Genetic Developmental and Epileptic Encephalopathy Natural History Study for Clinical Trial Readiness
Recruiting
Observational Study
MEF2C
DEE
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 22,068 (estimated)
- Sponsor
- Weill Medical College of Cornell University · Other
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About This Trial
Phase 0 non-interventional longitudinal study of children and adults with Developmental and Epileptic Encephalopathy (DEE) due to a genetic cause. There are six arms of the study. Arms 1, 2, and 3 are devoted to one example DEE, MEF2C Haploinsufficiency Syndrome (MCHS). Arms 4, 5, and 6 are open to all DEE. Arm 1 (in-person) will enroll children 0 to 15, who will make in-person visits to Weill Cor…
Trial Locations
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Eligibility Criteria
Inclusion Criteria
* Molecular diagnosis of a genetic disorder associated with DEE, as confirmed by the study investigators
* A neurological phenotype such as epilepsy or developmental delay as confirmed by the study investigators.
* English Speaking (Arms 1, 2, 4, 5). The registries may be complet…
Contacts