BBP-418 in Patients With LGMD2I

BBP-418 in Patients With LGMD2I
Recruitment Status: 
Status Last Updated: 
March 16, 2021
Study Purpose: 

This is an open label study in ambulatory and non-ambulatory subjects with LGMD2I (also known as LGMD R9). This is a study to determine the safety and tolerability of ascending dose levels of BBP-418 in those subjects.

Drug: BBP-418
Study Description: 

Subjects must have been enrolled in the MLB-01-001 study to participate in this trial. Three ascending dose cohorts of 4 subjects each are planned, but a fourth cohort may be added.

Study Type: 
Official Title: 
An Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003)
Study Start Date: 
February 18, 2021
Study Completion Date: 
August 5, 2021
Primary Objective(s): 
  1. Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation [ Time Frame: 90 days ]
Secondary Objective(s): 
  1. Pharmacokinetic profile of BBP-418 by assessment of maximum concentration (Cmax) [ Time Frame: Day 1 and Day 90 ]
  2. Changes in pharmacodynamic parameters by assessing changes in levels of N-terminal fragment of alpha dystroglycan (α-DG) [ Time Frame: Day 1 and Day 90 ]
  3. Changes in pharmacodynamic parameters by assessing muscle biopsy of the tibialis anterior [ Time Frame: Day 1 and Day 90 ]
  4. Pharmacokinetic profile of BBP-418 by assessment of area under the curve (AUC) [ Time Frame: Day 1 and Day 90 ]

Ages Eligible for Study:      12 Years to 55 Years   (Child, Adult)

Sexes Eligible for Study:      All

Accepts Healthy Volunteers:      No

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 MLB-01-001 study
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 and the Medical Monitor)
  • 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, planning to become 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 Site(s)/Location(s): 

United States, Virginia

Virginia Commonwealth University    

Richmond, Virginia, United States, 23219

Sponsors & Collaborators: 

ML Bio Solutions, Inc.

Principal Investigator(s): 

Principal Investigator:

Nicholas Johnson, MD

Virginia Commonwealth University

For more information, please contact the Study Coordinator: 



Phone: ID: