← 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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up minimum 2 years, until 24 months from randomization of last subject

Awards & highlights

MAGiNE Trial Summary

This trial will study the effects of N-Acetylmannosamine (ManNAc), an uncharged monosaccharide, on patients with GNE myopathy, a rare genetic muscle disease characterized by progressive muscle atrophy and weakness for which there is no current therapy available.

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.Check my eligibility

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.See study design

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.

MAGiNE Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

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.

MAGiNE Trial 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

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Muscle strength of ankle dorsiflexion, knee flexion, knee extension, shoulder abduction, elbow flexion and grip measured by fixed-frame Quantitative Muscle Assessment (QMA)

Secondary outcome measures

Inclusion Body Myositis Functional Rating Scale (IBMFRS)

Other outcome measures

Activities-specific Balance Confidence (ABC) scale

Adult Myopathy Assessment Tool

Adverse Events

+7 more

MAGiNE 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.

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Brigham and Women's HospitalOTHER

1,609 Previous Clinical Trials

11,470,273 Total Patients Enrolled

National Institute of Neurological Disorders and Stroke (NINDS)NIH

1,339 Previous Clinical Trials

649,252 Total Patients Enrolled

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NIH

483 Previous Clinical Trials

1,086,886 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many healthcare facilities have this research protocol been implemented in?

"Patients can participate in this study at University of Iowa, UCLA and the University of Kansas Medical Center. Additionally, there are 8 other sites recruiting for this trial."

Answered by AI

Is it possible for me to join this clinical investigation?

"Eligibility criteria for this research trial requires that participants have myopathy, are between 18 and 70 years old, and there is a need to recruit 51 patients."

Answered by AI

Does this research represent an unprecedented approach?

"ManNAc has been the subject of scientific inquiry since 2022, when Leadiant Biosciences, Inc. initiated a Phase 1 trial with 51 participants. As the drug moved through its approval stages and completed Phase 2 in that same year, there is now one ongoing study sponsored by Leadiant Biosciences to explore ManNAc's efficacy."

Answered by AI

Is there currently an open enrollment period for this experiment?

"According to the clinicaltrials.gov information, this clinical trial is still accepting participants and was initially posted on April 5th 2022 with its most recent update occurring on October 14th 2022."

Answered by AI

What hazards may be associated with ManNAc ingestion?

"Since this Phase 2 trial only has evidence of safety, our team at Power allocated ManNAc a score of 2 on the 1-3 scale."

Answered by AI

What successive experiments have been executed concerning ManNAc?

"Currently, one clinical trial is underway to explore the potential of ManNAc as a therapeutic intervention. This particular study has not yet reached Phase 3 and is being conducted in 10 different centres throughout Salt Lake City, Utah."

Answered by AI

Is the sample population for this research limited to those over 35 years old?

"This medical study has an age limit of 70 years old and is seeking participants that are at least 18."

Answered by AI

How many participants have been registered for this clinical trial?

"To reach the desired sample size, 51 qualified participants are required for this investigation. Candidates can participate from two different sites; University of Iowa in Iowa City and UCLA in Los Angeles."

Answered by AI

Recent research and studies

Molecular Genetics and MetabolismJournal

Safety, Pharmacokinetics and Sialic Acid Production After Oral Administration of N -acetylmannosamine (mannac) to Subjects with GNE Myopathy

Xu, Xin, Amy Q. Wang, Lea L. Latham, Frank Celeste, Carla Ciccone, May Christine Malicdan, Barry Goldspiel, et al.. 2017. “Safety, Pharmacokinetics and Sialic Acid Production After Oral Administration of N -acetylmannosamine (mannac) to Subjects with GNE Myopathy”. Molecular Genetics and Metabolism. Elsevier BV. doi:10.1016/j.ymgme.2017.04.010.

Statistics in MedicineJournal

Bayesian Model of Disease Progression in GNE Myopathy

Quintana, M., J. Shrader, C. Slota, G. Joe, J.C. McKew, M. Fitzgerald, W.A. Gahl, S. Berry, and N. Carrillo. 2018. “Bayesian Model of Disease Progression in GNE Myopathy”. Statistics in Medicine. Wiley. doi:10.1002/sim.8050.

NeurotherapeuticsJournal

GNE Myopathy: Etiology, Diagnosis, and Therapeutic Challenges

Carrillo, Nuria, May C. Malicdan, and Marjan Huizing. 2018. “GNE Myopathy: Etiology, Diagnosis, and Therapeutic Challenges”. Neurotherapeutics. Springer Science and Business Media LLC. doi:10.1007/s13311-018-0671-y.

clinicaltrials.govStudy