Your session is about to expire
← Back to Search
Unknown
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)
Have a body weight >30 kg
Must not have
Any significant concomitant medical condition, including cardiac, pulmonary, renal, hepatic or endocrine disease other than that associated with LGMD2I
Presence of a platelet disorder, bleeding disorder or other contraindication to muscle biopsy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 60 months
Awards & highlights
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 belowSelect...
I have a genetic diagnosis of LGMD2I and show signs of muscle weakness.
Select...
I weigh more than 30 kilograms.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I do not have major health issues unrelated to LGMD2I.
Select...
I have a condition that prevents safe muscle biopsy.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 60 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~60 months
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 moreTrial 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
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have a serious health condition alongside my cancer.I have a genetic diagnosis of LGMD2I and show signs of muscle weakness.I have not been on any experimental treatments or devices for the last 60 days.I am not on prescription medication for conditions other than LGMD2I, except for mild issues approved by the study leader.I have a gut condition or surgery that could affect how I absorb pills.I can walk 10 meters in 12 seconds or less, or I cannot walk this distance unaided.I do not have major health issues unrelated to LGMD2I.I weigh more than 30 kilograms.You haven't taken any supplements containing ribose or other sugar alcohols within 60 days before the start of the trial.I have not used corticosteroids in the last 60 days.I have a condition that prevents safe muscle biopsy.You have a history of drug abuse, including alcohol addiction, within the past 2 years before agreeing to participate.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 2
- Group 2: Cohort 1
- Group 3: Cohort 3
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.
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger