← Back to Clinical Trials
Recruiting NCT00272883

NCT00272883 Molecular and Genetic Studies of Congenital Myopathies

◆ AI Clinical Summary
Plain-language summary for patients
Clinical Trial Summary
NCT ID NCT00272883
Status Recruiting
Phase
Sponsor Boston Children's Hospital
Condition Central Core Disease
Study Type OBSERVATIONAL
Enrollment 4,000 participants
Start Date 2003-08
Primary Completion 2050-01

Eligibility & Interventions

Sex All sexes
Min Age N/A
Max Age N/A
Study Type OBSERVATIONAL

Eligibility Fast-Check

Enter your details for a quick preliminary check. This does not replace medical advice.

What to Expect as a Participant

This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.

This trial targets 4,000 participants in total. It began in 2003-08 with a primary completion date of 2050-01.

⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.

Brief Summary

In the Congenital Myopathy Research Program at Boston Children's Hospital and Harvard Medical School, the researchers are studying the congenital myopathies (neuromuscular diseases present from birth), including central core disease, centronuclear/myotubular myopathy, congenital fiber type disproportion, multiminicore disease, nemaline myopathy, rigid spine muscular dystrophy, SELENON (SEPN1), RYR1 myopathy, ADSS1 (ADSSL) Myopathy and undefined congenital myopathies. The primary goal of the research is to better understand the genes and proteins (gene products) involved in muscle functioning and disease. The researchers hope that our studies will allow for improved diagnosis and treatment of individuals with congenital myopathies in the future. For more information, visit the Laboratory Website at www.childrenshospital.org/research/beggs

Eligibility Criteria

Inclusion Criteria: * Individuals with a clinical or suspected diagnosis of a congenital myopathy and their family members Exclusion Criteria: * No specific exclusion criteria. Our studies do not include myotonia congenita or related conditions.

Contact & Investigator

Central Contact

Casie Genetti, M.S. C.G.C.

✉ BeggsLabGC@childrens.harvard.edu

📞 (617) 919-2169

Principal Investigator

Alan H. Beggs, Ph.D.

PRINCIPAL INVESTIGATOR

Children's Hospital Boston/Harvard Medical School

Frequently Asked Questions

Who can join the NCT00272883 clinical trial?

This trial is open to participants of all sexes, studying Central Core Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.

Is NCT00272883 currently recruiting?

Yes, NCT00272883 is actively recruiting participants. Contact the research team at BeggsLabGC@childrens.harvard.edu for enrollment information.

Where is the NCT00272883 trial being conducted?

This trial is being conducted at Boston, United States.

Who is sponsoring the NCT00272883 clinical trial?

NCT00272883 is sponsored by Boston Children's Hospital. The principal investigator is Alan H. Beggs, Ph.D. at Children's Hospital Boston/Harvard Medical School. The trial plans to enroll 4,000 participants.

Related Trials

ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional. Full Disclaimer  ·  Last Reviewed: September 2026  ·  Data Methodology