Everolimus for Treating Pediatric Patients With Recurrent or Refractory Tumors - Full Text View

Sponsors and Collaborators:	St. Jude Children's Research Hospital Novartis
Information provided by:	St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:	NCT00187174

Purpose

Patients with recurrent or refractory solid tumors or brain tumors that are unresponsive to conventional therapy, or with no known effective therapy, will be treated in the Phase I component of the study. Patients with recurrent or refractory rhabdomyosarcoma or non-rhabdomyosarcoma will be treated in the Phase II component of the study. Experiments in the laboratory have shown the experimental drug RAD001C (RAD001, Everolimus) can prevent cells from multiplying. RAD001 is now being tested in diseases such as cancer, in which excessive cell multiplication needs to be stopped. The drug has been tested in adult cancer patients and has been well tolerated by subjects in these studies. It is experimental and, therefore, available in clinical trials.

Condition	Intervention	Phase
Tumors Brain Tumors Rhabdomyosarcoma Sarcoma, Soft Tissue	Drug: Everolimus	Phase I Phase II

MedlinePlus related topics: Brain Cancer Cancer Childhood Brain Tumors Soft Tissue Sarcoma

Drug Information available for: Everolimus

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment
Official Title:	Phase I/II Trial of RAD001C (Everolimus) in Pediatric Patients With Recurrent Refractory Solid Tumors or Brain Tumors With Phase II Limited to Recurrent or Refractory Rhabdomyosarcomas and Non Rhabdomyosarcomatous Soft Tissue Sarcomas

Further study details as provided by St. Jude Children's Research Hospital:

Primary Outcome Measures:

To find the highest dose of RAD001 that can be given to children with refractory or relapsing solid tumors, leukemias, or brain tumors without causing severe side effects [ Time Frame: Within 30 days per subject ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

To find out if there are changes in the body's blood cells and tumor cells after treatment with RAD001 [ Time Frame: Within 30 days per subject ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	24
Study Start Date:	October 2004
Estimated Primary Completion Date:	October 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Phase 1: Experimental	Drug: Everolimus The drug is given orally in 28 day courses. The MTD determined in Phase I portion will be given to patients with recurrent or refractory rhabdomyosarcoma Phase II will include recurrent or refractory nonrhabdomyosarcomatous soft tissue sarcoma
Phase 2: Experimental	Drug: Everolimus The drug is given orally in 28 day courses. The MTD determined in Phase I portion will be given to patients with recurrent or refractory rhabdomyosarcoma Phase II will include recurrent or refractory nonrhabdomyosarcomatous soft tissue sarcoma

Detailed Description:

Initially the Phase I component of the study will include participants with the diagnosis of a recurrent or refractory solid tumor or brain tumor that is unresponsive to conventional therapy or with no known effective therapy. The main goal in this phase of the study is to establish the maximum tolerated dose (highest dose that can be given safely to participants). Participants with recurrent or refractory solid tumors or brain tumors will be enrolled in this component of the study. The tolerability of the maximum dose established with solid tumors or brain tumors will be studied in participants with recurrent or refractory leukemias. The Phase II portion of the study will look at the effectiveness of the drug. It will need to include two groups of participants, those with recurrent or refractory rhabdomyosarcomas and those with recurrent or refractory non-rhabdomyosarcomatous soft tissue sarcomas.

Eligibility

Ages Eligible for Study:	3 Years to 21 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Adequate performance status
Adequate bone marrow, kidney, heart, and liver function

Exclusion Criteria:

Must not be receiving concomitant anti-cancer treatment
Must not be pregnant

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00187174

Locations

United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105

Sponsors and Collaborators

St. Jude Children's Research Hospital

Novartis

Investigators

Principal Investigator:

Wayne L Furman, MD

St. Jude Children's Research Hospital

More Information

St. Jude Children's Research Hospital This link exits the ClinicalTrials.gov site

Responsible Party:	St. Jude Children's Research Hospital ( Wayne L. Furman, MD / Principal Investigator )
Study ID Numbers:	RAD001
Study First Received:	September 13, 2005
Last Updated:	September 26, 2008
ClinicalTrials.gov Identifier:	NCT00187174
Health Authority:	United States: Food and Drug Administration

Keywords provided by St. Jude Children's Research Hospital:

Brain tumors
Soft Tissue Sarcomas
Refractory solid tumors
Recurrent or Refractory Rhabdomyosarcomatous and Non-Rhabdomyosarcoma Soft Tissue Sarcomas

Study placed in the following topic categories:

Everolimus
Neoplasms, Connective and Soft Tissue
Brain Neoplasms
Malignant mesenchymal tumor
Sarcoma
Central Nervous System Diseases

Central Nervous System Neoplasms
Brain Diseases
Soft tissue sarcomas
Nervous System Neoplasms
Recurrence
Rhabdomyosarcoma

Additional relevant MeSH terms:

Neoplasms, Muscle Tissue
Neoplasms
Neoplasms by Histologic Type
Neoplasms by Site
Immunologic Factors

Myosarcoma
Physiological Effects of Drugs
Nervous System Diseases
Immunosuppressive Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 15, 2009