Validating biomarkers and clinical measures for CDKL5 deficiency disorder
Multi-Site Validation of Biomarkers and Core Clinical Outcome Measures for Clinical Trials Readiness in CDKL5 Deficiency Disorder
This study is working to create better ways to measure how well new treatments for CDKL5 deficiency disorder (CDD) are working, so that kids with CDD can have access to effective therapies in the future.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Colorado Denver NIH-funded |
| Lab location | 1 site (Aurora, UNITED STATES) |
| Project ID | NIH-11031358 on NIH RePORTER |
What this research studies
This research focuses on CDKL5 deficiency disorder (CDD), a severe condition caused by mutations in the CDKL5 gene that leads to significant cognitive and motor challenges. The project aims to develop and validate clinical outcome measures that can be used in future clinical trials, ensuring that therapies targeting CDD can be effectively evaluated. By leveraging insights from animal models and existing clinical data, the research seeks to create a robust framework for assessing treatment efficacy in patients with CDD. This work is crucial for overcoming barriers to clinical trial readiness and ensuring that new therapies can be tested successfully.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with CDKL5 deficiency disorder, particularly those experiencing cognitive and motor dysfunction.
Not a fit: Patients with other forms of developmental and epileptic encephalopathy that do not involve CDKL5 mutations may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective clinical trials and ultimately better treatment options for patients with CDKL5 deficiency disorder.
How similar studies have performed: Previous research has shown promise in developing outcome measures for related disorders, suggesting that this approach could be effective for CDKL5 deficiency disorder as well.
Where this research is happening
Aurora, UNITED STATES
- University of Colorado Denver — Aurora, United States (Active)
Researchers
- Principal investigator: Benke, Timothy a — University of Colorado Denver
- Study coordinator: Benke, Timothy a
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.