Actively Recruiting
The Natural History of Familial Dysautonomia
Led by NYU Langone Health · Updated on 2025-11-10
400
Participants Needed
2
Research Sites
618 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
The Natural History of Familial Dysautonomia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients of any age with a diagnosis of familial dysautonomia (FD) with molecular confirmation of the IKBKAP mutation.
- Ability to provide informed consent (or assent) and comply with the study protocol
You will not qualify if you...
- Subjects that do not wish to be a part of the study.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
Dysautonomia Center - School of Medicine -NYU Langone Medical Center
New York, New York, United States, 10016
Actively Recruiting
2
Sheba Medical Center - Safra Children's Hospital
Tel Litwinsky, Ramat Gan, Israel, 52621
Actively Recruiting
Research Team
B
Britney A. Paredes Lopez
CONTACT
H
Horacio Kaufmann, MD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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