MEHMO Natural History and Biomarkers

Recruiting Observational Study
Intellectual Disability Epilepsy Hypogonadisms Microcephaly Nervous System Malformations Obesity
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.
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At a Glance
Age
1 Week – 100
Sex
Any
Study type
Observational
Participants needed
150 (estimated)
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · NIH
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About This Trial
This observational natural history study will follow individuals with MEHMO (Mental disability, Epileptic seizure, Hypopituitarism/Hypogenitalism, Microcephaly, Obesity) syndrome or an eIF2-pathway related disorder, who have symptoms such as intellectual delay, seizures, abnormal hormone and blood sugar levels, and decreased motor skills. No current treatment for these conditions is available. A …
Trial Locations
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Eligibility Criteria
* INCLUSION CRITERIA: To be eligible to participate in this study, an individual must meet the following criteria: Be \>= 1-week of age if affected, or \>=1-month of age if unaffected. For Screening: 1. Have a combination of signs/symptoms suggestive of MEHMO syndrome, AND no or inconclu…
Contacts

An N Dang Do, M.D.

(301) 496-8849

an.dangdo@nih.gov

CONTACT