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New treatment option for Laminopathies
Official title Modifying Factors in Striated Muscle Laminopathies
ClinicalTrials.gov ID: NCT05394506
What this study is testing
- What it's testing
- Mutations in the LMNA gene, which codes for lamins A and C, proteins of the nuclear lamina, are responsible for a wide spectrum of pathologies, including a group specifically affecting striated skeletal and cardiac muscles, with cardiac involvement being life-threatening. At the skeletal muscle level, a wide phenotypic spectrum has been described, ranging from severe forms of congenital muscular dystrophy to less severe forms of limb-girdle muscular dystrophy.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 and older
You may be able to join if
- Patient with an LMNA mutation that has led to the diagnosis of laminopathy affecting striated muscle
- Presenting the symptoms of the disease, whether they are index cases or related to this index case (muscle weakness, tendon retractions with or...
- Have no contraindication to muscle or skin biopsy, i.e., 1) presence of a history of allergy to latex, antiseptics, local anesthetics and adhesive...
- Patients (adult participant) or both holders of parental authority (minor participant) must sign a free and informed consent. If a minor has only 1...
- Patients affiliated to the general French social security system, to the French Universal Medical Coverage (CMU) or to any French equivalent scheme.
You likely can't join if
- Pregnant or breastfeeding women
- Adult subject to legal protection measures (safeguard of justice, curatorship and guardianship).
The study team makes the final eligibility decision.
Where it's taking place
- Bron, Auvergne-Rhône-Alpes, France
- Paris, France, France
- Montpellier, Hérault, France
- Rennes, Ille-et-Vilaine, France
- Nantes, Loire-Atlantique, France
- Rouen, Normandy, France
- Paris, Paris, France
- Garches, Île-de-France Region, France
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bron, Auvergne-Rhône-Alpes, France; Paris, France, France; Montpellier, Hérault, France; Rennes, Ille-et-Vilaine, France; Nantes, Loire-Atlantique, France; Rouen, Normandy, France and 2 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.