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NCT02991807 · ClinicalTrials.gov registry record · Phase 1
RAD001 and Neurocognition in PTEN Hamartoma Tumor Syndrome
A Phase 1 study, sponsored by Boston Children's Hospital.
- Completed
- Registry status
- Phase 1
- Development phase
- 46
- Enrollment target
NCT02991807: Completed Phase 1 study, sponsored by Boston Children's Hospital.
NCT02991807 is a Phase 1 study that has completed, run by Boston Children's Hospital. The registered enrollment target is 46 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (23% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02991807, a Phase 1 study, has completed, sponsored by Boston Children's Hospital.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 46 participants
- Enrollment target
Study Summary
Phosphatase and TENsin homolog (PTEN) gene germline mutations are associated with a spectrum of clinical manifestations characterized by neurocognitive deficits, intellectual disability, autism symptomatology, skin lesions, macrocephaly, hamartomatous overgrowth of tissues, and an increased risk of cancers. Investigators are conducting research to evaluate the potential safety and efficacy of RAD001 (everolimus) in this patient population, and the potential neurocognitive benefits from treatment with RAD001 or placebo for a six month period. The investigators hope this trial will lead to a better understanding of PTEN and to new forms of treatment that may benefit children and adults with PTEN in the future.
Primary Outcome
The primary endpoint will be safety as measured by study drop-out rate due to side effects, comparing everolimus vs. placebo. We will also determine the frequency of adverse events by type and severity. This section displays participant dropout rate and for what reason.
Interventions
- DRUG Placebo
- DRUG RAD001
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 46 participants |
| Start Date | 2017-06-12 |
| Est. Completion | 2021-12-22 |
| Phase | Phase 1 |
What the finished NCT02991807 record still lists
NCT02991807 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 46 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (23% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT02991807 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02991807 about?
NCT02991807 is a clinical study titled "RAD001 and Neurocognition in PTEN Hamartoma Tumor Syndrome". Phosphatase and TENsin homolog (PTEN) gene germline mutations are associated with a spectrum of clinical manifestations characterized by neurocognitive deficits, intellectual disability, autism symptomatology, skin lesions, macrocephaly, hamartomatous overgrowth of tissues, and an increased risk of ...
What is the current status of trial NCT02991807?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 46 participants. The study started on 2017-06-12. Estimated completion is 2021-12-22.
What interventions are being tested in trial NCT02991807?
The interventions under investigation include: Placebo (DRUG), RAD001 (DRUG).
Who is sponsoring clinical trial NCT02991807?
This trial is sponsored by Boston Children's Hospital, which has 462 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02991807's enrollment target sits among peer trials
46 1058th of 2000 higher than 935 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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