NCT01182324 - Clinical Trial References

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Citation(s)

Cohen PD, Herman L, Jedlinski S, Willocks P, Wittekind P
Ethical issues in clinical neuroscience research: a patient's perspective. Neurotherapeutics. 2007 Jul;4(3):537-44. Review.
Holroyd J, Guthrie D
Family stress with chronic childhood illness: cystic fibrosis, neuromuscular disease, and renal disease. J Clin Psychol. 1986 Jul;42(4):552-61.
Webb CL
Parents' perspectives on coping with Duchenne muscular dystrophy. Child Care Health Dev. 2005 Jul;31(4):385-96.

The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor

Details for clinical trial NCT01182324

Clinical trial NCT01182324 - References