Validating biomarkers and clinical measures for CDKL5 Deficiency Disorder
Multi-site validation of biomarkers and core clinical outcome measures for clinical trial readiness in CDKL5 Deficiency Disorder - Supplement
This study is working to create better ways to measure how well new treatments might help kids with CDKL5 Deficiency Disorder, so that we can find the best options for them in future clinical trials.
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-11248090 on NIH RePORTER |
What this research studies
This research focuses on validating biomarkers and clinical outcome measures to prepare for clinical trials in patients with CDKL5 Deficiency Disorder (CDD). It aims to address the challenges in measuring the effectiveness of potential therapies by developing and adapting outcome measures specifically for CDD. The research involves collaboration among experts and utilizes data from animal models to inform the development of these measures. By ensuring that the right tools are in place, the project seeks to facilitate the successful testing of new treatments for this severe condition.
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 success in developing clinical outcome measures for related disorders, indicating a promising approach for this novel focus on CDKL5 Deficiency Disorder.
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.