- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04800874
Study of BBP-418 in Patients With LGMD2I
April 18, 2024 updated by: ML Bio Solutions, Inc.
An Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003)
BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I).
This is an open label study to determine the safety and tolerability of ascending dose levels of BBP-418 in the treatment of ambulatory and non-ambulatory patients with LGMD2I for which no approved therapy currently exists.
Study Overview
Detailed Description
This is an open label study in ambulatory and non-ambulatory subjects with LGMD2I (also known as LGMD R9) previously enrolled in the natural history Study MLB-01-001.
This is a study to determine the safety and tolerability of ascending dose levels of BBP-418 in those subjects.
Study Type
Interventional
Enrollment (Actual)
14
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Virginia
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Richmond, Virginia, United States, 23219
- Virginia Commonwealth University
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
12 years to 55 years (Child, Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Have a body weight >30 kg
- 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
- Willing to use an adequate method of contraception from time of consent through 12 weeks after last dose
- Previous enrolment in the Natural History study MLB-01-001
Exclusion Criteria:
Evidence of clinically significant concomitant disease, including:
- Any history of a gastrointestinal condition, including surgeries, which may affect absorption after oral administration
- Any significant concomitant medical condition, including cardiac, pulmonary, renal, hepatic or endocrine disease other than that associated with LGMD2I
- Any condition other than LGMD2I requiring therapy with prescription medicine (medication for common and mild concomitant conditions may be permitted after consultation with the PI)
- Any other laboratory, vital sign, ECG abnormality, or clinical history or finding that, in the investigator's opinion, is likely to unfavorably alter the risk-benefit of study participation, confound study results, or interfere with study conduct or compliance
- If pregnant and/or breastfeeding or planning to conceive children within the projected duration of the study through 12 weeks after the last dose of study treatment.
- History of drug abuse including alcoholism within 2 years prior to consenting
- Use of ribose or other sugar alcohol-containing supplement within 60 days of Day 1
- Use of a corticosteroid within 60 days of Day 1
- Presence of a platelet disorder, bleeding disorder or other contraindication to muscle biopsy
- Actively on an experimental therapy or device or was on an experimental therapy or device within 60 days prior to Day 1.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Cohort 1
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.
|
BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) for which no approved therapy currently exists.
It targets the molecular defect at the source by supplying excess substrate to the mutant enzyme thus boosting glycosylation of muscle α-dystroglycan.
The BBP-418 drug product will be packaged in sachets and provided in a carton for in-clinic and at home use.
|
Experimental: Cohort 2
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.
|
BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) for which no approved therapy currently exists.
It targets the molecular defect at the source by supplying excess substrate to the mutant enzyme thus boosting glycosylation of muscle α-dystroglycan.
The BBP-418 drug product will be packaged in sachets and provided in a carton for in-clinic and at home use.
|
Experimental: Cohort 3
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.
|
BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) for which no approved therapy currently exists.
It targets the molecular defect at the source by supplying excess substrate to the mutant enzyme thus boosting glycosylation of muscle α-dystroglycan.
The BBP-418 drug product will be packaged in sachets and provided in a carton for in-clinic and at home use.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
Time Frame: 60 months
|
60 months
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Pharmacokinetic profile of BBP-418 by assessment of maximum concentration (Cmax)
Time Frame: 24 months
|
24 months
|
Changes in pharmacodynamic parameters by assessing changes in levels of N-terminal fragment of alpha dystroglycan (α-DG)
Time Frame: 24 months
|
24 months
|
Changes in pharmacodynamic parameters by assessing muscle biopsy of the tibialis anterior
Time Frame: 24 months
|
24 months
|
Pharmacokinetic profile of BBP-418 by assessment of area under the curve (AUC)
Time Frame: 24 months
|
24 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Amy Harper, MD, Virginia Commonwealth University
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
February 18, 2021
Primary Completion (Estimated)
November 1, 2026
Study Completion (Estimated)
November 1, 2026
Study Registration Dates
First Submitted
January 25, 2021
First Submitted That Met QC Criteria
March 14, 2021
First Posted (Actual)
March 16, 2021
Study Record Updates
Last Update Posted (Actual)
April 22, 2024
Last Update Submitted That Met QC Criteria
April 18, 2024
Last Verified
April 1, 2024
More Information
Terms related to this study
Keywords
Other Study ID Numbers
- MLB-01-003
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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