Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
Conditions
Keywords
LGMD2I/R9
Brief summary
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.
Interventions
Drug: BBP-418. Single arm. Participants who completed Study MLB-01-005 and meet eligibility criteria will receive BBP-418 (ribitol) taken orally twice daily at 9 or 12g (based on body weight measured) for up to 36 months.
Sponsors
ML Bio Solutions, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
This is an open-label extension study in which participants who completed Study MLB-01-005 will receive BBP-418 administered orally twice daily for up to 36 months.
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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). * The participant (or parent/guardian) who signs the ICF understands the study procedures and agrees to participate in the study by giving informed consent (or assent, if \<18 years of age). * Is willing and able to complete all study procedures according to the Schedule of Assessments. * A WOCBP or a nonsterile male participant must be willing to use an acceptable method of contraception from the time of consent through 30 days after the last dose of study drug in this study.
Exclusion criteria
* Has developed clinically significant concomitant disease that would, in the Investigator's opinion, be likely to unfavorably impact study participation, including: * Any significant concomitant medical condition, including 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 the Baseline / Day 1 pregnancy test result) and/or breastfeeding or planning to conceive children within the projected duration of the study through 30 days after the last dose of study drug in this study. * Has active suicidal ideation, defined as having a suicide ideation score of 4 (Active Suicidal Ideation with Some Intent to Act, without Specific Plan) or 5 (Active Suicidal Ideation with Specific Plan and Intent) on the C-SSRS at Baseline / Day 1.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Frequency and severity of treatment-emergent adverse events to assess long-term safety of BBP-418. | 36 months |
| Change from baseline in North Star Assessment for LGMD2I/R9 to assess long-term efficacy of BBP-418. | 36 months |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in 10MWT (10-meter walk test) (m/s) for LGMD2I/R9 to assess long-term efficacy of BBP-418. | 36 months |
| Change from baseline FVC (forced vital capacity) (percent predicted, performed in a sitting position) for LGMD2I/R9 to assess long-term efficacy of BBP-418. | 36 months |
| Change from baseline in PUL 2.0 (performance of the upper limb) total score for LGMD2I/R9 to assess long-term efficacy of BBP-418. | 36 months |
| Change from baseline in 100MTT (100-meter timed test) (m/s) for LGMD2I/R9 to assess long-term efficacy of BBP-418. | 36 months |
| Change from baseline in Serum CK (creatine kinase) for LGMD2I/R9 to assess long-term biomarker changes in participants. | 36 months |
Countries
Australia, Denmark, Germany, Italy, Netherlands, Norway, United Kingdom, United States
Outcome results
None listed