RECRUITING

MEHMO Natural History and Biomarkers

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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 major impediment to the testing of potential therapeutic interventions is the lack of well-defined outcome measures. This protocol seeks to identify biochemical and clinical markers to monitor disease progression, and better understand the natural history of these conditions. Any person diagnosed with MEHMO syndrome or related conditions, who can travel to the NIH Clinical Center can participate in this study. The study involves: * General health assessment and evaluation * Imaging studies * Laboratory tests * Collection of blood, urine, spinal fluid, skin biopsy.

Official Title

Investigations of Individuals With MEHMO Syndrome or eIF2-Pathway Related Conditions

Quick Facts

Study Start:2024-04-24
Study Completion:2053-09-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06019182

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:1 Week to 100 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Contact

An N Dang Do, M.D.
CONTACT
(301) 496-8849
an.dangdo@nih.gov

Principal Investigator

An N Dang Do, M.D.
PRINCIPAL_INVESTIGATOR
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Study Locations (Sites)

National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
United States

Collaborators and Investigators

Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

  • An N Dang Do, M.D., PRINCIPAL_INVESTIGATOR, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-04-24
Study Completion Date2053-09-01

Study Record Updates

Study Start Date2024-04-24
Study Completion Date2053-09-01

Terms related to this study

Keywords Provided by Researchers

  • MEHMO
  • X-linked MEHMO Syndrome
  • eIF2-Pathway Related Conditions
  • EIF2S3

Additional Relevant MeSH Terms

  • Intellectual Disability
  • Epilepsy
  • Hypogonadisms
  • Microcephaly
  • Nervous System Malformations
  • Obesity