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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 is a Phase 1 study that has completed, run by Boston Children's Hospital. The registered enrollment target is 46 participants.

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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.

Interventions

  • DRUG Placebo
  • DRUG RAD001

Trial Details

FieldValue
Enrollment Target 46 participants
Start Date 2017-06-12
Est. Completion 2021-12-22
Phase Phase 1

Sponsor

Boston Children's Hospital

462 total trials

What the Registry Record Tells You About NCT02991807

The ClinicalTrials.gov registry entry for NCT02991807 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 46 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Boston Children's Hospital, which has 462 total studies on file at ClinicalTrials.gov.

The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.

NCT02991807 reports 0 study locations.

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.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.