Long-term observation of rare genetic brain disorders
Longitudinal Study of Neurodegenerative Disorders
University of Pittsburgh · NCT03333200
This study is tracking how rare genetic brain disorders progress over time to better understand their effects on patients.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 1500 (estimated) |
| Sex | All |
| Sponsor | University of Pittsburgh (other) |
| Locations | 1 site (Pittsburgh, Pennsylvania) |
| Trial ID | NCT03333200 on ClinicalTrials.gov |
What this trial studies
This observational study aims to track the progression of rare genetic neurodegenerative disorders such as MLD, Krabbe Disease, ALD, MPS I, and MPS II. Patients will be assessed by a multidisciplinary team at designated intervals: every 3 months during the first year, every 6 months in the second year, and annually thereafter. The collected data will help researchers understand the natural history of these conditions and the impact of various interventions.
Who should consider this trial
Good fit: Ideal candidates for this study include any patient diagnosed with a genetic neurodegenerative disorder.
Not a fit: Patients who do not have a genetic neurodegenerative disorder will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the progression of rare neurodegenerative disorders, potentially leading to improved management and treatment strategies.
How similar studies have performed: While this study focuses on rare conditions, similar longitudinal studies have shown success in understanding other neurodegenerative disorders, suggesting a potential for valuable findings.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Any patient with a genetic neurodegenerative disorder Exclusion Criteria: * none
Where this trial is running
Pittsburgh, Pennsylvania
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (RECRUITING)
Study contacts
- Study coordinator: Deepa Rajan, MD
- Email: rajands@upmc.edu
- Phone: 412-692-8388
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.
Conditions: MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis