A 6-month multicenter, randomised, double-blind, placebo-controlled, Phase IIa proof of principle study of naproxcinod (HCT 3012) 750 mg bid in patients with Becker Muscular Dystrophy.
NicOx SA, France Non-confidential report
NicOx SA submitted a proposal to use naproxcinod (HCT3012) in Becker muscular dystrophy (BMD) subjects in a phase IIa clinical trial. Naproxcinod is an anti-inflammatory proprietary compound synthesized by adding a nitric oxide (NO-donating moiety) to naproxen. It has been used extensively and safely in clinical trials for osteoarthritis (OA) (more than 4000 subjects in studies up to 65 weeks without significant adverse effects). At the time of the submission of the protocol to TACT, HCT 3012 pre-clinical testing was relatively limited in dystrophin-deficient mice (e.g., mdx) and the TACT panel recommended that more data be available before going to clinical trial. The proposed, 6-month, clinical trial in BMD using HCT 3012 was placebo-controlled with diagnosis based on muscle biopsy and DNA analysis. The clinical protocol in BMD was of interest but the review panel suggested that the Duchenne muscular dystrophy (DMD) patient population potentially offered advantages for clinical testing. Two attractive features of the DMD population include a greater number of subjects available for recruitment and better defined outcome measures for clinical trials.
Name of applicant: Fabrizio Dolfi, MD Reviewed: October, 2011 in Lisbon, Portugal
NicOx SA submitted a proposal to use naproxcinod (HCT3012) in Becker muscular dystrophy (BMD) subjects in a phase IIa clinical trial. Naproxcinod is an anti-inflammatory proprietary compound synthesized by adding a nitric oxide (NO-donating moiety) to naproxen. It has been used extensively and safely in clinical trials for osteoarthritis (OA) (more than 4000 subjects in studies up to 65 weeks without significant adverse effects). At the time of the submission of the protocol to TACT, HCT 3012 pre-clinical testing was relatively limited in dystrophin-deficient mice (e.g., mdx) and the TACT panel recommended that more data be available before going to clinical trial. The proposed, 6-month, clinical trial in BMD using HCT 3012 was placebo-controlled with diagnosis based on muscle biopsy and DNA analysis. The clinical protocol in BMD was of interest but the review panel suggested that the Duchenne muscular dystrophy (DMD) patient population potentially offered advantages for clinical testing. Two attractive features of the DMD population include a greater number of subjects available for recruitment and better defined outcome measures for clinical trials.
