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

NCT01182324 | Completed

• 2 other identifiers: 99991017110-HG-N171
• Study Type: observational
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to describe the experiences of parents, clinician researchers, and industry professionals who were involved in phase II clinical trials of Ataluren for Duchenne muscular dystrophy. We are especially interested in learning about motivations for being involved in the clinical trial, expectations of the trial, the experience of the trial, and relationships between the parents of children involved in the trial, the clinician researchers, and PTC Therapeutics. In addition, we would like to learn more about whether and how families and advocacy organizations experiences in following the progress of the drug, encouraging the clinical trial, and supporting the phase II trials may have affected participants thoughts and feelings about the study.

Conditions

Duchenne Muscular Dystrophy

Keywords

Expectations; Duchenne Muscular Dystrophy; Clinical Trial; Motivations

Outcome Measures

Primary Outcomes (1)

• To describe, inclusive of the perspectives and voices of all of the major participants, the shared experiences of parents, clinician researchers, and industry professionals who were involved in phase II clinical trials for Duchenne Muscular Dyst...

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• US residents over 18 years of age who have at least one child with Duchenne Muscular Dystrophy who was enrolled in the phase IIa extension trial or phase IIb trial of PTC124. Participants must be a primary caregiver for their children, must have been involved in deciding whether the child would participate in the clinical trial, and must have accompanied their child to at least one visit to the clinical trial site.
• Clinician researchers over 18 years of age who were involved with implementing the clinical trial at a study site.
• Representatives of PTC Therapeutics over 18 years of age.
• All participants must be willing and able to complete an approximately 1-hour long telephone interview in English.

Sponsors & Collaborators

• National Human Genome Research Institute (NHGRI): lead

Study Sites (1)

National Human Genome Research Institute (NHGRI), 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• 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. doi: 10.1016/j.nurt.2007.04.008.

  PMID: 17599719 BACKGROUND

• 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. doi: 10.1002/1097-4679(198607)42:43.0.co;2-8.

  PMID: 3745452 BACKGROUND

• Webb CL. Parents' perspectives on coping with Duchenne muscular dystrophy. Child Care Health Dev. 2005 Jul;31(4):385-96. doi: 10.1111/j.1365-2214.2005.00518.x.

  PMID: 15948875 BACKGROUND

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Barbara B Biesecker

  National Human Genome Research Institute (NHGRI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Time Perspective: RETROSPECTIVE
• Sponsor Type: NIH

Study Record Dates

• First Submitted: August 13, 2010
• First Posted: August 16, 2010
• Study Start: July 30, 2010
• Study Completion: June 24, 2013
• Last Updated: April 5, 2018

Record last verified: 2013-06-24

Locations

US (1)