Background:
Availability of easy to swallow formulations contributes to medication compliance. Literature suggests that solid dosage forms are acceptable for school age children; data in children with Duchenne muscular dystrophy (DMD) is limited. In edasalonexent clinical trials, capsule size selection and ability to swallow among trial participants were assessed.
Methods:
The Phase 3 PolarisDMD randomized, double-blind, placebo-controlled trial is evaluating safety and efficacy of edasalonexent in boys with DMD ages 4 to 7 (up to 8th birthday); 151 boys screened at 37 sites in 8 countries. The completed MoveDMD Phase 2 trial enrolled boys with DMD in the same age range; 33 boys screened at 5 US sites. Both trials required the ability to swallow soft-gel 100 mg or 250 mg size capsules. Supportive strategies included pill-swallowing techniques and aids like Medi-straw®.
Results:
In both Phase 2 and 3, 97% of boys screened were able to swallow capsules. In Phase 2, 77% of the enrolled started on the smaller capsule with most transitioning to the larger capsule by Week 24. Compliance was high (~98%) with no discontinuations due to capsule burden. In Phase 3, while 80 of 131 enrolled (61%) selected the larger, 250 mg, capsule initially, most of those initially on 100 mg capsules have transitioned to the 250 mg capsule. Sites that only offered the larger size capsule found that all boys were able to take the larger capsule. There were no obvious geographical differences in ability to swallow larger capsules and size selection was not correlated with age.
Conclusions:
Edasalonexent capsules were well-accepted in boys with DMD. Site experience, clinical trial personnel confidence and initial capsule size presentation had the most significant impact in selection. In these trials, age did not impact ability to take capsules. Soft-gel capsules can successfully be used in clinical trials of boys as young as 4 years with appropriate support.