DM1NCT02084407
Induction of Pluripotent Stem Cells From Human Fibroblasts of DM1 Patients
A study testing Clinical examination, skin biopsy, blood and urine sampling in people with DM1, run by Institut de Myologie, France at 1 site in France.
Trial summary
The objective of the study is the modelisation of human cardiomyocyts from pluripotent stem cells in order to study the cardiac alterations induced by CTG expansions. Read more about this study
- Who is it for?
- Ages 20 to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- DM1
- What is being tested?
- Clinical examination, skin biopsy, blood and urine sampling
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteFrance
- Study dates
- Nov 2013to Aug 2014
- Phase
- Interventional
- 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 20 to 50 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- aged 20-50
- DM1 confirmed by molecular analysis
- Confirmed cardiomyopathy (infrahissiens conduction disorders)
- able to sign the consent form
- affiliated to european social security.
- aged 20-50
Show all 16Show fewer
- DM1 confirmed by molecular analysis
- No cardiomyopathy (no infrahissiens conduction disorders or other trouble linked to DM1)
- able to sign the consent form
- affiliated to european social security.
- aged over 18
- brother, sister, father or mother of the DM1 patient
- No DM1 confirmed by molecular analysis (CTG repetition under 38)
- No cardiac disorders.
- able to sign the consent form
- affiliated to european social security.
Who cannot join
- no pragnant or nursing women
- patient unable to understand informed consent
- patient under juridic protection
Trial locations and contacts
1 site in 1 country.
About this study
The objective of the study is the modelisation of human cardiomyocyts from pluripotent stem cells in order to study the cardiac alterations induced by CTG expansions.
- Conditions
- DM1
- Being tested
- Clinical examination, skin biopsy, blood and urine sampling
- Keywords
- DM1, Cardiopathy, Adult
- Medical subject headings
- Heart Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Clinical examination, skin biopsy, blood and urine samplingOther
| Study group | Role | What they receive |
|---|---|---|
| DM1 subject with cardiopathy | Active comparator | Clinical examination, Skin biopsy, Blood and urine sampling Receives: Other: Clinical examination, skin biopsy, blood and urine sampling |
| DM1 subject without cardiopathy | Active comparator | Clinical examination, Skin biopsy, Blood and urine sampling Receives: Other: Clinical examination, skin biopsy, blood and urine sampling |
| Not DM1subject | Active comparator | Clinical examination, Skin biopsy, Blood and urine sampling Receives: Other: Clinical examination, skin biopsy, blood and urine sampling |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measure of DM1 alterations in human cardiomyocytes derived from induced pluripotent stem cells
Measured: At baseline
Secondary outcomes (1)
Molecular changes in human DM1 cardiomyocytes expressing pathogenic CUGexp-RNA
Measured: At baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2014 (estimated)
- Study startNov 2013
- Primary completionAug 2014
- Study completionAug 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DM1Studies 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institut de Myologie, France
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Laurent Servais, MD PhD, Principal investigator, Institute of Myology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02084407
- Sponsor's protocol ID
- DM1-Fibroblasts
- Status last verified
- Mar 2014
- First submitted
- 4 Mar 2014
- Last updated
- 26 Sep 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Sep 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Induction of Pluripotent Stem Cells From Human Fibroblasts of DM1 Patients
- ClinicalTrials.gov ID
- NCT02084407
- Acronym / sponsor ID
- DM1-Fibroblasts
- Conditions
- DM1
- Keywords
- DM1, Cardiopathy, Adult
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Institut de Myologie, FranceUniversity, hospital or other
- Study start
- Nov 2013
- Primary completion
- Aug 2014 (actual)
- Study completion
- Aug 2014 (actual)
- First posted
- 12 Mar 2014 (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 objective of the study is the modelisation of human cardiomyocyts from pluripotent stem cells in order to study the cardiac alterations induced by CTG expansions.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| DM1 subject with cardiopathy | Active comparator | Other: Clinical examination, skin biopsy, blood and urine samplingClinical examination, Skin biopsy, Blood and urine sampling |
| DM1 subject without cardiopathy | Active comparator | Other: Clinical examination, skin biopsy, blood and urine samplingClinical examination, Skin biopsy, Blood and urine sampling |
| Not DM1subject | Active comparator | Other: Clinical examination, skin biopsy, blood and urine samplingClinical examination, Skin biopsy, Blood and urine sampling |
Who may qualify
- Ages
- 20 to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (16 listed)
- aged 20-50
- DM1 confirmed by molecular analysis
- Confirmed cardiomyopathy (infrahissiens conduction disorders)
- able to sign the consent form
- affiliated to european social security.
Key exclusion criteria (3 listed)
- no pragnant or nursing women
- patient unable to understand informed consent
- patient under juridic protection
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Clinical examination, skin biopsy, blood and urine sampling | Other | DM1 subject with cardiopathy, DM1 subject without cardiopathy, Not DM1subject |
Primary outcome
Measure of DM1 alterations in human cardiomyocytes derived from induced pluripotent stem cells
Time frame: At baseline
1 secondary outcome
Molecular changes in human DM1 cardiomyocytes expressing pathogenic CUGexp-RNA
Time frame: At baseline
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 26 Sep 2014.
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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.
Laurent Servais, MD PhD principal investigator
Institute of Myology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Clinical examination, skin biopsy, blood and urine sampling in people with DM1, 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 16 inclusion and 3 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 26 Sep 2014. 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.
What does the primary outcome measure?
Measure of DM1 alterations in human cardiomyocytes derived from induced pluripotent stem cells (time frame: At baseline). 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 26 Sep 2014.