Trial Profile
A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability and Pharmacokinetics Study of AVI-4658 (Eteplirsen), a Phosphorodiamidate Morpholino Oligomer, Administered Over 28 Weeks in the Treatment of Ambulant Subjects With Duchenne Muscular Dystrophy
Status:
Completed
Phase of Trial:
Phase II
Latest Information Update: 06 Nov 2021
Price :
$35
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At a glance
- Drugs Eteplirsen (Primary)
- Indications Duchenne muscular dystrophy
- Focus Therapeutic Use
- Sponsors Sarepta Therapeutics
- 20 May 2020 Results assessing psychometric properties to determine their suitability for assessing patient-perceived treatment benefit in DMD, presented at the 25th Annual International Meeting of the International Society for Pharmacoeconomics and Outcomes Research
- 27 Dec 2017 Results from Study 201/202 presented in a Sarepta Therapeutics media release.
- 27 Dec 2017 According to a Sarepta Therapeutics media release, pulmonary function results from eteplirsen-treated Duchenne muscular dystrophy (DMD) patients (N=12) in Study 201/202 compared to natural history were published in The Journal of Neuromuscular Diseases.