Study for Treatment of Cancer in Children With Ataxia-telangiectasia - Full Text View

Purpose

This is a pilot/feasibility study designed to investigate the feasibility of treating children with Ataxia-Telangiectasia (A-T) and cancer with regimens nearly as intense as non-A-T patients with cancer would receive.

Condition	Intervention
Ataxia-Telangiectasia	Drug: vinblastine, vincristine, prednisone, daunorubicin Drug: doxorubicin, methotrexate, cyclophosphamide, L-asparaginase Drug: etoposide, cytarabine, mercaptopurine Drug: dexamethasone, procarbazine Procedure: chemotherapy, intrathecal chemotherapy, steroid therapy

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Pilot Study I for Treatment of Cancer in Children With Ataxia-Telangiectasia

Resource links provided by NLM:

Genetics Home Reference related topics: ataxia-telangiectasia autosomal recessive cerebellar ataxia type 1 complement factor I deficiency infantile-onset spinocerebellar ataxia Marinesco-Sjögren syndrome spinocerebellar ataxia type 1 spinocerebellar ataxia type 2 spinocerebellar ataxia type 3 spinocerebellar ataxia type 6 VLDLR-associated cerebellar hypoplasia

MedlinePlus related topics: Ataxia Telangiectasia Cancer Steroids

Drug Information available for: Dexamethasone Cyclophosphamide Mercaptopurine Prednisone Methotrexate Vinblastine sulfate Cytarabine Procarbazine hydrochloride Procarbazine Vinblastine Dexamethasone acetate Vincristine sulfate Dexamethasone sodium phosphate Asparaginase Methotrexate sodium Daunorubicin Doxorubicin Daunorubicin hydrochloride Doxorubicin hydrochloride Etoposide Etoposide phosphate Daunorubicin citrate

U.S. FDA Resources

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

Primary Outcome Measures:

To determine the feasibility of delivering modified intensive chemotherapy to children with A-T who present with cancer. [ Time Frame: The completion of treatment ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	30
Study Start Date:	September 2002
Estimated Study Completion Date:	September 2013
Primary Completion Date:	August 2006 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1 Acute Lymphoblastic Leukemia (ALL) Low Risk	Drug: vinblastine, vincristine, prednisone, daunorubicin See Detailed Description section for details of treatment interventions. Drug: doxorubicin, methotrexate, cyclophosphamide, L-asparaginase See Detailed Description section for details of treatment interventions. Drug: etoposide, cytarabine, mercaptopurine See Detailed Description section for details of treatment interventions. Drug: dexamethasone, procarbazine See Detailed Description section for details of treatment interventions. Procedure: chemotherapy, intrathecal chemotherapy, steroid therapy See Detailed Description section for details of treatment interventions.
2 Acute Lymphoblastic Leukemia (ALL) - High Risk	Drug: vinblastine, vincristine, prednisone, daunorubicin See Detailed Description section for details of treatment interventions. Drug: doxorubicin, methotrexate, cyclophosphamide, L-asparaginase See Detailed Description section for details of treatment interventions. Drug: etoposide, cytarabine, mercaptopurine See Detailed Description section for details of treatment interventions. Drug: dexamethasone, procarbazine See Detailed Description section for details of treatment interventions. Procedure: chemotherapy, intrathecal chemotherapy, steroid therapy See Detailed Description section for details of treatment interventions.
3A B-Cell Non-Hodgkins Lymphoma (Group A)	Drug: vinblastine, vincristine, prednisone, daunorubicin See Detailed Description section for details of treatment interventions. Drug: doxorubicin, methotrexate, cyclophosphamide, L-asparaginase See Detailed Description section for details of treatment interventions. Drug: etoposide, cytarabine, mercaptopurine See Detailed Description section for details of treatment interventions. Drug: dexamethasone, procarbazine See Detailed Description section for details of treatment interventions. Procedure: chemotherapy, intrathecal chemotherapy, steroid therapy See Detailed Description section for details of treatment interventions.
3B B-Cell Non-Hodgkins Lymphoma (Group B)	Drug: vinblastine, vincristine, prednisone, daunorubicin See Detailed Description section for details of treatment interventions. Drug: doxorubicin, methotrexate, cyclophosphamide, L-asparaginase See Detailed Description section for details of treatment interventions. Drug: etoposide, cytarabine, mercaptopurine See Detailed Description section for details of treatment interventions. Drug: dexamethasone, procarbazine See Detailed Description section for details of treatment interventions. Procedure: chemotherapy, intrathecal chemotherapy, steroid therapy See Detailed Description section for details of treatment interventions.
4 Hodgkins Disease	Drug: vinblastine, vincristine, prednisone, daunorubicin See Detailed Description section for details of treatment interventions. Drug: doxorubicin, methotrexate, cyclophosphamide, L-asparaginase See Detailed Description section for details of treatment interventions. Drug: etoposide, cytarabine, mercaptopurine See Detailed Description section for details of treatment interventions. Drug: dexamethasone, procarbazine See Detailed Description section for details of treatment interventions. Procedure: chemotherapy, intrathecal chemotherapy, steroid therapy See Detailed Description section for details of treatment interventions.

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Eligibility

Ages Eligible for Study:	up to 10 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Patient must have a diagnosis of Ataxia-Telangiectasia (A-T).
Patient must have a diagnosis of either acute lymphoblastic leukemia (ALL) or lymphoma (non-Hodgkin lymphoma or Hodgkin's disease).
Patients with other malignancies (solid tumors, rare malignancies, or relapsed hematopoietic malignancies) will be eligible for the biologic studies of this protocol; they will receive best clinical management chemotherapy.
Patients do not have to be previously untreated. If prior chemotherapy has already started (up through induction), therapy will be continued according to protocol at a clinically appropriate time point.

Exclusion Criteria:

Patients who do not have a diagnosis of Ataxia Telangiectasia (A-T).

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00187057

Locations

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

Sponsors and Collaborators

St. Jude Children's Research Hospital

Children's Hospital of Philadelphia

National Cancer Institute (NCI)

Investigators

Principal Investigator:

John T. Sandlund, MD

St. Jude Children's Research Hospital

More Information

Additional Information:

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

Publications:

Sandlund JT, Kastan MB, Kennedy W, Behm F, Entrekin E, Pui CH, Kalwinsky DT, Raimondi SC. A subtle t(3;8) results in plausible juxtaposition of MYC and BCL6 in a child with Burkitt lymphoma/leukemia and ataxia-telangiectasia. Cancer Genet Cytogenet. 2006 Jul 1;168(1):69-72.

Responsible Party:	St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:	NCT00187057 History of Changes
Other Study ID Numbers:	AT-1
Study First Received:	September 12, 2005
Last Updated:	July 24, 2012
Health Authority:	United States: Institutional Review Board

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

Ataxia
alphafetoprotein

Additional relevant MeSH terms:

Ataxia
Ataxia Telangiectasia
Telangiectasis
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinocerebellar Ataxias
Cerebellar Ataxia
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Neurocutaneous Syndromes
Vascular Diseases
Cardiovascular Diseases

Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
6-Mercaptopurine
Cytarabine
Methotrexate
Cyclophosphamide
Asparaginase
Daunorubicin
Dexamethasone
Doxorubicin
Etoposide
Prednisone

ClinicalTrials.gov processed this record on May 23, 2013