Duchenne Muscular Dystrophypre U7-53NCT01385917
Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
An observational study of Duchenne Muscular Dystrophy, run by Genethon at 2 sites in 2 countries.
Trial summary
PreU7-53 is a natural history study. The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with Duchenne Muscular Dystrophy (DMD), potentially treatable with AAV-mediated exon 53 skipping. Read more about this study
- Who is it for?
- Ages 12 to 20 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 45 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Oct 2011to Dec 2018
- 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 12 to 20 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Patients with theoretically exon 53 skipping-treatable DMD
Who may be able to join
- Diagnosis of Duchenne muscular dystrophy confirmed by at least genetic testing, theoretically treatable by exon 53 skipping.
- Age between ≥ 12 and <20 years old.
- Non ambulant patients (i;e; inability to walk more than 10 meters without any of assistance).
- Patients covered by a national health insurance scheme.
- Signed informed consent.
Who cannot join
- Patient incapable of sitting upright in a wheelchair for at least one hour.
- Patients with severe intellectual impairment preventing them from fully understanding the exercises to be performed.
- Recent (less than 6 months ago) upper limb surgery or trauma This criteria is however no definitive. Patients who have undergone upper limb surgery or trauma may nonetheless be enrolled once the 6 month period is over.
- Known immune deficiency.
- Contraindications to NMR exams
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
France1 site, 1 recruiting
- Recruiting
Myology Institute
Paris
Laurent Servais, MD, 01 42 16 66 47, l.servais@institut-myologie.org
Laurent SERVAIS, MD
United Kingdom1 site, 1 recruiting
- Recruiting
Great ormond Street Hospital & University College London Hospital
London, WC1N 1EH
Francesco Muntoni, Pr, HD, +44 1 02079052111, f.muntoni@ich.ucl.ac.uk
About this study
Official title: Study of Clinical and Radiological Changes in Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
PreU7-53 is a natural history study. The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with Duchenne Muscular Dystrophy (DMD), potentially treatable with AAV-mediated exon 53 skipping.
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, strength and function tests, MRI
- Medical subject headings
- Muscular Dystrophy, Duchenne
What is being measured
The primary outcome is the main question the study is designed to answer.
PreU7-53 is a natural history study
The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with DMD, potentially treatable with AAV-mediated exon 53 skipping.
Measured: Every year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2011 (estimated)
- Study startOct 2011
- Primary completionDec 2018 (estimated)
- Study completionDec 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Genethon
- Sponsor type
- University, hospital or other
- Collaborators
- Institute of Myology
- Responsible party
- Sponsor
- Investigators
- Laurent SERVAIS, MD, Principal investigator, Myology Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01385917
- Sponsor's protocol ID
- GHN007.10
- Acronym
- pre U7-53
- Status last verified
- Mar 2016
- First submitted
- 29 Jun 2011
- Last updated
- 12 Apr 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Clinical and Radiological Changes in Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
- ClinicalTrials.gov ID
- NCT01385917
- Acronym / sponsor ID
- pre U7-53 / GHN007.10
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, strength and function tests, MRI
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- GenethonUniversity, hospital or other
- Collaborators
- Institute of Myology
- Study start
- Oct 2011
- Primary completion
- Dec 2018 (estimated)
- Study completion
- Dec 2018 (estimated)
- First posted
- 30 Jun 2011 (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.
PreU7-53 is a natural history study. The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with Duchenne Muscular Dystrophy (DMD), potentially treatable with AAV-mediated exon 53 skipping.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 12 to 20 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients with theoretically exon 53 skipping-treatable DMD
Key inclusion criteria (5 listed)
- Diagnosis of Duchenne muscular dystrophy confirmed by at least genetic testing, theoretically treatable by exon 53 skipping.
- Age between ≥ 12 and <20 years old.
- Non ambulant patients (i;e; inability to walk more than 10 meters without any of assistance).
- Patients covered by a national health insurance scheme.
- Signed informed consent.
Key exclusion criteria (5 listed)
- Patient incapable of sitting upright in a wheelchair for at least one hour.
- Patients with severe intellectual impairment preventing them from fully understanding the exercises to be performed.
- Recent (less than 6 months ago) upper limb surgery or trauma This criteria is however no definitive. Patients who have undergone upper limb surgery or trauma may nonetheless be enrolled once the 6 month period is over.
- Known immune deficiency.
- Contraindications to NMR exams
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
PreU7-53 is a natural history study
Time frame: Every year
The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with DMD, potentially treatable with AAV-mediated exon 53 skipping.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Apr 2016.
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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.
Great ormond Street Hospital & University College London Hospital
London, United Kingdom
Laurent SERVAIS, MD principal investigator
Myology Institute
2 sites in 2 countries: 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 Genethon at 2 sites in 2 countries.
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 5 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 12 Apr 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in France, United Kingdom. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PreU7-53 is a natural history study (time frame: Every year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 12 Apr 2016.