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Sponsors and Collaborators: |
Neurofibromatosis Consortium at the University of Alabama at Birmingham National Cancer Institute (NCI) |
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Information provided by: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT00652990 |
RATIONALE: Sirolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor.
PURPOSE: This phase II trial is studying how well sirolimus works in treating patients with neurofibromatosis type 1 and plexiform neurofibromas that cannot be removed by surgery.
Condition | Intervention | Phase |
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Neurofibromatosis Type 1 (nf1) Precancerous/Nonmalignant Condition |
Drug: sirolimus |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Open Label |
Official Title: | A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas |
Estimated Enrollment: | 70 |
Study Start Date: | December 2007 |
Estimated Primary Completion Date: | June 2010 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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Stratum 1: Experimental
(Radiographically progressive plexiform neurofibromas): Patients receive oral sirolimus twice daily on days 1-28. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicity.
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Drug: sirolimus
Given orally
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Stratum 2: Experimental
(Lexiform neurofibromas without documented radiographic progression): Patients receive oral sirolimus as in stratum 1. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients with documented radiographic partial or complete response after completion of 6 courses may receive up to 6 additional courses of sirolimus after documentation of the best radiographic response.
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Drug: sirolimus
Given orally
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OBJECTIVES:
Primary
Secondary
OUTLINE: This is a multicenter study. Patients are stratified according to disease status (radiographically progressive plexiform neurofibromas [stratum 1] vs plexiform neurofibromas without documented radiographic progression [stratum 2]).
Patients undergo MRI at baseline, after courses 3, 6, 9, and 15, after every 6 courses, and then at the end of treatment for volumetric analysis of their plexiform neurofibromas.
After completion of study therapy, patients are followed periodically for at least 1 month.
Ages Eligible for Study: | 3 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
Clinical diagnosis* of neurofibromatosis type 1 (NF1), according to NIH Consensus Conference criteria, with plexiform neurofibromas (PN) AND at least 1 of the following diagnostic criteria for NF1:
Inoperable PN that has the potential to cause significant morbidity including, but not limited to, any of the following:
Measurable PN amenable to volumetric MRI analysis
Meets 1 of the following criteria:
Progressive PN, as defined by 1 of the following (stratum 1):
PATIENT CHARACTERISTICS:
Creatinine clearance or radioisotope glomerular filtration rate ≥ 70 mL/min OR creatinine normal based on age as follows:
Fasting LDL cholesterol < 160 mg/dL
No other concurrent severe and/or uncontrolled medical disease that could compromise participation in the study, including any of the following:
No known history of HIV seropositivity or known immunodeficiency
No impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of sirolimus, including any of the following:
PRIOR CONCURRENT THERAPY:
More than 1 week since prior and no concurrent strong CYP3A4 inhibitors, including any of the following:
More than 1 week since prior and no concurrent strong CYP3A4 inducers, including any of the following:
More than 1 week since prior and no other concurrent enzyme-inducing anticonvulsants, including any of the following:
No concurrent chronic treatment with systemic steroids or another immunosuppressive agent
United States, Alabama | |
Lurleen Wallace Comprehensive Cancer at University of Alabama - Birmingham | Recruiting |
Birmingham, Alabama, United States, 35294 | |
Contact: Clinical Trials Office - Lurleen Wallace Comprehensive Cancer 205-934-0309 | |
United States, District of Columbia | |
Children's National Medical Center | Recruiting |
Washington, District of Columbia, United States, 20010-2970 | |
Contact: Clinical Trials Office - Children's National Medical Center 202-884-2549 | |
United States, Illinois | |
University of Chicago Cancer Research Center | Recruiting |
Chicago, Illinois, United States, 60637-1470 | |
Contact: Clinical Trials Office - University of Chicago Cancer Research 773-834-7424 | |
United States, Maryland | |
Warren Grant Magnuson Clinical Center - NCI Clinical Trials Referral Office | Recruiting |
Bethesda, Maryland, United States, 20892-1182 | |
Contact: Clinical Trials Office - Warren Grant Magnusen Clinical Center 888-NCI-1937 | |
United States, Missouri | |
Siteman Cancer Center at Barnes-Jewish Hospital - Saint Louis | Recruiting |
Saint Louis, Missouri, United States, 63110 | |
Contact: David Gutmann, MD, PhD 314-632-7379 | |
United States, Pennsylvania | |
Children's Hospital of Philadelphia | Recruiting |
Philadelphia, Pennsylvania, United States, 19104-4318 | |
Contact: Peter C. Phillips, MD 215-590-2107 | |
United States, Utah | |
Huntsman Cancer Institute at University of Utah | Recruiting |
Salt Lake City, Utah, United States, 84112 | |
Contact: Clinical Trials Office - Huntsman Cancer Institute at Universi 801-581-4477 clinical.trials@hci.utah.edu |
Study Chair: | Brian Weiss, MD | Children's Hospital Medical Center, Cincinnati |
Investigator: | Bruce R. Korf, MD, PhD | Lurleen Wallace Comprehensive Cancer at University of Alabama-Birmingham |
Investigator: | Michael Fisher, MD | Children's Hospital of Philadelphia |
Investigator: | Brigitte C. Widemann, MD | NCI - Pediatric Oncology Branch |
Investigator: | John P. Perentesis, MD | Children's Hospital Medical Center, Cincinnati |
Study ID Numbers: | CDR0000592898, NF-102, NCI-08-C-0096 |
Study First Received: | April 3, 2008 |
Last Updated: | September 22, 2008 |
ClinicalTrials.gov Identifier: | NCT00652990 |
Health Authority: | Unspecified |
plexiform neurofibroma neurofibromatosis type 1 (NF1) |
Sirolimus Precancerous Conditions Clotrimazole Miconazole Tioconazole Neurodegenerative Diseases Neurofibromatosis type 1 Neurofibromatosis 1 Neoplastic Syndromes, Hereditary Heredodegenerative Disorders, Nervous System |
Genetic Diseases, Inborn Neurofibroma Neuromuscular Diseases Peripheral Nervous System Diseases Neurofibromatoses Peripheral Nervous System Neoplasms Neurofibroma, Plexiform Nerve Sheath Neoplasms Nervous System Neoplasms Neurocutaneous Syndromes |
Anti-Infective Agents Neoplasms by Histologic Type Immunologic Factors Antineoplastic Agents Neoplasms, Nerve Tissue Nervous System Diseases Physiological Effects of Drugs |
Antibiotics, Antineoplastic Immunosuppressive Agents Pharmacologic Actions Anti-Bacterial Agents Neoplasms Therapeutic Uses Antifungal Agents |