Recruiting NA Spinal Muscular Atrophy (SMA)

New treatment option for Spinal Muscular Atrophy (SMA)

Official title Long Read Analysis in Spinal Muscular Atrophy - LOREASI

ClinicalTrials.gov ID: NCT07332702

What this study is testing

What is blood sample?

blood sample is an investigational medicine, being studied as a potential treatment for spinal muscular atrophy (sma).

Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.

What it's testing
Spinal Muscular Atrophy (SMA) is a severe neuromuscular disease caused by deletion of the SMN1 gene, with the most severe form leading to death in children without treatment. Genetic counselling to detect couples where both partners are carriers is particularly important.

A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.

Who can take part

Ages 18 and older

You may be able to join if

  • Adult Subject:
  • Subject with either:
  • 1 or 3 copies of the SMN1 gene (control group) and a variable number of copies of the SMN2 gene
  • 2 copies of the SMN1 gene in cis (2+0 genotype) (test group)
  • Affiliation to French health insurance

You likely can't join if

  • Pregnant or breastfeeding women
  • Individuals deprived of liberty by an administrative or judicial decision, or those under guardianship or curatorship
See the full eligibility criteria
Who can join
  • Adult Subject:
  • Subject with either:
  • 1 or 3 copies of the SMN1 gene (control group) and a variable number of copies of the SMN2 gene
  • 2 copies of the SMN1 gene in cis (2+0 genotype) (test group)
  • Affiliation to French health insurance
  • Signed consent form
What rules you out
  • Pregnant or breastfeeding women
  • Individuals deprived of liberty by an administrative or judicial decision, or those under guardianship or curatorship

The study team makes the final eligibility decision.

Where it's taking place

  • Rouen, 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, 18 years and older. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Rouen, France. Enter your location above to see the nearest site and check your eligibility.

Explore other conditions

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.