Long-Term Effects of BBP-418 (Ribitol) in Participants with LGMD2I/R9

Status Authorised, recruitment pending · 5 EU/EEA countries · 5 sites · Protocol MLB-01-007

Overview

Sponsor-declared trial summary

Phase Therapeutic confirmatory (Phase III)

Status Authorised, recruitment pending

Participants planned 107

Countries 5

Sites 5

Limb Girdle Muscular Dystrophy (LGMD) Type 2I

To assess the long-term safety of BBP-418 in participants with LGMD2I/R9 To assess the long-term clinical efficacy of BBP-418 in participants with LGMD2I/R9

Key facts

Sponsor: ML Bio Solutions Inc.
Participant type: Patients
Age range: 18-64 years
Gender: Male and Female
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Decision date (initial): 2026-06-16
Transition trial: No
Low-intervention: No
Rare-disease indication: Yes
Vulnerable population: Yes
Funding sources: ML Bio Solutions, Inc – United States

External identifiers

EU CT number: 2025-524371-23-00
ClinicalTrials.gov: NCT11111334

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Efficacy

To assess the long-term safety of BBP-418 in participants with LGMD2I/R9
To assess the long-term clinical efficacy of BBP-418 in participants with LGMD2I/R9

Secondary objectives 1

To assess the long-term clinical efficacy of BBP-418 in participants with LGMD2I/R9 To assess long-term biomarker changes in participants with LGMD2I/R9

Conditions and MedDRA coding

Limb Girdle Muscular Dystrophy (LGMD) Type 2I

Version	Level	Code	Term	System organ class
20.0	PT	10028356	Muscular dystrophy	100000004850

Regulatory references

Scientific advice from competent authorities: European Medicines Agency, Medicines And Healthcare Products Regulatory Agency, Food And Drug Administration
EMA paediatric investigation plan (PIP): EMEA-002887-PIP01-20
Plan to share IPD: No
IPD plan description: N/A

EU CT number	Title	Sponsor
2023-503379-33-01	A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (ribitol) in Patients with Limb Girdle Muscular Dystrophy 2I (LGMD2I)	ML Bio Solutions Inc.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. 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). 2. 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). 3. Is willing and able to complete all study procedures according to the Schedule of Assessments. 4. A WOCBP or a nonsterile male participant must be willing to use an acceptable method of contraception (see Section 5.3) from the time of consent through 30 days after the last dose of study drug in this study.

Exclusion criteria 1

1. Has developed clinically significant concomitant disease that would, in the Investigator’s opinion, be likely to unfavorably impact study participation, including: a. Any significant concomitant medical condition, including psychiatric, cardiac, renal, pulmonary, hepatic, or endocrine disease other than that associated with LGMD2I/R9. b. Any other significant laboratory, vital sign, ECG abnormality, clinical history, or finding. 2. 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. 3. 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

-Frequency and severity of TEAEs and treatment-emergent SAEs - Change from baseline in NSAD at EOT

Secondary endpoints 1

• Change from baseline in the following endpoints at EOT: o 10MWT (m/s) o FVC (percent predicted, performed in a sitting position) o PUL 2.0 (for participants who are non-ambulatory at OLE baseline) o 100MTT • Change from baseline in serum CK at EOT

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Ribitol

PRD10352711 · Product

Active substance: Ribitol
Pharmaceutical form: GRANULES FOR ORAL SOLUTION
Route of administration: ORAL USE
Max daily dose: 24 g gram(s)
Max total dose: 26280 g gram(s)
Max treatment duration: 36 Month(s)
Authorisation status: Not Authorised
MA holder: ML BIO SOLUTIONS INC.
Paediatric formulation: No
Orphan designation: Yes
Orphan designation number: EU/3/20/2337

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

ML Bio Solutions Inc.

Sponsor organisation: ML Bio Solutions Inc.
Address: 1800 Owens Street Suite C1200
City: San Francisco
Postcode: 94158-2584
Country: United States

Scientific contact point

Organisation: ML Bio Solutions Inc.
Contact name: Chief Medical Officer

Public contact point

Organisation: ML Bio Solutions Inc.
Contact name: Chief Medical Officer

Third parties 2

Organisation	City, country	Duties
DLRC Limited ORG-100004400	Letchworth Garden City, United Kingdom	Code 12
Fortrea Inc. ORG-100012602	Durham, United States	On site monitoring, Code 10, Code 11, Code 12, Code 13, Code 14, Other, Code 2, Interactive response technologies (IRT), Laboratory analysis, Code 5, Data management, E-data capture, Code 8, Code 9

Locations

5 EU/EEA countries · 5 investigational sites