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NCT02276716 · ClinicalTrials.gov registry record · Phase 2
The Nutritional Supplement Phosphatidylserine in Patients With Familial Dysautonomia
A Phase 2 study of Familial Dysautonomia, sponsored by NYU Langone Health.
- Completed
- Registry status
- Phase 2
- Development phase
- 26
- Enrollment target
- 1
- Study location
NCT02276716: Completed Phase 2 study of Familial Dysautonomia, sponsored by NYU Langone Health.
NCT02276716 is a Phase 2 study of Familial Dysautonomia that has completed, run by NYU Langone Health. The registered enrollment target is 26 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (80% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02276716, a Phase 2 study of Familial Dysautonomia, has completed, sponsored by NYU Langone Health.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 26 participants
- Enrollment target
- 1
- Study location
Study Summary
Familial dysautonomia (FD) is a devastating hereditary disease in which the development of selective neuronal populations is impaired because of a deficiency of the protein IKAP (Slaugenhaupt, 2002). There is no known cure. Treatments are supportive, often ineffective and around half of all patients die before reaching age 40 (Axelrod et al., 2002). Phosphatidylserine is an FDA approved food supplement that was shown recently to correct the genetic abnormality and restore IKAP protein levels in cell lines derived from patients with FD (Keren et al., 2011) and a humanized mouse model of the disease (Bochner et al., 2013). Despite its safety and efficacy in this fragile population being unknown, many patients with FD are currently taking phosphatidylserine The investigators propose to conduct a safety, tolerability and early proof of concept efficacy study of phosphatidylserine in patients with FD. The study will be divided into two independent arms. The first phase of the study will be an open-label dose titration study to determine the safety and optimal dose of phosphatidylserine and its effect of normal IKBKAP mRNA levels in 40 patients with FD. The second phase will be a longitudinal observational study in which we will follow, on a yearly basis, patients with FD of all ages who opt to take phosphatidylserine. In this study, we will evaluate the long-term safety of phosphatidylserine in patients with FD and hope to determine whether phosphatidylserine has any impact on the clinical evolution of the disorder. Our long-term goal is to find an effective therapy that will improve the quality of life for patients with FD and alter disease prognosis. We believe that the promise of phosphatidylserine and its availability in health food shops warrants a controlled safety, tolerability and efficacy study to determine whether it should be taken by patients with FD. This study is not intended to determine whether phosphatidylserine has a new indication to treat FD.
Primary Outcome
blood lab values, CBC, metabolic panel,physical exam, vital signs, 12 lead ECG
Conditions Studied
Interventions
- DIETARY_SUPPLEMENT Phosphatidylserine
Study Locations (1)
New York
- NYU Langone Medical Center, Dyautonomia Center, Suite 9Q - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 26 participants |
| Start Date | 2011-11 |
| Est. Completion | 2019-08-01 |
| Phase | Phase 2 |
What the finished NCT02276716 record still lists
NCT02276716 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 26 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (80% lower).
The record links to 1 condition, with Familial Dysautonomia appearing as the primary indexed condition, and to 1 intervention - of which Phosphatidylserine is the first listed.
NCT02276716 reports a single indexed study location in New York.
Frequently Asked Questions
What is clinical trial NCT02276716 about?
NCT02276716 is a clinical study titled "The Nutritional Supplement Phosphatidylserine in Patients With Familial Dysautonomia". Familial dysautonomia (FD) is a devastating hereditary disease in which the development of selective neuronal populations is impaired because of a deficiency of the protein IKAP (Slaugenhaupt, 2002). There is no known cure. Treatments are supportive, often ineffective and around half of all patients...
What is the current status of trial NCT02276716?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 26 participants. The study started on 2011-11. Estimated completion is 2019-08-01.
What conditions does trial NCT02276716 study?
This clinical trial studies the following conditions: Familial Dysautonomia.
What interventions are being tested in trial NCT02276716?
The interventions under investigation include: Phosphatidylserine (DIETARY_SUPPLEMENT).
Who is sponsoring clinical trial NCT02276716?
This trial is sponsored by NYU Langone Health, which has 1,164 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02276716 being conducted?
This trial has 1 study location across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT02276716's enrollment target sits among peer trials
26 1708th of 2000 higher than 282 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
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