Myotonic DystrophyDM1-Heart-RNCT01136330
DM1 Heart Registry - DM1 Respiratory Registry
An observational study of Myotonic Dystrophy, run by Institut de Myologie, France at 1 site in France.
Trial summary
Myotonic dystrophy type 1 (DM1) is the most frequent neuromuscular disease in adults. DM1 patients have an impaired prognosis (mean age of death <60 years) due to cardiac and respiratory complications. Our primary objective was to identify cardiac and respiratory prognostic factors in DM1. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myotonic Dystrophy
- How many people
- 914 participants
- Where
- 1 siteFrance
- Study dates
- May 2010to Sep 2011
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Myotonic dystrophy type 1
Who may be able to join
- DM1 mutation (>50 CTG repeats)
- Age > 18 years
Who cannot join
- Patient refusal
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Cardiac and Respiratory Prognostic Factors in Patients With Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is the most frequent neuromuscular disease in adults. DM1 patients have an impaired prognosis (mean age of death <60 years) due to cardiac and respiratory complications.
Our primary objective was to identify cardiac and respiratory prognostic factors in DM1.
Read the detailed description
- Patients with genetically proven DM1 who were admitted in Pitié Salpêtrière Hospital from 2000 and 2010 will be identified. These patients systematically underwent neurological, cardiac and respiratory investigations.
- Baseline medical and genetic information will be entered in a dedicated database, including cardiac and respiratory investigations.
- The occurence of severe cardiac and respiratory adverse events will also be collected.
- Statistical analysis will be performed to look for correlations between baseline patient characteristics and cardiac or respiratory adverse events during follow up.
- Conditions
- Myotonic Dystrophy
- Keywords
- Myotonic dystrophy, Prognosis, Sudden death, Respiratory failure
- Medical subject headings
- Myotonic Dystrophy, Death, Sudden, Respiratory Insufficiency
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jun 2010 (estimated)
- Study startMay 2010
- Primary completionSep 2011
- Study completionSep 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Myotonic 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
- Retrospective
- Enrolment
- 914 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institut de Myologie, France
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Karim WAHBI, Institut de Myologie, France
- Investigators
- Denis Duboc, MD,PhD, Study director, APHP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01136330
- Sponsor's protocol ID
- AFM-13286
- Acronym
- DM1-Heart-R
- Status last verified
- Feb 2012
- First submitted
- 2 Jun 2010
- Last updated
- 20 Feb 2012
- Results posted
- No
- Data monitoring committee
- No
Publications
Chong-Nguyen C, Wahbi K, Algalarrondo V, Becane HM, Radvanyi-Hoffman H, Arnaud P, Furling D, Lazarus A, Bassez G, Behin A, Fayssoil A, Laforet P, Stojkovic T, Eymard B, Duboc D. Association Between Mutation Size and Cardiac Involvement in Myotonic Dystrophy Type 1: An Analysis of the DM1-Heart Registry. Circ Cardiovasc Genet. 2017 Jun;10(3):e001526. doi: 10.1161/CIRCGENETICS.116.001526.
PubMed 28611030Wahbi K, Babuty D, Probst V, Wissocque L, Labombarda F, Porcher R, Becane HM, Lazarus A, Behin A, Laforet P, Stojkovic T, Clementy N, Dussauge AP, Gourraud JB, Pereon Y, Lacour A, Chapon F, Milliez P, Klug D, Eymard B, Duboc D. Incidence and predictors of sudden death, major conduction defects and sustained ventricular tachyarrhythmias in 1388 patients with myotonic dystrophy type 1. Eur Heart J. 2017 Mar 7;38(10):751-758. doi: 10.1093/eurheartj/ehw569.
PubMed 27941019Wahbi K, Algalarrondo V, Becane HM, Fressart V, Beldjord C, Azibi K, Lazarus A, Berber N, Radvanyi-Hoffman H, Stojkovic T, Behin A, Laforet P, Eymard B, Hatem S, Duboc D. Brugada syndrome and abnormal splicing of SCN5A in myotonic dystrophy type 1. Arch Cardiovasc Dis. 2013 Dec;106(12):635-43. doi: 10.1016/j.acvd.2013.08.003. Epub 2013 Oct 17.
PubMed 24140416Wahbi K, Meune C, Porcher R, Becane HM, Lazarus A, Laforet P, Stojkovic T, Behin A, Radvanyi-Hoffmann H, Eymard B, Duboc D. Electrophysiological study with prophylactic pacing and survival in adults with myotonic dystrophy and conduction system disease. JAMA. 2012 Mar 28;307(12):1292-301. doi: 10.1001/jama.2012.346.
PubMed 22453570
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cardiac and Respiratory Prognostic Factors in Patients With Myotonic Dystrophy Type 1
- ClinicalTrials.gov ID
- NCT01136330
- Acronym / sponsor ID
- DM1-Heart-R / AFM-13286
- Conditions
- Myotonic Dystrophy
- Keywords
- Myotonic dystrophy, Prognosis, Sudden death, Respiratory failure
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 914 participants (actual)
- Lead sponsor
- Institut de Myologie, FranceUniversity, hospital or other
- Study start
- May 2010
- Primary completion
- Sep 2011 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 3 Jun 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.
Myotonic dystrophy type 1 (DM1) is the most frequent neuromuscular disease in adults. DM1 patients have an impaired prognosis (mean age of death <60 years) due to cardiac and respiratory complications.
Our primary objective was to identify cardiac and respiratory prognostic factors in DM1.
Detailed description
- Patients with genetically proven DM1 who were admitted in Pitié Salpêtrière Hospital from 2000 and 2010 will be identified. These patients systematically underwent neurological, cardiac and respiratory investigations.
- Baseline medical and genetic information will be entered in a dedicated database, including cardiac and respiratory investigations.
- The occurence of severe cardiac and respiratory adverse events will also be collected.
- Statistical analysis will be performed to look for correlations between baseline patient characteristics and cardiac or respiratory adverse events during follow up.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Myotonic dystrophy type 1
Key inclusion criteria (2 listed)
- DM1 mutation (>50 CTG repeats)
- Age > 18 years
Key exclusion criteria (1 listed)
- Patient refusal
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
Not reported in the source record.
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 20 Feb 2012.
- Derived from this study: PubMed 28611030Chong-Nguyen C, Wahbi K, Algalarrondo V, Becane HM, Radvanyi-Hoffman H, Arnaud P, Furling D, Lazarus A, Bassez G, Behin A, Fayssoil A, Laforet P, Stojkovic T, Eymard B, Duboc D. Association Between Mutation Size and Cardiac Involvement in Myotonic Dystrophy Type 1: An Analysis of the DM1-Heart Registry. Circ Cardiovasc Genet. 2017 Jun;10(3):e001526. doi: 10.1161/CIRCGENETICS.116.001526.
- Derived from this study: PubMed 27941019Wahbi K, Babuty D, Probst V, Wissocque L, Labombarda F, Porcher R, Becane HM, Lazarus A, Behin A, Laforet P, Stojkovic T, Clementy N, Dussauge AP, Gourraud JB, Pereon Y, Lacour A, Chapon F, Milliez P, Klug D, Eymard B, Duboc D. Incidence and predictors of sudden death, major conduction defects and sustained ventricular tachyarrhythmias in 1388 patients with myotonic dystrophy type 1. Eur Heart J. 2017 Mar 7;38(10):751-758. doi: 10.1093/eurheartj/ehw569.
- Derived from this study: PubMed 24140416Wahbi K, Algalarrondo V, Becane HM, Fressart V, Beldjord C, Azibi K, Lazarus A, Berber N, Radvanyi-Hoffman H, Stojkovic T, Behin A, Laforet P, Eymard B, Hatem S, Duboc D. Brugada syndrome and abnormal splicing of SCN5A in myotonic dystrophy type 1. Arch Cardiovasc Dis. 2013 Dec;106(12):635-43. doi: 10.1016/j.acvd.2013.08.003. Epub 2013 Oct 17.
- Derived from this study: PubMed 22453570Wahbi K, Meune C, Porcher R, Becane HM, Lazarus A, Laforet P, Stojkovic T, Behin A, Radvanyi-Hoffmann H, Eymard B, Duboc D. Electrophysiological study with prophylactic pacing and survival in adults with myotonic dystrophy and conduction system disease. JAMA. 2012 Mar 28;307(12):1292-301. doi: 10.1001/jama.2012.346.
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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.
Denis Duboc, MD,PhD study director
APHP
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 Myotonic Dystrophy, run by Institut de Myologie, France at 1 site in France.
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 2 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 20 Feb 2012. 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 France. 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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01136330, which also lists 4 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 20 Feb 2012.