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Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment

NCT05099107 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2025-06-19

No results posted yet for this study

Summary

Congenital myopathies (CM) is a large group of muscle disorders, presenting with hypotonia and non-progressive generalised muscle weakness, which can lead to motor developmental delay.More than 20 genes can cause CM and currently there is no curative treatment for this disorder.

Case reports and a smaller study have previous reported that oral salbutamol has benefited subjects with different types of congenital myopathies by increasing their muscle strength.The exact effect of salbutamol in muscle cells isn't exactly known but it has been hypothesized to have an anabolic effect by triggering different pathways inside the muscle cells which increase cell proliferation, decrease apoptosis, decreases proteolysis and increases protein synthesis.

The aim of our study is evaluate if daily oral salbutamol can increase the muscle function and muscle strength in these patients after 6 months on treatment, compared to no treatment.

Conditions

  • Congenital Myopathy
  • Neuromuscular Diseases
  • Musculoskeletal Diseases
  • Nemaline Myopathy
  • Centronuclear Myopathy
  • Myosin Storage Myopathy

Interventions

DRUG

Salbutamol (as Salbutamol Sulfate) 2 Mg Oral Tablet

taken 3 times daily for 6 months

DRUG

Salbutamol Only Product in Oral Dose Form

taken 3 times daily for 6 months

Sponsors & Collaborators

  • Vastra Gotaland Region

    lead OTHER_GOV

Principal Investigators

  • Niklas Darin, M.D · Vastra Gotaland Region

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
CROSSOVER

Eligibility

Min Age
6 Years
Max Age
30 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-10-25
Primary Completion
2025-03-17
Completion
2025-03-17

Countries

  • Sweden

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05099107 on ClinicalTrials.gov