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Deflazacort TREATment in LMNArelated
EU CTIS ID: 2024-519672-12-00
What this study is testing
Evaluation of the effect of therapy with Deflazacort drops 0.9 mg/kg/day on clinical outcome and secretome in adult and pediatric patients with L-CMD.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Corticosteroid-naïve patients, aged 3 to 40 years of either sex and of Caucasian origin, with clinical and genetic diagnosis of L-CMD or EDMD2 with age onset before 5 years. Patients will be eligible if they can provide reproducible measurements of forced vital capacity (FVC) (variability <15% between two repeated measures of FVC) at the time of recruitment. Signature of informed consent by the patient, in the case of an adult individual, or parents/legal guardian, in the case of a minor individual.
You likely can't join if
- Hypersensitivity to the active ingredient or any of the excipients Chronic therapy with corticosteroids or immunosuppressive drugs Diabetes mellitus or other comorbidities that strongly contraindicate the use of corticosteroids, such as Active tuberculosis, peptic ulcer, ocular herpes simplex, systemic fungal infections, psychosis; administration of live attenuated vaccine Hereditary fructose intolerance Pregnancy and lactation. Pregnancy and contraception guidelines for clinical trials are attached.
The study team makes the final eligibility decision.
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, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.