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BBP-418 for LGMD2I

Not currently recruiting at 19 trial locations
Age: Any Age
Sex: Any
Trial Phase: Phase 3
Sponsor: ML Bio Solutions, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This is an open-label extension (rollover) study designed to evaluate the long-term safety and efficacy of BBP-418 (ribitol) in participants with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) who have previously participated in Study MLB-01-005 (Fortify).

Participants will receive BBP-418 administered orally at protocol-defined doses and schedules. The study will assess long-term safety through monitoring of adverse events, clinical laboratory evaluations, and other safety assessments. Efficacy will be evaluated using functional measures and other clinical endpoints relevant to LGMD2I/R9.

Participants will be followed for up to 36 months, with a final safety follow-up assessment conducted approximately 30 days after the last dose of study drug.

Are You a Good Fit for This Trial?

This trial is for people with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) who have already taken part in a previous study of BBP-418. Only those who completed the earlier study can join this extension.

Inclusion Criteria

Completed Study MLB-01-005 on study drug through the final clinic visit (Month 36 or another qualifying end-of-study visit as determined by the Sponsor)
Is willing and able to complete all study procedures according to the Schedule of Assessments
I understand the study and agree to participate by signing the consent form.
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Exclusion Criteria

Has developed clinically significant concomitant disease likely to unfavorably impact study participation, including any significant concomitant medical condition (psychiatric, cardiac, renal, pulmonary, hepatic, or endocrine disease other than that associated with LGMD2I/R9)
Any other significant laboratory, vital sign, ECG abnormality, clinical history, or finding
Is pregnant (based on Baseline / Day 1 pregnancy test) and/or breastfeeding or planning to conceive children within the study duration through 30 days after last dose of study drug
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What Are the Treatments Tested in This Trial?

Interventions

  • BBP-418 (ribitol)

Trial Overview

The study tests long-term use of BBP-418 (ribitol), taken by mouth, to see if it remains safe and effective over up to three years for LGMD2I/R9 patients. All participants receive the drug; there is no placebo group.

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: BBP-418Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

ML Bio Solutions, Inc.

Lead Sponsor

Trials
3
Recruited
200+

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