Assessing the ability of boys with Duchenne muscular dystrophy age 4-7 years to swallow softgel capsules: Clinical trial experience with edasalonexent

What is known and objective: There is limited information on acceptability of solid dosage forms by young patients with neuromuscular disorders such as Duchenne muscular dystrophy (DMD). Capsule size selection and ability to swallow the NF-kappa B inhibitor edasalonexent were assessed in males 4-7 years of age with DMD enrolled in clinical trials for a new therapeutic. Methods: The Phase 3 PolarisDMD randomized, double-blind, placebo-controlled trial enrolled 131 patients from 8 countries. The Phase 2 MoveDMD trial enrolled 31 patients in the United States. As part of enrolment criteria, these trials assessed the ability to swallow softgel 100 mg (similar to 10 mm) or 250 mg (similar to 15 mm) capsules formulated with a phosphatidylcholine-containing coating. Supportive strategies included pill-swallowing techniques and aids. Results: Most (97%; 175/181) patients screened were able to swallow capsules. In Phase 2 and 3, respectively, 77% (24/31) and 61% (80/131) of enrolled patients selected the larger capsule and among those selecting the smaller capsule, most transitioned to the larger capsule. There were no obvious geographical differences in ability to swallow capsules and size selection was not correlated with age. Compliance was high (92%-98%) through 52 weeks of dosing with no discontinuations due to capsule burden. What is new and conclusion: Swallowing of capsules was not a barrier for drug administration in young patients with DMD. Capsule formulations may be an acceptable alternative to liquid formulations for children as young as 4 years of age.