Understanding TANGO2-related Disorder and Creating a Biorepository
Natural History Study and Establishment of a Biorepository-TANGO2-related Disorder
This study is collecting samples from people with TANGO2 deficiency to learn more about the condition and help find better treatments.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 300 (estimated) |
| Ages | 0 Days and up |
| Sex | All |
| Sponsor | Baylor College of Medicine Academic / other |
| Locations | 1 site (Houston, Texas) |
| Trial ID | NCT05374616 on ClinicalTrials.gov |
What this trial studies
This observational study focuses on individuals with TANGO2 deficiency, a rare genetic disorder that can lead to severe health issues such as rhabdomyolysis and cardiac arrhythmias. The study aims to establish a biorepository of biological samples from affected individuals to support scientific research and create a comprehensive clinical database. By understanding the natural history of the disease, researchers hope to gain insights into its mechanisms and develop potential therapies to improve patient outcomes.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals diagnosed with pathogenic TANGO2 variants.
Not a fit: Patients without TANGO2-related disease will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to better understanding and management of TANGO2-related disorder, ultimately improving patient care.
How similar studies have performed: While this study is focused on a rare condition, similar approaches in establishing biobanks for genetic disorders have shown promise in advancing understanding and treatment options.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: All patients with pathogenic TANGO2 variants will be included. Exclusion Criteria: Patients who do not have TANGO2-related disease will be excluded.
Where this trial is running
Houston, Texas
- Baylor College of Medicine — Houston, Texas, United States (Recruiting)
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.