drugset / Trial / NCT02760277
An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Phase 2
Completed
48 enrolled
ReveraGen BioPharma, Inc.
Cooperative International Neuromuscular Research Group · collabNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) · collabNational Institute of Neurological Disorders and Stroke (NINDS) · collabUniversity of Pittsburgh · collab
Non-randomizedSequentialOpen-labelTreatment
Summary
The main purposes of this study are to see if it is safe to use a new medication called vamorolone for more than two weeks in children with Duchenne muscular dystrophy (DMD), to see if vamorolone works for the treatment for DMD, and to see how any potential side effects compare to those seen in boys using steroids.
Timeline
- Start
- 2016-07-28
- Primary completion
- 2018-04-26
- Completion
- 2018-04-26
Publications
- Hoffman E, Gaglianone S, Ketema R, Tu W, Peay H, Clemens P, Dang U, Conklin L. Return of participant-level clinical trial results to participants: pilot of a simplified centralised approach. BMJ Open. 2024 Mar 23;14(3):e080097. doi: 10.1136/bmjopen-2023-080097.
- Smith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, Kerchner L, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, Gordish-Dressman H, Hagerty L, Dang UJ, Damsker JM, Schwartz BD, Mengle-Gaw LJ, McDonald CM; CINRG VBP15 and DNHS Investigators. Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. PLoS Med. 2020 Sep 21;17(9):e1003222. doi: 10.1371/journal.pmed.1003222. eCollection 2020 Sep.
- Li X, Conklin LS, van den Anker J, Hoffman EP, Clemens PR, Jusko WJ. Exposure-Response Analysis of Vamorolone (VBP15) in Boys With Duchenne Muscular Dystrophy. J Clin Pharmacol. 2020 Oct;60(10):1385-1396. doi: 10.1002/jcph.1632. Epub 2020 May 20.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Vamorolone | Small molecule | 0.25 mg/kg | Oral |
| Subject | Vamorolone | Small molecule | 0.75 mg/kg | Oral |
| Subject | Vamorolone | Small molecule | 2 mg/kg | Oral |
| Subject | Vamorolone | Small molecule | 6 mg/kg | Oral |