Status:
COMPLETED
The Safety and Tolerability of Kinetin, in Patients With Familial Dysautonomia
Lead Sponsor:
NYU Langone Health
Conditions:
Familial Dysautonomia
Eligibility:
All Genders
16+ years
Phase:
PHASE1
Brief Summary
This is a study of kinetin, a nutritional supplement that corrects the mRNA splicing defect in patients with familial dysautonomia (FD, also known as Riley Day syndrome or hereditary sensory and auton...
Detailed Description
Familial dysautonomia (FD, also called Riley Day syndrome or hereditary sensory and autonomic neuropathy type III) is an autosomal recessive disease caused by a point mutation in the kinase complex as...
Eligibility Criteria
Inclusion
- Male of female patients aged 16 and older
- Confirmed diagnosis of familial dysautonomia by genetic testing
- Written informed consent to participate in the trial and understanding that they can withdraw consent at anytime without affecting their future care.
- Ability to comply with the requirements of the study procedures.
Exclusion
- Patients who have taken other nutritional supplements that may affect IKAP mRNA splicing within the last 30 days
- Patients with a known hypersensitivity to any component of the nutritional supplement kinetin
- Patients with atrial fibrillation, angina or an electrocardiogram documenting significant abnormality that may jeopardize the patient's health.
- Patients with significant pulmonary, liver, renal (creatinine \>2.5 mg/ml) or cardiac illness
- Women who are pregnant or lactating
- Women of childbearing potential who are not using medically accepted methods of contraception.
- Patients who have a significant abnormality on clinical examination that may, in the investigator's opinion, jeopardize their healthy participating in this pilot trial.
- Patients taking allopurinol, other xanthine oxidase inhibitors or other compounds that may interfere with the metabolism of kinetin including oral calcium supplements.
Key Trial Info
Start Date :
November 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 4 2019
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT02274051
Start Date
November 1 2009
End Date
May 4 2019
Last Update
June 18 2019
Active Locations (1)
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1
NYU Langone Medical Center
New York, New York, United States, 10016