Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)Phase 3NCT07678775
Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9
A Phase 3 study testing BBP-418 (ribitol) in people with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I), run by ML Bio Solutions, Inc. at 20 sites in 8 countries.
Trial 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). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
- What is being tested?
- BBP-418 (ribitol)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 107 participants
- Where
- 20 sitesin 8 countries
- Study dates
- 5 Jun 2026to Oct 2030
- Phase
- Phase 3
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
20 sites in 8 countries.
20 sites in 8 countries
United States12 sites
University of Colorado Hospital (UCH) - Anschutz Medical Campus
Aurora, Colorado, 80045
Kennedy Krieger Institute
Baltimore, Maryland, 21205
University of Florida Health Center for Pediatric Neuromuscular and Rare Diseases
Gainesville, Florida, 32608
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, 66160
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
University of Minnesota Medical School
Minneapolis, Minnesota, 55455
Children's Hospital of the Kings Daughters
Norfolk, Virginia, 23510
University of California Irvine Medical Center
Orange, California, 92868
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Washington University School of Medicine
St Louis, Missouri, 63110
United Kingdom2 sites
UCL Great Ormond Street Hospital
London
Newcastle University - John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne
Denmark1 site
Rigshospitalet, Neuromuscular Clinic and Research Unit
Copenhagen
Germany1 site
Charité Universitätsmedizin Berlin and Max Delbrück Center
Berlin
About this study
Official title: An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of BBP-418 (Ribitol) in Participants With Limb-Girdle Muscular Dystrophy 2I/R9 (LGMD2I/R9)
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.
- Being tested
- BBP-418 (ribitol)
- Keywords
- LGMD2I/R9
- Medical subject headings
- Muscular Dystrophy, Limb-Girdle, Type 2I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BBP-418 (ribitol)Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| BBP-418 | Experimental | Drug: BBP-418. Single arm. Receives: Drug: BBP-418 (ribitol) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency and severity of treatment-emergent adverse events to assess long-term safety of BBP-418.
Measured: 36 months
Change from baseline in North Star Assessment for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Measured: 36 months
Secondary outcomes (5)
Change from baseline in 10MWT (10-meter walk test) (m/s) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Measured: 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.
Measured: 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.
Measured: 36 months
Change from baseline in 100MTT (100-meter timed test) (m/s) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Measured: 36 months
Change from baseline in Serum CK (creatine kinase) for LGMD2I/R9 to assess long-term biomarker changes in participants.
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2026
- Study start5 Jun 2026
- Primary completionOct 2030 (estimated)
- Study completionOct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)Studies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- 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.
- Masking
- None (open label)
- Enrolment
- 107 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ML Bio Solutions, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07678775
- Sponsor's protocol ID
- MLB-01-007
- Status last verified
- Jun 2026
- First submitted
- 25 Jun 2026
- Last updated
- 2 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 2 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of BBP-418 (Ribitol) in Participants With Limb-Girdle Muscular Dystrophy 2I/R9 (LGMD2I/R9)
- ClinicalTrials.gov ID
- NCT07678775
- Acronym / sponsor ID
- MLB-01-007
- Keywords
- LGMD2I/R9
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 107 participants (estimated)
- Lead sponsor
- ML Bio Solutions, Inc.Industry (pharma, biotech, devices)
- Study start
- 5 Jun 2026 (actual)
- Primary completion
- Oct 2030 (estimated)
- Study completion
- Oct 2030 (estimated)
- First posted
- 1 Jul 2026 (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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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.
| Arm | Type | Receives |
|---|---|---|
| BBP-418 | Experimental | Drug: BBP-418 (ribitol)Drug: BBP-418. Single arm. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (5 listed)
- Has developed clinically significant concomitant disease that would, in the Investigator's opinion, be likely to unfavorably impact study participation, including:
- 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.
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-418 (ribitol)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. | Drug | BBP-418 |
Primary outcome
Frequency and severity of treatment-emergent adverse events to assess long-term safety of BBP-418.
Time frame: 36 months
Change from baseline in North Star Assessment for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time frame: 36 months
5 secondary outcomes
Change from baseline in 10MWT (10-meter walk test) (m/s) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time frame: 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.
Time frame: 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.
Time frame: 36 months
Change from baseline in 100MTT (100-meter timed test) (m/s) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time frame: 36 months
Change from baseline in Serum CK (creatine kinase) for LGMD2I/R9 to assess long-term biomarker changes in participants.
Time frame: 36 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 2 Jul 2026.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
20 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing BBP-418 (ribitol) in people with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I), run by ML Bio Solutions, Inc. at 20 sites in 8 countries.
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 4 inclusion and 5 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 2 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 20 sites in United States, United Kingdom, Australia, Denmark, Germany and 3 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Frequency and severity of treatment-emergent adverse events to assess long-term safety of BBP-418. (time frame: 36 months). The study lists 2 primary outcomes in all. 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 2 Jul 2026.