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Completed Phase 2 Interventional Results available

A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas

ClinicalTrials.gov ID: NCT00634270

Public ClinicalTrials.gov record NCT00634270. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Oct 27, 2026, 8:33 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

Treatment Overview This phase II study will evaluate the activity of sirolimus in children and adults with NF1 and inoperable plexiform neurofibromas that have the potential to cause significant morbidity. The following disease strata will be studied: Stratum 1: Progressive plexiform neurofibroma(s) that have the potential to cause significant morbidity. The endpoint will be time to tumor progression based on volumetric tumor measurements. Stratum 2: Plexiform neurofibromas without documented radiographic progression at trial entry. The endpoint will be radiographic response. As of May 2009, Stratum 2 was closed to enrollment. Stratum 1 is active.

Study identification

NCT ID
NCT00634270
Recruitment status
Completed
Study type
Interventional
Phase
Phase 2
Enrollment
58 participants

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
3 Years to 75 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Apr 2008 (Type not available)
Primary completion
Nov 2014 (Actual)
Completion
Dec 2015 (Actual)
Last update posted
Nov 17, 2017

2008 – 2015

United States locations

U.S. sites
9
U.S. states
9
U.S. cities
9
Facility City State ZIP Site status
The University of Alabama at Birmingham Birmingham Alabama 35294 —
Children's National Medical Center Washington D.C. District of Columbia 20010 —
University of Chicago Chicago Illinois 60637 —
National Cancer Institute (NCI) Bethesda Maryland 20892 —
Children's Hospital Boston Boston Massachusetts 02115 —
Washington University St Louis Missouri 63110 —
Cincinnati Children's Hospital Medical Center Cincinnati Ohio 45229-4006 —
Children's Hospital of Philadelphia Philadelphia Pennsylvania 19096 —
University of Utah Salt Lake City Utah 84132 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT00634270, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Nov 17, 2017 · Synced Oct 27, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT00634270 live on ClinicalTrials.gov.

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