ataluren 임상3상 시작

• 3 단계임상은  약물ataluren 읽기를 통해 정지 코돈 220 명으로 임상을 진행 Duchenne 또는 난센스 돌연변이 (조기 정지 코돈)에 의한 베커 근 위축증을 가진 사람들을 위해 열었습니다.

• PTC 치료학에 의해 실시되는 임상은  지금 신시내티, 오하이오, 사이트 만 열린 상태이지만 세계의 많은 사이트에서 열 예정입니다.

 • 이전 단계 2b 예심에서 결과는 약물의 특정 매일 투약 처방 넌센스 돌연변이에 의해 발생 DMD 또는 BMD와 소년의 보행 능력을의 감소를 둔화되는 것을 확인했습니다.

 • PTC 치료학에 2005 년 부터 MDA 지원을 통해 개발되고있습니다. 결과는 2015년 6월입니다.

원문입니다.

A large-scale, multinational, phase 3 trial of the experimental drug ataluren has opened its first trial site, in Cincinnati, Ohio.

The trial is recruiting boys with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) caused by a nonsense mutation — also known as a premature stop codon — in the dystrophin gene. This type of mutation causes cells to stop synthesizing a protein before the process is complete, resulting in a short, nonfunctional protein. Nonsense mutations are believed to cause DMD or BMD in approximately 10 to 15 percent of boys with these disorders.

Ataluren — sometimes referred to as a stop codon read-through drug — has the potential to overcome the effects of a nonsense mutation and allow functional dystrophin — the muscle protein that's missing in Duchenne MD and deficient in Becker MD — to be produced.

The orally delivered drug is being developed by PTC Therapeutics, a South Plainfield, N.J., biotechnology company, to which MDA gave a $1.5 million grant in 2005.

In 2010, PTC provided an analysis of results of a phase 2b trial of ataluren showing that a specific dosage regimen of the drug taken for 48 weeks slowed the decline of walking ability, as measured by the distance walked by trial participants in six minutes.

Trial to test ataluren's effect on walking

The new 48-week, phase 3 ataluren trial is slated to enroll 220 participants at multiple sites, with the goal of determining the effect of ataluren on walking ability in DMD or BMD caused by a nonsense mutation.

The only trial site currently open is at Cincinnati Children's Hospital Medical Center, but PTC plans to open sites around the world.

One group will take ataluren three times a day — 10 milligrams per kilogram of body weight in the morning and at midday and 20 milligrams per kilogram in the evening. Another group will take a placebo three times a day. Neither the trial participants nor the investigators will know who is taking which substance until after the trial has been completed.

Assessments will be performed every eight weeks.

The primary outcome measure is the six-minute walk test — the distance that can be walked in six minutes.

Secondary outcome measures include evaluations of physical function, assessments of activities of daily living and disease symptoms, quality-of-life measurements, safety tests, ataluren blood levels, and assessments of how well participants complied with the treatment.

Results are expected after June 2015.