Duchenne Muscular DystrophyNCT01182324
The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor
An observational study of Duchenne Muscular Dystrophy, run by National Human Genome Research Institute (NHGRI) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Jul 2010to 24 Jun 2013
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- -Must meet inclusion criteria.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Human Genome Research Institute (NHGRI), 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
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.
Read the detailed description
Some aspects of the traditional process for the development of new potential therapeutics for rare disorders and the ensuing research on safety and efficacy are changing. This is due, in part, to the increased ability of families, parents, individuals with rare disorders, and advocacy organizations to access and control information, to provide monetary or other incentives, and to communicate directly with biopharmaceutical companies and clinical investigators. Increasingly, advocacy organizations (and by extension, the families they serve) create or participate in disorder registries intended to facilitate research by industry and access of families to clinical trials; fund promising bench and clinical research; and lobby for outcomes beneficial to drug developers and researchers, such as increased funding or facilitated FDA approval. The involvement of families in the research process is likely to have implications for how willing individuals are to participate in studies, their expectations and perceptions of the study experience, and their behavior during the study. Their increased level of involvement may also affect how families perceive and respond to a clinical trial in which the results are not as promising as hoped, as quickly as hoped. This partnership approach has implications for biopharmaceutical companies who sponsor treatment trials and for clinical investigators who lead and implement the trials. This qualitative interview study aims to describe, from the perspectives of parents of children involved in a clinical trial, research clinicians implementing the trial, and the company sponsoring the trial, the experience of this new, more collaborative research process. In this case, we will examine the Ataluren trial for Duchenne muscular dystrophy. The results from this study are hypothesis-generating for downstream research and can inform biopharmaceutical companies and clinician researchers as they plan and implement clinical trials for rare disorders and help guide advocacy groups and parent advocates as they partner with researchers and industry.
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Expectations, Duchenne Muscular Dystrophy, Clinical Trial, Motivations
- Medical subject headings
- Muscular Dystrophy, Duchenne
What is being measured
The primary outcome is the main question the study is designed to answer.
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...
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2010 (estimated)
- Study start30 Jul 2010
- Study completion24 Jun 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Time perspective
- Retrospective
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- Sponsor type
- US National Institutes of Health
- Investigators
- Barbara B Biesecker, Principal investigator, National Human Genome Research Institute (NHGRI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01182324
- Sponsor's protocol ID
- 999910171
- Other IDs
- 10-HG-N171
- Status last verified
- 24 Jun 2013
- First submitted
- 13 Aug 2010
- Last updated
- 5 Apr 2018
- Results posted
- No
Publications
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.
PubMed 17599719Holroyd 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.
PubMed 3745452Webb 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.
PubMed 15948875
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor
- ClinicalTrials.gov ID
- NCT01182324
- Acronym / sponsor ID
- 999910171
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Expectations, Duchenne Muscular Dystrophy, Clinical Trial, Motivations
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- National Human Genome Research Institute (NHGRI)US National Institutes of Health
- Study start
- 30 Jul 2010
- Study completion
- 24 Jun 2013
- First posted
- 16 Aug 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Some aspects of the traditional process for the development of new potential therapeutics for rare disorders and the ensuing research on safety and efficacy are changing. This is due, in part, to the increased ability of families, parents, individuals with rare disorders, and advocacy organizations to access and control information, to provide monetary or other incentives, and to communicate directly with biopharmaceutical companies and clinical investigators. Increasingly, advocacy organizations (and by extension, the families they serve) create or participate in disorder registries intended to facilitate research by industry and access of families to clinical trials; fund promising bench and clinical research; and lobby for outcomes beneficial to drug developers and researchers, such as increased funding or facilitated FDA approval. The involvement of families in the research process is likely to have implications for how willing individuals are to participate in studies, their expectations and perceptions of the study experience, and their behavior during the study. Their increased level of involvement may also affect how families perceive and respond to a clinical trial in which the results are not as promising as hoped, as quickly as hoped. This partnership approach has implications for biopharmaceutical companies who sponsor treatment trials and for clinical investigators who lead and implement the trials. This qualitative interview study aims to describe, from the perspectives of parents of children involved in a clinical trial, research clinicians implementing the trial, and the company sponsoring the trial, the experience of this new, more collaborative research process. In this case, we will examine the Ataluren trial for Duchenne muscular dystrophy. The results from this study are hypothesis-generating for downstream research and can inform biopharmaceutical companies and clinician researchers as they plan and implement clinical trials for rare disorders and help guide advocacy groups and parent advocates as they partner with researchers and industry.
Study design
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (1 listed)
- -Must meet inclusion criteria.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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...
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Apr 2018.
- Background: PubMed 17599719Cohen 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.
- Background: PubMed 3745452Holroyd 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.
- Background: PubMed 15948875Webb 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Barbara B Biesecker principal investigator
National Human Genome Research Institute (NHGRI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy, run by National Human Genome Research Institute (NHGRI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.... It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01182324, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Apr 2018.