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NCT02274051 · ClinicalTrials.gov registry record · Phase 1
The Safety and Tolerability of Kinetin, in Patients With Familial Dysautonomia
A Phase 1 study, sponsored by NYU Langone Health.
- Completed
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
NCT02274051: Completed Phase 1 study, sponsored by NYU Langone Health.
NCT02274051 is a Phase 1 study that has completed, run by NYU Langone Health. The registered enrollment target is 15 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (75% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02274051, a Phase 1 study, has completed, sponsored by NYU Langone Health.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
Study 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 autonomic neuropathy type III). FD is a rare fatal autosomal recessive disease in which the growth and development of selective neuronal populations is impaired. The disease is the result of a point mutation in the gene sequence that encodes for kinase complex associated protein (IKAP) in chromosome 9q31. The mutation, at the start of the non-encoding intron 20, weakens the splice site, causing the spliceosome to wrongly join together exons 19 and 21 when transcribing the mRNA strand and miss out exon 20. The mutated mRNA produces a short unstable IKAP protein that is quickly degraded. Interestingly, the mutation does not lead to a complete loss of function. Instead, it results in a tissue specific deficiency in splicing efficiency with both normal (wild type) and mutant IKAP mRNA being expressed in different ratios in different tissues. Some cells, like fibroblasts, produce mostly normal mRNA and protein, where as others, like neurons, produce mostly mutant mRNA and almost no functional protein product.
Primary Outcome
safety blood labs (CBC, metabolic panel)
Interventions
- DIETARY_SUPPLEMENT Kinetin
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2009-11 |
| Est. Completion | 2019-05-04 |
| Phase | Phase 1 |
What the finished NCT02274051 record still lists
NCT02274051 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (75% lower).
The record links to 0 conditions, and to 1 intervention - of which Kinetin is the first listed.
NCT02274051 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02274051 about?
NCT02274051 is a clinical study titled "The Safety and Tolerability of Kinetin, in Patients With Familial Dysautonomia". 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 autonomic neuropathy type III). FD is a rare fatal autosomal recessive disease in which the growth and de...
What is the current status of trial NCT02274051?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2009-11. Estimated completion is 2019-05-04.
What interventions are being tested in trial NCT02274051?
The interventions under investigation include: Kinetin (DIETARY_SUPPLEMENT).
Who is sponsoring clinical trial NCT02274051?
This trial is sponsored by NYU Langone Health, which has 1,164 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02274051's enrollment target sits among peer trials
15 1798th of 2000 higher than 157 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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