Actively Recruiting
Natural History Study for Patients With Nemaline Myopathy in Spain
Led by Hospital Universitari Vall d'Hebron Research Institute · Updated on 2026-03-23
100
Participants Needed
1
Research Sites
313 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The objective of this natural history study is to comprehensively characterize the disease progression and clinical features of nemaline myopathies. The study aims to establish a well-defined cohort of patients in Spain, enabling long-term follow-up and facilitating recruitment for future clinical trials.
CONDITIONS
Official Title
Natural History Study for Patients With Nemaline Myopathy in Spain
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with a confirmed clinical and genetic diagnosis of nemaline myopathy (mutations in ACTA1, NEB, TPM2, TPM3, KBTBD13, CFL2, KLHL40, KLHL41, LMOD3, MYPN, TNNT1, TNNT3), or under discussion if they only have a compatible biopsy
- Signed informed consent by the patient or Legal Authority Responsible, and/or assent by the subject (in pediatric population)
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
University Hospital Vall d'Hebron
Barcelona, Spain, 08035
Actively Recruiting
Research Team
L
Laura Costa-Comellas, M.D.
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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