To assess the safety and tolerability of EDG-5506 in children with DMD
This is a 2-part multi-center, Phase 2 study to evaluate the effect of EDG-5506 on safety, PK and biomarkers of muscle damage in approximately 27 children with DMD treated with oral, once daily EDG-5506 for 12 months. Part A will include a 12-week, randomized, double-blind, placebo-controlled dose-ranging study period followed by Part B, a 40-week open-label extension.
Aged 4 to 9 years, inclusive at screening, with a documented mutation on the DMD gene and phenotype consistent with DMD 2. Able to complete stand from supine in ≤ 10 seconds at the Screening and Baseline visits 3. Able to perform the 4-stair climb in < 10 seconds at the Screening and Baseline visits 4. Male sex at birth 5. Body weight greater than or equal to 15 kg and less than 35 kg at the Screening visit 6. Body mass index (BMI) between 5th and 95th percentile for age (Kuczmarski 2002) 7. Treatment with a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit 8. Able to swallow placebo tablet at the Screening visit 9. Followed by a doctor or medical professional who coordinates DMD care on a regular basis and willingness to disclose participant’s study participation with this medical professional
Principal Investigator