LGMD2IPhase 2NCT04800874
Study of BBP-418 in Patients With LGMD2I
A Phase 2 study testing BBP-418 in people with LGMD2I, run by ML Bio Solutions, Inc. at 1 site in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 12 to 55 years
- Conditions the study is for
- LGMD2I
- What is being tested?
- BBP-418
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Feb 2021to 1 Nov 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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.
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- 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 locations and contacts
1 site in 1 country.
United States1 site
Virginia Commonwealth University
Richmond, Virginia, 23219
About this study
Official title: An Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003)
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.
Read the detailed 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.
- Conditions
- LGMD2I
- Being tested
- BBP-418
- Keywords
- LGMD, LGMD R9, Limb Girdle Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophy, Limb-Girdle, Type 2I, Muscular Dystrophies, Limb-Girdle
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BBP-418Drug
BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) for which no approved therapy currently exists. It targets the molecular defect at the source by supplying excess substrate to the mutant enzyme thus boosting glycosylation of muscle α-dystroglycan. The BBP-418 drug product will be packaged in sachets and provided in a carton for in-clinic and at home use.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 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. Receives: Drug: BBP-418 |
| Cohort 2 | Experimental | 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. Receives: Drug: BBP-418 |
| Cohort 3 | Experimental | 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. Receives: Drug: BBP-418 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
Measured: 60 months
Secondary outcomes (4)
Pharmacokinetic profile of BBP-418 by assessment of maximum concentration (Cmax)
Measured: 24 months
Changes in pharmacodynamic parameters by assessing changes in levels of N-terminal fragment of alpha dystroglycan (α-DG)
Measured: 24 months
Changes in pharmacodynamic parameters by assessing muscle biopsy of the tibialis anterior
Measured: 24 months
Pharmacokinetic profile of BBP-418 by assessment of area under the curve (AUC)
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2021
- Study start18 Feb 2021
- Primary completion1 Nov 2026 (estimated)
- Study completion1 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LGMD2IStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Subjects must have been enrolled in the MLB-01-001 study to participate in this trial. Three ascending dose cohorts of 4-6 subjects each are planned.
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- ML Bio Solutions, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Amy Harper, MD, Principal investigator, Virginia Commonwealth University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04800874
- Sponsor's protocol ID
- MLB-01-003
- Status last verified
- Jul 2026
- First submitted
- 25 Jan 2021
- Last updated
- 21 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003)
- ClinicalTrials.gov ID
- NCT04800874
- Acronym / sponsor ID
- MLB-01-003
- Conditions
- LGMD2I
- Keywords
- LGMD, LGMD R9, Limb Girdle Muscular Dystrophy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 14 participants (actual)
- Lead sponsor
- ML Bio Solutions, Inc.Industry (pharma, biotech, devices)
- Study start
- 18 Feb 2021 (actual)
- Primary completion
- 1 Nov 2026 (estimated)
- Study completion
- 1 Nov 2026 (estimated)
- First posted
- 16 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Subjects must have been enrolled in the MLB-01-001 study to participate in this trial. Three ascending dose cohorts of 4-6 subjects each are planned.
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: BBP-418Subjects 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. |
| Cohort 2 | Experimental | Drug: BBP-418Subjects 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. |
| Cohort 3 | Experimental | Drug: BBP-418Subjects 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. |
Who may qualify
- Ages
- 12 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (11 listed)
- Evidence of clinically significant concomitant disease, including:
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BBP-418BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) for which no approved therapy currently exists. It targets the molecular defect at the source by supplying excess substrate to the mutant enzyme thus boosting glycosylation of muscle α-dystroglycan. The BBP-418 drug product will be packaged in sachets and provided in a carton for in-clinic and at home use. | Drug | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
Time frame: 60 months
4 secondary outcomes
Pharmacokinetic profile of BBP-418 by assessment of maximum concentration (Cmax)
Time frame: 24 months
Changes in pharmacodynamic parameters by assessing changes in levels of N-terminal fragment of alpha dystroglycan (α-DG)
Time frame: 24 months
Changes in pharmacodynamic parameters by assessing muscle biopsy of the tibialis anterior
Time frame: 24 months
Pharmacokinetic profile of BBP-418 by assessment of area under the curve (AUC)
Time frame: 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jul 2026.
- Individual participant dataSharing planned: No
Related trials for LGMD2I
Active, not recruitingPhase 3
Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
Active, not recruitingPhase 1/2
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2
Active, not recruitingPhase 3
Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
RecruitingObservational study
Long-Term Development of Muscular Dystrophy Outcome Assessments
LGMD1B, LGMD1C
Active, not recruitingPhase 1
ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
LGMDR9
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Amy Harper, MD principal investigator
Virginia Commonwealth University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BBP-418 in people with LGMD2I, run by ML Bio Solutions, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 21 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation (time frame: 60 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jul 2026.