Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)

Summary

The purpose of the study is to establish the pharmacokinetic profile of omigapil in paediatric and adolescent patients with CMD and to evaluate the safety and tolerability of omigapil. Funding source - FDA OOPD

Timeline

Start

2014-12

Primary completion

2017-12-05

Completion

2018-01-29

Outcome

Met primary endpoint

paper The trial met its primary end point PMID 38915423 ↗

release “CALLISTO met its primary objective to establish the pharmacokinetic profile of omigapil, applied as liquid formulation, and demonstrated that in this study the drug was safe” santhera.com ↗

Publications

• Foley AR, Yun P, Leach ME, Neuhaus SB, Averion GV, Hu Y, Hayes LH, Donkervoort S, Jain MS, Waite M, Parks R, Bharucha-Goebel DX, Mayer OH, Zou Y, Fink M, DeCoster J, Mendoza C, Arevalo C, Hausmann R, Petraki D, Cheung K, Bonnemann CG. Phase 1 Open-Label Study of Omigapil in Patients With LAMA2- or COL6-Related Dystrophy. Neurol Genet. 2024 May 29;10(3):e200148. doi: 10.1212/NXG.0000000000200148. eCollection 2024 Jun.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Omigapil	Small molecule	0.02 mg/kg	Oral
Subject	Omigapil	Small molecule	0.04 mg/kg	Oral
Subject	Omigapil	Small molecule	0.06 mg/kg	Oral
Subject	Omigapil	Small molecule	0.08 mg/kg	Oral

Indications