Clinical trial NCT00118326: Donor Bone Marrow Transplant in Treating Young Patients With Cancer or a Non-Cancerous Disease
Official title: Feasibility of Granulocyte-Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow From Pediatric Donors as a Stem Cell Source for Allogeneic Bone Marrow Transplant
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Kidney Cancer
Leukemia
Lymphoma
Myelodysplastic Syndromes
Neuroblastoma
Sarcoma
Keywords: de novo myelodysplastic syndromes
disseminated neuroblastoma
previously treated myelodysplastic syndromes
recurrent neuroblastoma
secondary myelodysplastic syndromes
childhood acute myeloid leukemia in remission
childhood acute lymphoblastic leukemia in remission
childhood chronic myelogenous leukemia
juvenile myelomonocytic leukemia
previously treated childhood rhabdomyosarcoma
recurrent Wilms tumor and other childhood kidney tumors
recurrent/refractory childhood Hodgkin lymphoma
recurrent childhood acute lymphoblastic leukemia
recurrent childhood acute myeloid leukemia
recurrent childhood large cell lymphoma
recurrent childhood lymphoblastic lymphoma
recurrent childhood rhabdomyosarcoma
recurrent childhood small noncleaved cell lymphoma
untreated childhood acute lymphoblastic leukemia
untreated childhood acute myeloid leukemia and other myeloid malignancies
Lead sponsor: Fred Hutchinson Cancer Center
Sponsor class: OTHER
First posted: 2005-07-11
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

Brief summary: RATIONALE: A bone marrow transplant from a brother or sister may be able to replace blood-forming cells that were destroyed by chemotherapy or radiation therapy. Colony-stimulating factors, such as G-CSF, cause the body to make blood cells. Giving G-CSF to the donor may help the body make more stem cells that can be collected for bone marrow transplant and may cause fewer side effects in the patient after the transplant.

PURPOSE: This phase I/II trial is studying the side effects of donor bone marrow transplant and to see how well it works in treating young patients with cancer or a non-cancerous disease.

Detailed description: OBJECTIVES:

Primary

* Determine the safety and feasibility of filgrastim (G-CSF)-mobilized bone marrow from an HLA-identical pediatric sibling donor as a stem cell source for pediatric patients undergoing allogeneic bone marrow transplantation for malignant or non-malignant disease.

Secondary

* Determine the time to neutrophil and platelet engraftment, number of red blood cell and platelet transfusions, number of febrile days, and number of hospitalization days in patients treated with this regimen.
* Determine the number of nucleated cells and CD34-positive cells, absolute lymphocyte count, and lymphocyte subsets (CD3/CD4/CD8) in G-CSF-mobilized bone marrow from these donors.

OUTLINE: This is a multicenter, pilot study.

Donors receive filgrastim (G-CSF) subcutaneously once daily on days -4 to 0. Donors then undergo standard bone marrow harvest on day 0.

Patients receive pre-transplantation conditioning and graft-versus-host disease prophylaxis according to the disease for which the patient is being treated and the treatment plan or clinical trial for which the patient is enrolled on. Patients undergo allogeneic bone marrow transplantation on day 0.

After completion of bone marrow harvest, donors are followed at 7 and 30 days. After completion of study treatment, patients are followed for 100 days post-transplantation and then periodically thereafter.

PROJECTED ACCRUAL: A total of 80 participants (40 donors and 40 patients) will be accrued for this study within 18 months.

Eligibility criteria: DISEASE CHARACTERISTICS:

* Patients (recipients):

  * Undergoing a myeloablative or nonmyeloablative allogeneic bone marrow transplantation for 1 of the following diseases:

    * Hematologic malignancy
    * Non-hematologic malignancy
    * Non-malignant disease
  * Not undergoing T-cell depleted bone marrow transplantation
* Donors:

  * Healthy sibling of a patient meeting eligibility requirements for this protocol
  * HLA-identically matched with patient

PATIENT CHARACTERISTICS:

Age

* 18 and under (patient and donor)

Performance status

* Karnofsky 90-100% (donor) OR
* Lansky 90-100% (donor)

Life expectancy

* Not specified

Hematopoietic

* No sickle cell anemia (donor)

Hepatic

* Not specified

Renal

* Not specified

Immunologic

* HIV negative (patient and donor)
* No uncontrolled bacterial, viral, fungal, or parasitic infection (donor)
* No potentially life threatening autoimmune disease (donor)

Other

* Not pregnant or nursing (patient and donor)
* Fertile patients must use effective contraception (patient)
* No other illness that would severely limit life expectancy (patient)
* No pre-existing medical condition that would confer a high risk for bone marrow donation (donor)
* No medical condition or psychiatric trait that would preclude G-CSF administration or bone marrow harvesting (donor)

PRIOR CONCURRENT THERAPY:

Biologic therapy

* More than 4 years since prior allogeneic blood transfusion (donor)
* No concurrent growth factors post-transplantation (donor)

Chemotherapy

* Not specified

Endocrine therapy

* Not specified

Radiotherapy

* Not specified

Surgery

* Not specified

Other

* Concurrent participation in another treatment clinical trial allowed provided the use of filgrastim (G-CSF)-mobilized bone marrow is not excluded (patient)
* No other concurrent investigational agents (donor)

Primary outcomes:
measure: Safety and feasibility
Secondary outcomes:
Other outcomes:
Locations:
city: Nashville
country: United States
facility: Vanderbilt-Ingram Cancer Center
state: Tennessee
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Research Center
state: Washington
Study officials:
affiliation: Fred Hutchinson Cancer Center
name: Ann E. Woolfrey, MD
role: PRINCIPAL_INVESTIGATOR
References: