CMD: Phase 1 Omigapil Trial Remains Open

Santhera Pharmaceuticals is testing anti-cell death compound omigapil in children and teens with merosin-deficient and Ullrich congenital muscular dystrophies

Article Highlights:

  • Omigapil is an experimental drug that has shown promise in mice with a disorder resembling merosin-deficient congenital muscular dystrophy.
  • The compound inhibits apoptosis, a type of cell death process.
  • A phase 1 trial of omigapil is ongoing and open in Bethesda, Md.
by Margaret Wahl on March 26, 2015 - 6:57pm

Santhera Pharmaceuticals continues to recruit participants for a phase 1 trial of omigapil in children and adolescents with the type 1A form of congenital muscular dystrophy (CMD), also known as MDC1A and merosin-deficient CMD; or with the Ullrich form of CMD.

About omigapil

Omigapil interferes with a type of cell death known as apoptosis. A 2009 study found that mice with a disorder mimicking type 1A CMD that received treatment with omigapil showed less apoptosis and more protection of muscle tissue, reduced body weight loss, and increased motor activity compared to their untreated counterparts. 

For more, see the omigapil section of the Santhera website.

About the omigapil in CMD trial

The trial is taking place at the National Institutes of Health in Bethesda, Md., under the direction of neuromuscular disease specialist Carsten Bönnemann. It is slated to include 24 children and adolescents ages 5-16 with type 1A CMD or Ullrich CMD who meet clinical, genetic and tissue-based study criteria. All criteria are described in the trial announcement on the website (see below).

The purpose of this phase 1 trial is to see how the body handles omigapil (such as how the drug is absorbed and distributed), whether the drug is safe, and how well it is tolerated.

To participate

Contact study coordinator Gilberto (Mike) Averion at (301) 594-2760 or For complete information, see the announcement on the site at  Ascending, Multiple-Dose Cohort Study in Children and Adolescents of Safety and Tolerability of Omigapil in Congenital Muscular Dystrophy (CALLISTO); or enter NCT01805024 in the search box at

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