A phase 1b/2a clinical trial to test the safety, tolerability and pharmacokinetics of single and multiple doses of HT-100 (also called delayed-release halofuginone) in boys with Duchenne muscular dystrophy (DMD) is now open at four sites in Maryland, Missouri and Ohio, with an additional site expected to open in California.
Investigators plan to enroll 30 boys with DMD ages 6 to 20 in the trial.
HT-100, in development by DART Therapeutics in Cambridge, Mass., and affiliate Halo Therapeutics in Newton, Mass., is a small molecule designed to reduce scarring (fibrosis) and inflammation, and promote healthy muscle fiber regeneration in DMD.
DART Therapeutics announced the opening of the trial in a July 15, 2013, press release.
The phase 1b trial will test the safety, tolerability and pharmacokinetics of single and multiple doses of HT-100, in boys ages 6 through 20 — an age range DART says is "atypical," but that will make it possible for study investigators to evaluate the experimental drug's safety in a broad population and study its effects at different stages of disease.
The trial is open label, which means all participants will receive treatment with HT-100, and participants and investigators will know what dose each participant is receiving; no placebos or control drugs will be used.
Trial participants will be assigned to one of five groups, each of which corresponds to a different HT-100 dosing regimen.
The investigators plan to use a noninvasive imaging technique called electrical impedance myography (EIM) to measure muscle health and track changes in the muscle over time.
A six-month 2a extension study is planned.
HT-100 does not target a specific mutation, so, if successful, it potentially could work in DMD caused by any genetic mutation.
The U.S. Food and Drug Administration (FDA) granted HT-100 "orphan drug designation" in the United States in January 2012. The designation, meant to promote development of therapies to treat rare diseases, makes the drug developer eligible for a range of incentives that include a seven-year period of marketing exclusivity in the United States following FDA approval.
In May 2012, the European Medicines Agency granted "orphan medicinal product" designation to HT-100 for the treatment of DMD in the European Union.
Prospective applicants must be boys with DMD ages 6 to 20. In addition, they:
For more information about the trial, see Safety, Tolerability and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy, or enter trial ID NCT01847573 into the search box at ClinicalTrials.gov.
Study site locations are:
California (not yet recruiting)
Nationwide Children's Hospital (recruiting)