N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
Summary
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC). There are two phases to this study: the Parent Study, and the Extension Phase. The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the symptomatic treatment of NPC. The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of NPC.
Timeline
- Start
- 2019-09-04
- Primary completion
- 2022-11-07
- Completion
- 2022-11-07
Outcome
Met primary endpoint
paper NALL met the CI-CS primary endpoint (mean difference 0.86, SD = 2.52, 90% CI 0.25, 1.75, p = 0.029), as well as secondary endpoints. PMID 34387740 ↗
registry analysis (superiority test); Parent Study; p = 0.029; Hodges-Lehmann Estimator 1.00 (90% CI 0.25 to 1.75); 1-sided Wilcoxon signed-rank test NCT03759639 ↗
release “(IB1001-201), which met both its primary and secondary endpoints” intrabio.com ↗
release “IB1001-201 – NCT03759639), which also showed a statistically significant and clinically meaningful improvement” intrabio.com ↗
Publications
- Bremova-Ertl T, Claassen J, Foltan T, Gascon-Bayarri J, Gissen P, Hahn A, Hassan A, Hennig A, Jones SA, Kolnikova M, Martakis K, Raethjen J, Ramaswami U, Sharma R, Schneider SA. Efficacy and safety of N-acetyl-L-leucine in Niemann-Pick disease type C. J Neurol. 2022 Mar;269(3):1651-1662. doi: 10.1007/s00415-021-10717-0. Epub 2021 Aug 13.
- Churchill GC, Strupp M, Factor C, Bremova-Ertl T, Factor M, Patterson MC, Platt FM, Galione A. Acetylation turns leucine into a drug by membrane transporter switching. Sci Rep. 2021 Aug 4;11(1):15812. doi: 10.1038/s41598-021-95255-5.
- Fields T, Patterson M, Bremova-Ertl T, Belcher G, Billington I, Churchill GC, Davis W, Evans W, Flint S, Galione A, Granzer U, Greenfield J, Karl R, Kay R, Lewi D, Mathieson T, Meyer T, Pangonis D, Platt FM, Tsang L, Verburg C, Factor M, Strupp M. A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia. Trials. 2021 Jan 22;22(1):84. doi: 10.1186/s13063-020-05009-3.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | trenonacog alfa | Protein / enzyme biologic | 2 g | Oral |
| Subject | trenonacog alfa | Protein / enzyme biologic | 3 g | Oral |
| Subject | trenonacog alfa | Protein / enzyme biologic | 4 g | Oral |