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The Natural History of Familial Dysautonomia

NCT03920774 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 400

Last updated 2025-11-10

No results posted yet for this study

Summary

The study will collect clinical information from patients with FD and allow them to give blood to help develop biological markers of the disease to aid diagnosis and treatment.

This is a non-invasive, non-interventional, observation study that poses only minimal risk for participants. The study will document the clinical features of patients with FD overtime by storing their routine clinical test results in a central database. The study will involve collaborators at other specialist clinics around the world who follow/evaluate patients with FD annually. Providing blood for future use is optional.

Conditions

  • Familial Dysautonomia (Riley-Day Syndrome)
  • Hereditary Sensory and Autonomic Neuropathies
  • Hereditary Sensory and Autonomic Neuropathy 3

Sponsors & Collaborators

Principal Investigators

  • Horacio Kaufmann, MD · NYU Langone Health

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-02-22
Primary Completion
2027-02-21
Completion
2028-12-31

Countries

  • United States
  • Israel

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03920774 on ClinicalTrials.gov