Global registry for FKRP-related muscular dystrophies
Global Fukutin-Related Protein Registry
This study is collecting information from people with FKRP-related muscular dystrophies to help improve care and find candidates for future clinical trials.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 1000 (estimated) |
| Sex | All |
| Sponsor | Newcastle University Academic / other |
| Locations | 1 site (Newcastle upon Tyne) |
| Trial ID | NCT04001595 on ClinicalTrials.gov |
What this trial studies
The Global FKRP Registry aims to gather comprehensive data on patients with FKRP-related conditions, including Limb Girdle Muscular Dystrophy type R9 and other related disorders. This observational registry collects demographic, clinical, and genetic information through an online questionnaire, which is updated annually. The data will help identify potential participants for future clinical trials and improve standards of care for these conditions. The registry is maintained by Newcastle University and is supported by various research organizations.
Who should consider this trial
Good fit: Ideal candidates for this registry are individuals with a confirmed diagnosis of an FKRP-related condition, verified through genetic testing.
Not a fit: Patients without a confirmed FKRP-related condition or those who do not wish to participate in data collection may not benefit from this registry.
Why it matters
Potential benefit: If successful, this registry could enhance understanding of FKRP-related conditions and facilitate access to clinical trials for patients.
How similar studies have performed: Other registries for rare diseases have shown success in improving patient outcomes and facilitating clinical trials, indicating a positive precedent for this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * All patients with a confirmed diagnosis of an FKRP-related condition are eligible for inclusion. Diagnosis will be confirmed via genetic testing results. Exclusion Criteria: * There is no exclusion criteria for registration with this patient registry.
Where this trial is running
Newcastle upon Tyne
- John Walton Muscular Dystrophy Research Centre, Newcastle University — Newcastle upon Tyne, United Kingdom (Recruiting)
Study contacts
- Principal investigator: Volker Straub, MD, PhD — John Walton Muscular Dystrophy Research Centre
- Study coordinator: Patient Registry Manager and Curator
- Email: fkrpregistry@newcastle.ac.uk
- Phone: 0191 2418640
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.