Natural History Study for Patients With Nemaline Myopathy in Spain

Recruiting now

Conditions studied: Nemaline Myopathy, Myopathies, Myopathic Conditions, NEB, ACTA1, TPM2, TPM3, TNNT1, CFL2, KBTBD13, KLHL40, KLHL41, LMOD3, Cohort Studies, MYPN, TNNT3

In brief

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.

Key facts

Study ID

NCT07488806

Run by

Hospital Universitari Vall d'Hebron Research Institute

People needed

100

Starts

2026-06-01

Expected to finish

2032-06-01

Last updated by the study team

2026-03-23

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patients with a confirmed clinical and genetic diagnosis of MN (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).

Where it is running

• University Hospital Vall d'Hebron — Barcelona, Spain (enrolling)

Full record on ClinicalTrials.gov

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