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BBP-418 for Limb-Girdle Muscular Dystrophy

Phase 2
Waitlist Available
Led By Amy Harper, MD
Research Sponsored by ML Bio Solutions, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Have a genetically confirmed diagnosis of LGMD2I and be clinically affected (defined as demonstrating clinical weakness on bedside evaluation in either a limb-girdle pattern, or in a distal extremity)
Able to complete the 10-meter walk test in ≤ 12 seconds unaided ("moderate disease") or are with "severe disease"/non-ambulatory as defined by being unable to complete the 10-meter walk unaided in >12 seconds
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 60 months
Awards & highlights

Study Summary

This trial will test the safety and tolerability of a new drug, BBP-418, for people with a certain kind of muscle dystrophy. There is currently no approved treatment for this condition.

Who is the study for?
This trial is for individuals over 30 kg with genetically confirmed LGMD2I, showing clinical weakness. Participants must be able to walk 10 meters unaided in ≤12 seconds or be non-ambulatory. They should agree to contraception use and not have significant other diseases or recent drug abuse history.Check my eligibility
What is being tested?
BBP-418 is being tested for safety and tolerability in different dose levels on ambulatory and non-ambulatory patients with LGMD2I, a condition without approved treatments. This open label study allows all participants to receive the drug.See study design
What are the potential side effects?
Specific side effects of BBP-418 are not listed but will include any adverse reactions observed during the trial as doses increase. Safety assessments like muscle biopsies may also carry risks such as bleeding or infection.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a genetic diagnosis of LGMD2I and show signs of muscle weakness.
Select...
I can walk 10 meters in 12 seconds or less, or I cannot walk this distance unaided.
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I weigh more than 30 kilograms.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~60 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 60 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
Secondary outcome measures
Changes in pharmacodynamic parameters by assessing changes in levels of N-terminal fragment of alpha dystroglycan (α-DG)
Changes in pharmacodynamic parameters by assessing muscle biopsy of the tibialis anterior
Pharmacokinetic profile of BBP-418 by assessment of area under the curve (AUC)
+1 more

Trial Design

3Treatment groups
Experimental Treatment
Group I: Cohort 3Experimental Treatment1 Intervention
Subjects will receive 12 grams of BBP-418 twice daily (BID, at least 8 hours apart) x 90 days, then 12 grams BID of BBP-418 daily until study completion.
Group II: Cohort 2Experimental Treatment1 Intervention
Subjects will receive 6 grams of BBP-418 twice daily (BID, at least 8 hours apart) x 90 days, then 12 grams BID of BBP-418 daily until study completion.
Group III: Cohort 1Experimental Treatment1 Intervention
Subjects will receive 6 grams of BBP-418 once daily x 90 days, then 12 grams twice daily (BID, a least 8 hours apart) of BBP-418 daily until study completion.

Find a Location

Who is running the clinical trial?

ML Bio Solutions, Inc.Lead Sponsor
2 Previous Clinical Trials
182 Total Patients Enrolled
Amy Harper, MDPrincipal InvestigatorVirginia Commonwealth University

Media Library

BBP-418 (Unknown) Clinical Trial Eligibility Overview. Trial Name: NCT04800874 — Phase 2
Limb-Girdle Muscular Dystrophy Research Study Groups: Cohort 2, Cohort 1, Cohort 3
Limb-Girdle Muscular Dystrophy Clinical Trial 2023: BBP-418 Highlights & Side Effects. Trial Name: NCT04800874 — Phase 2
BBP-418 (Unknown) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04800874 — Phase 2

Frequently Asked Questions

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

Does this experiment allow senior citizens to participate?

"Applicants accepted into this medical trial must be between 12 and 55 years of age."

Answered by AI

Are there any unfilled spots available in this clinical trial?

"Per the details published on clinicaltrials.gov, this medical experiment is not currently enrolling candidates. This particular investigation was first posted online in February 2021 and most recently updated in December of last year; however, there are two distinct studies that remain open for recruitment at present."

Answered by AI

Am I eligible to participate in this clinical research?

"This clinical trial is recruiting 14 individuals aged 12-55 years, who have lgmd2i (fkrp) and meet the following criteria: Able to complete a 10-meter walk test in ≤12 seconds without assistance ("moderate disease") or are with "severe disease"/non-ambulatory as indicated by an inability to accomplish a 10-meter walk unaided in >12 seconds; Have body weight exceeding 30 kg, Willingness to utilize an appropriate contraceptive method from the time of consent through 12 weeks after they receive their last dose."

Answered by AI

Has the FDA sanctioned BBP-418 for medical use?

"Our experts at Power appraised the safety of BBP-418 on a scale from 1 to 3, and gave it an intermediate score of 2. This is because there are some available data points demonstrating its security but none indicating efficacy."

Answered by AI

Who else is applying?

What site did they apply to?
Virginia Commonwealth University
What portion of applicants met pre-screening criteria?
Met criteria
~6 spots leftby Nov 2026