Combination Chemotherapy Followed By Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Severe Aplastic Anemia

NCT ID: NCT00054236

Last Updated: 2020-07-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 55 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2002-05-31
• Study Completion Date: 2011-03-31

Brief Summary

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Umbilical cord blood transplantation may be able to replace cells destroyed by chemotherapy.

PURPOSE: Phase I trial to study the effectiveness of combination chemotherapy followed by umbilical cord blood transplantation in treating patients who have hematologic cancer or severe aplastic anemia.

Detailed Description

OBJECTIVES:

• Determine the incidence and severity of acute toxicity in patients with hematologic malignancies or severe aplastic anemia treated with a non-myeloablative conditioning regimen followed by umbilical cord blood transplantation.
• Determine the incidence and severity of acute and chronic graft-versus-host-disease in patients treated with this regimen.
• Determine the incidence of relapse, disease-free survival, and overall survival of patients treated with this regimen.
• Determine the survival rate at 100 days post-transplantation in patients treated with this regimen.
• Determine the incidence of regimen-related complications (infection, hepatic veno-occlusive disease, and interstitial pneumonitis) in patients treated with this regimen.
• Determine the incidence of primary and secondary graft failure in patients treated with this regimen.
• Determine the rates and kinetics of donor-derived lymphoid, myeloid, neutrophil, RBC, and platelet engraftment in patients treated with this regimen.

OUTLINE: Patients receive a non-myeloablative conditioning regimen comprising fludarabine IV over 30 minutes on days -8 to -4, cyclophosphamide IV over 2 hours on days -3 to -2, and anti-thymocyte globulin (ATG) IV over at least 4 hours on days -2 to -1. Patients unable to tolerate ATG may receive methylprednisolone IV over 1 hour on days -3 to -1.

Patients undergo multiple unit umbilical cord blood transplantation on days 0-1. Patients receive filgrastim (G-CSF) subcutaneously beginning on day 7 and continuing until blood counts recover.

Patients are followed monthly for 6 months; at 9, 12, 14, 16, 18, and 24 months; and then annually thereafter.

PROJECTED ACCRUAL: A total of 24 patients will be accrued for this study within 2 years.

Conditions

Chronic Myeloproliferative Disorders; Leukemia; Lymphoma; Multiple Myeloma and Plasma Cell Neoplasm; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Diseases

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

non-myeloablative conditioning regimen

Group Type: EXPERIMENTAL

anti-thymocyte globulin

Intervention Type: BIOLOGICAL

anti-thymocyte globulin (ATG) IV over at least 4 hours on days -2 to -1

filgrastim

Intervention Type: BIOLOGICAL

Patients receive filgrastim (G-CSF) subcutaneously beginning on day 7 and continuing until blood counts recover.

cyclophosphamide

Intervention Type: DRUG

cyclophosphamide IV over 2 hours on days -3 to -2

fludarabine phosphate

Intervention Type: DRUG

fludarabine IV over 30 minutes on days -8 to -4

umbilical cord blood transplantation

Intervention Type: PROCEDURE

Patients undergo multiple unit umbilical cord blood transplantation on days 0-1.

methylprednisolone

Intervention Type: DRUG

Patients unable to tolerate ATG may receive methylprednisolone IV over 1 hour on days -3 to -1.

Interventions

anti-thymocyte globulin

anti-thymocyte globulin (ATG) IV over at least 4 hours on days -2 to -1

Intervention Type: BIOLOGICAL

filgrastim

Patients receive filgrastim (G-CSF) subcutaneously beginning on day 7 and continuing until blood counts recover.

Intervention Type: BIOLOGICAL

cyclophosphamide

cyclophosphamide IV over 2 hours on days -3 to -2

Intervention Type: DRUG

fludarabine phosphate

fludarabine IV over 30 minutes on days -8 to -4

Intervention Type: DRUG

umbilical cord blood transplantation

Patients undergo multiple unit umbilical cord blood transplantation on days 0-1.

Intervention Type: PROCEDURE

methylprednisolone

Patients unable to tolerate ATG may receive methylprednisolone IV over 1 hour on days -3 to -1.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• If under age 55, must meet at least 1 of the following criteria:
• Received extensive prior therapy
• Organ toxicity or infection precluding eligibility for allogeneic transplantation with full ablation conditioning

• Availability of 2-5 umbilical cord blood units that are at least a 4/6 HLA match
• No active CNS disease
• No primary or grade 3 or 4 myelofibrosis

PATIENT CHARACTERISTICS:

Age

• Any age

Performance status

• Karnofsky 70-100% (for patients 16 years of age and older)
• Lansky 50-100% (for patients under 16 years of age)

Life expectancy

• At least 3 months

Hematopoietic

• See Disease Characteristics

Hepatic

• ALT/AST less than 4 times normal
• Bilirubin less than 2.0 mg/dL (unless due to hepatic infiltration by primary malignancy)

Renal

• Creatinine clearance greater than 40 mL/min

Cardiovascular

• Shortening fraction or ejection fraction greater than 40% of normal value for age by echocardiogram or radionuclide scan

Pulmonary

• FVC and FEV_1 greater than 60% of predicted
• DLCO greater than 60% of predicted (adult patients)
• Clearance by pulmonologist required if patient cannot perform pulmonary function tests

Other

• Not pregnant or nursing
• No uncontrolled active infection (viral, bacterial, or fungal)
• HIV negative

PRIOR CONCURRENT THERAPY:

Biologic therapy

• See Disease Characteristics
• More than 3 months since prior autologous stem cell transplantation

Chemotherapy

• See Disease Characteristics
• At least 4 weeks since prior chemotherapy

Endocrine therapy

• Not specified

Radiotherapy

• Not specified

Surgery

• Not specified

Other

• Recovered from prior therapy
• No other concurrent investigational agents that would preclude study participation or increase risk to patient

• Investigational diagnostic procedures allowed

• Maximum Eligible Age: 120 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Case Comprehensive Cancer Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Brenda Cooper, MD

Role: PRINCIPAL_INVESTIGATOR

Case Medical Center, University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center

Locations

Case Medical Center, University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center

Cleveland, Ohio, United States

Countries

United States

Other Identifiers

CWRU6Y01

Identifier Type: -

Identifier Source: org_study_id