Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function
- Hoffman, EP
- Schwartz, BD
- Mengle-Gaw, LJ
- Smith, EC
- Castro, D
- Mah, JK
- McDonald, CM
- Kuntz, NL
- Finkel, RS
- Guglieri, M
- Bushby, K
- Tulinius, M
- Nevo, Y
- Ryan, MM
- Webster, R
- Smith, AL
- Morgenroth, LP
- Arrieta, A
- Shimony, M
- Siener, C
- Jaros, M
- Shale, P
- McCall, JM
- Nagaraju, K
- van den Anker, J
- Conklin, LS
- Cnaan, A
- Gordish-Dressman, H
- Damsker, JM
- Clemens, PR
DOIAbstract
OBJECTIVE: To study vamorolone, a first-in-class steroidal anti-inflammatory drug, in Duchenne muscular dystrophy (DMD). METHODS: An open-label, multiple-ascending-dose study of vamorolone was conducted in 48 boys with DMD (age 4-<7 years, steroid-naive). Dose levels were 0.25, 0.75, 2.0, and 6.0 mg/kg/d in an oral suspension formulation (12 boys per dose level; one-third to 10 times the glucocorticoid dose in DMD). The primary goal was to define optimal doses of vamorolone. The primary outcome for clinical efficacy was time to stand from supine velocity. RESULTS: Oral administration of vamorolone at all doses tested was safe and well tolerated over the 24-week treatment period. The 2.0-mg/kg/d dose group met the primary efficacy outcome of...
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