[듀센형,DMD,엑손스키핑,Exon]HT-100(근육 상처 및 염증과 싸우는 약물)의 테스트를 위한 다기관 임상오픈

•Affiliates DART Therapeutics와  Halo Therapeutics이  Duchenne 근육 퇴행 위축 (DMD)를 가진 소년의 HT-100의 인내를 시험하는 단계인 1a/2b 임상 시험을 열었습니다. 실험 약물의 안전성과 내약성 및 신체가 약물을 처리하는 방법에 대한 실험입니다.

• HT-100는 근육 조직에 상처 및 염증을 감소시키고 건강한 근육 섬유의 재생을 촉진하도록 설계되었습니다. 잠재적으로 돌연변이의 유형에 의해 발생 DMD에서 효과적 일 수 있습니다.
• 소년 (나이 충족 기준 6-20세) 의 DMD 기준으로 캘리포니아, 메릴랜드, 미주리 오하이오 및 기타 사이트에서 실시되고 30 명이 임상에 참여할 수 있습니다 

엑손스킵이 아닌 치료제 입니다. 이제 1차 이고요 좀 지켜봐야 겠습니다.

원문입니다.

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 mblaustein@halotherapeutics.com; or Jane Davis Golden at (603) 554-6434, or jane@enpointeresearch.com. 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 ClinicalTrials.gov.

출처:http://quest.mda.org/news/dmd-multicenter-trial-test-drug-fights-muscle-scarring-inflammation