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Abstracts & Posters

Safety and Tolerability of Ataluren in a Phase 3 Study of Patients with Nonsense Mutation Duchenne Muscular Dystrophy

key information

source: American Academy of Neurology

year: 2015

authors: Craig Campbell, Perry Shieh, Thomas Sejersen, Xiaohui Luo, Gary Elfring, Hans Kroger, Peter Riebling, Tuyen Ong, Robert Spiegel, Stuart Peltz, Brenda Wong


Examine the safety/tolerability of ataluren in the Ataluren Confirmatory Trial in Duchenne Muscular Dystrophy (ACT DMD), a randomized, double-blind, placebo-controlled Phase 3 study of patients with nonsense mutation DMD (nmDMD).

A randomized, double-blind, placebo-controlled Phase 2b study of ataluren in patients with nmDMD reported that ataluren was generally well tolerated.

In this Phase 3, ACT DMD, multicenter study, males aged 7-16 years with nmDMD, baseline six-minute walk distance (6MWD) >=150m and <=80[percnt] of predicted, and steroid use >=6 months were randomized 1:1 to ataluren 10, 10, 20 mg/kg or placebo orally 3 times daily for 48 weeks.

230 patients were randomized (ataluren, n=115; placebo, n=115). Demographics were well balanced across treatment arms. Overall, 96.1[percnt] of patients completed the 48-week trial, and 97[percnt] of those chose to continue in the extension study. 103 (89.6[percnt]) patients on ataluren and 100 (87.0[percnt]) patients on placebo experienced treatment-emergent adverse events (TEAEs). The most common TEAEs in the ataluren and placebo arms, respectively, were vomiting (22.6[percnt] and 18.3[percnt]), nasopharyngitis (20.9[percnt] and 19.1[percnt]), fall (19.1[percnt] and 17.4[percnt]), headache (18.3[percnt] for both), and cough (16.5[percnt] and 11.3[percnt]). Four patients in each arm had at least 1 serious adverse event (SAEs). In the ataluren arm, these SAEs were pneumonia and bronchiolitis in 1 patient, pneumonia and post-traumatic pain in 1 patient, tendon disorder in 1 patient, and adenoidal and nasal turbinate hypertrophy in 1 patient; none were considered ataluren-related by the investigator. Only 1 patient in each arm discontinued treatment due to TEAEs: 1 patient discontinued ataluren due to Grade 2 constipation considered possibly related to treatment, and 1 patient discontinued placebo due to loss of ambulation.

Ataluren was generally well-tolerated by patients with nmDMD, and the spectrum and severity of adverse events was consistent with previous studies.

organization: Cincinnati Children'S Hospital Medical Center, USA; Karolinska Institutet, Sweden; London Health Sciences Centre , Western University, Canada; PTC Therapeutics, USA; University of California Los Angeles, USA

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