Study of BBP-418 in Patients With LGMD2I

ML Bio Solutions

Status and phase: Active, not recruiting; Phase 2

Conditions: LGMD2I

Treatments: Drug: BBP-418

Study type: Interventional

Funder types: Industry

Identifiers

NCT04800874

MLB-01-003

Details and patient eligibility

About

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.

Full 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.

Enrollment: 14 patients

Sex: All

Ages: 12 to 55 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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14 participants in 3 patient groups

Cohort 1

Experimental group

Description:

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.

Treatment:

Drug: BBP-418

Cohort 2

Experimental group

Description:

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.

Treatment:

Drug: BBP-418

Cohort 3

Experimental group

Description:

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.

Treatment:

Drug: BBP-418