DMD: Multicenter Trial To Test Drug that Fights Muscle Scarring, Inflammation

30-person trial will test the safety, tolerability and pharmacokinetics of the experimental drug HT-100 in boys with Duchenne muscular dystrophy

Article Highlights:
  • Affiliates DART Therapeutics and Halo Therapeutics have opened a phase 1a/2b clinical trial to test the pharmacokinetics (the way the body handles a drug), safety and tolerability of the experimental drug HT-100 in boys with Duchenne muscular dystrophy (DMD).
  • HT-100 is designed to reduce scarring and inflammation in muscle tissue and promote healthy muscle fiber regeneration. It potentially could be effective in DMD caused by any type of mutation.
  • Boys ages 6 to 20 who have DMD and meet other criteria may be eligible to participate in the 30-person trial, which is being conducted at sites in California, Maryland, Missouri and Ohio.
by Amy Madsen on July 30, 2013 - 1:51pm

Update (Jan. 6, 2014): In a Dec. 23, 2013, announcement, Halo said the U.S. Food and Drug Administration (FDA) had placed this trial on "clinical hold" because of some adverse events that occurred in dogs being treated with HT-100. No further dosing of patients will occur until this issue has been resolved. For details, see the Dec. 23 Q&A from Halo.

Update (Nov. 14, 2013): Recruitment for this trial is complete. MDA began funding the development of HT-100 in November 2013.

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.

Trial will test single and multiple doses of HT-100

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.

In addition:

  • Trial investigators will monitor adverse events, data from physical evaluations, clinical laboratory test results and other diagnostic tests to assess the safety and tolerability of HT-100.
  • To assess pharmacokinetics — the way the body handles a drug — blood levels of HT-100 and other data will be assessed.
  • Early pharmacodynamic signals of HT-100 will be assessed after four weeks of continuous dosing, to include measurements of pulmonary (breathing) function, motor function, muscle composition, and biochemical and imaging markers.

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 potentially could work in all DMD

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.

How to participate in the trial

Prospective applicants must be boys with DMD ages 6 to 20. In addition, they:

  • must have confirmation that they are producing little or no dystrophin — the protein deficient or absent in DMD;
  • may be ambulatory (walking) or nonambulatory;
  • must not be taking corticosteroids, or be on a stable dose and treatment regimen for a minimum of 12 months; and
  • must meet other criteria.

Contact Marc Blaustein at (617) 431-7250, or; or Jane Davis Golden at (603) 554-6434, or Or, contact the study site near you.

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

Study site locations are:

California (not yet recruiting)
University of California, Davis Medical Center, Sacramento, Calif.

Maryland (recruiting)
Kennedy Krieger Institute, Johns Hopkins School of Medicine, Baltimore, Md.
Principal investigator: Kathryn Wagner
Contact: Genila Bibat (

Missouri (recruiting)
Washington University School of Medicine, St. Louis, Mo.
Principal investigator: Anne Connolly
Contact: Linda Schimmoeller (

Cincinnati Children's Hospital Medical Center (recruiting)
Cincinnati, Ohio
Principal investigator: Brenda Wong
Contact: Ma Leilani (Lani) Relucio (

Nationwide Children's Hospital (recruiting)
Columbus, Ohio
Principal investigator: Kevin Flanigan
Contact: Susan Gailey (

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