BioinvestGPT

BioinvestGPT ApS · Research use only

NCT05775848 (BBP-418 in Limb-Girdle Muscular Dystrophy Type 2I): BVCT ex-ante prediction vs. reported outcome

On 26 Jan 2025, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 27 Oct 2025 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically significant yet weak clinical benefit and commercially probably insufficient.

Clinical Trial

NCTID
NCT05775848
Acronym
Fortify
Drug Name
BBP-418 (Ribitol)
Drug MoA
prodrug ribitol
Drug Modality
small molecule
Drug Class
First-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
Human Patients
81
Sponsor
BridgeBio Pharma
Phase
Phase 3

Prediction of , index 1890

Prospective Prediction
Prediction Index
1890
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in NSAD/10MWR non-superior to FKRP gene therapy)
Technical Prediction
FAILURE (statistically maybe significant yet very weak clinical benefit in NSAD/10MWR compared with placebo)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
274 days in advance
Readout Data Interpretation
BBP418 achieved 0.27m/s placebo-adjusted improvement in 100MTT (p<0.0001);which is below the MCID threshold (>=0.55m/s placebo-adjusted improvement in 100MTT; calculated using the 112.2(127.9) baseline 100MTT of patients in the FORTIFY trial (see add'l link below) and the 1/3 SD MCID definition (doi.org/10.1212/WNL.92.15_supplement.S33.003)
Press Release
Additional Readout Data
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant yet weak clinical benefit and commercially probably insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

ClinicalTrials.gov record, retrieved 2026-09-24. Third-party data.

Official title
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)
Phase
Phase 3
Status
Active not recruiting
Enrollment
112
Lead sponsor
ML Bio Solutions, Inc.
Conditions
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
Interventions
DRUG: BBP-418 (ribitol); OTHER: Placebo
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2023-05-31
Primary completion
2027-09
Completion
2027-09
Primary outcomes
  • Change from baseline in North Star Assessment for Limb Girdle Muscular Dystrophy following 36 months of treatment to assess efficacy of BBP-418 or placebo (36 months)
  • Frequency and severity of treatment-emergent adverse events following 36 months of treatment to assess safety of BBP-418 or placebo (36 months)

NCT05775848 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

How to read these records: evidence · methodology · all trials