ManNAc (Monosaccharide) Clinical Trial Eligibility Overview. Trial Name: NCT04231266 — Phase 2
GNE Myopathy Research Study Groups: Placebo, ManNAc
GNE Myopathy Clinical Trial 2023: ManNAc Highlights & Side Effects. Trial Name: NCT04231266 — Phase 2
ManNAc (Monosaccharide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04231266 — Phase 2
~15 spots leftby Oct 2025