BBP-418 for Limb-Girdle Muscular Dystrophy

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: ML Bio Solutions, Inc.

Must not be taking: Corticosteroids, Ribose, Sugar alcohols, others

Disqualifiers: Cardiac, Pulmonary, Renal, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 90 days

14 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but it mentions that medication for common and mild conditions may be allowed after consulting with the principal investigator. You cannot use ribose, sugar alcohol supplements, or corticosteroids within 60 days before starting the trial.

How is the drug BBP-418 different from other treatments for limb-girdle muscular dystrophy?

There is no standard treatment currently known for limb-girdle muscular dystrophy, making BBP-418 potentially unique as a new option for this condition. ^{1 2 3 4 5}

Research Team

Amy Harper, MD

Principal Investigator

Virginia Commonwealth University

Eligibility Criteria

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.

Inclusion Criteria

Previous enrolment in the Natural History study MLB-01-001

Willing to use an adequate method of contraception from time of consent through 12 weeks after last dose

I have a genetic diagnosis of LGMD2I and show signs of muscle weakness.

Exclusion Criteria

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

I have a serious health condition alongside my cancer.

I have not been on any experimental treatments or devices for the last 60 days.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive BBP-418 with ascending dose levels to determine safety and tolerability

90 days

Dose Escalation

Participants continue receiving BBP-418 at 12 grams BID until study completion

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• BBP-418

Trial Overview 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.

Participant Groups

Group I: Cohort 3 Experimental Treatment 1 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 2 Experimental Treatment 1 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 1 Experimental Treatment 1 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.

Who Is Running the Clinical Trial?

ML Bio Solutions, Inc.

Lead Sponsor

Trials

3

Recruited

200+

References

1. United States pubmed.ncbi.nlm.nih.gov

LGMDD1 natural history and phenotypic spectrum: Implications for clinical trials. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Painful enlargement of the calf muscles in limb girdle muscular dystrophy type 2B (LGMD2B) with a novel compound heterozygous mutation in DYSF. [2017]

3. England pubmed.ncbi.nlm.nih.gov

TRIM32 biallelic defects cause limb-girdle muscular dystrophy R8: identification of two novel mutations and investigation of genotype-phenotype correlation. [2023]

4. Belgium pubmed.ncbi.nlm.nih.gov

[Progressive muscular weakness of lower limbs revealing a limb girdle muscular dystrophy]. [2018]

5. United States pubmed.ncbi.nlm.nih.gov

Lariat branch point mutation in the dysferlin gene with mild limb-girdle muscular dystrophy. [2022]