Improving clinical trials for Ataxia Telangiectasia (A-T)
Methods to Improve Clinical Trials for A-T
['FUNDING_R01'] · JOHNS HOPKINS UNIVERSITY · NIH-10570376
This study is working to improve clinical trials for Ataxia Telangiectasia (A-T) by looking at a wide range of patients to better understand how the condition affects people differently, so they can create a new scale that really reflects what matters to those living with A-T.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | JOHNS HOPKINS UNIVERSITY (nih funded) |
| Locations | 1 site (BALTIMORE, UNITED STATES) |
| Trial ID | NIH-10570376 on ClinicalTrials.gov |
What this research studies
This research aims to enhance the effectiveness of clinical trials for Ataxia Telangiectasia (A-T), a rare genetic disorder. By utilizing a diverse patient population from the largest clinical center for A-T and collaborating with major medical centers and support groups, the project seeks to better understand the variability in disease presentation and severity. The researchers will stratify patients into more specific severity groups and develop an A-T specific functional scale to accurately capture changes in disease severity that matter to patients.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Ataxia Telangiectasia, particularly those who exhibit a range of disease severities.
Not a fit: Patients with conditions unrelated to Ataxia Telangiectasia or those who do not meet the specific criteria for participation may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more tailored and effective clinical trials for patients with A-T, ultimately improving treatment outcomes.
How similar studies have performed: Previous research has shown that stratifying patients based on specific characteristics can lead to more effective clinical trials, suggesting that this approach may yield positive results for A-T as well.
Where this research is happening
BALTIMORE, UNITED STATES
- JOHNS HOPKINS UNIVERSITY — BALTIMORE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LEDERMAN, HOWARD M. — JOHNS HOPKINS UNIVERSITY
- Study coordinator: LEDERMAN, HOWARD M.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.