Donor Stem Cell Transplant Followed by Cyclophosphamide in Treating Patients With Hematological Diseases

NCT ID: NCT02248597

Last Updated: 2023-10-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 27 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-02-25
• Study Completion Date: 2023-01-18

Brief Summary

This pilot clinical trial studies donor stem cell transplant followed by cyclophosphamide in treating patients with hematological diseases. Giving chemotherapy before a donor stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Giving cyclophosphamide after the transplant may stop this from happening.

Detailed Description

PRIMARY OBJECTIVES:

I. To determine if haploidentical stem cell transplant using post-transplant cyclophosphamide results in 60% or better disease free survival (DFS) at 12 months at our institution.

SECONDARY OBJECTIVES:

I. To determine the rate of acute and chronic graft-versus-host disease (GvHD), non-relapse mortality, and relapse.

OUTLINE:

PREPARATIVE REGIMEN: Patients receive fludarabine phosphate intravenously (IV) once daily (QD) on days -6 to -2. Patients receiving myeloablative conditioning receive busulfan IV every 6 hours for 16 doses on days -7 to -4 and patients receiving reduced intensity conditioning receive busulfan IV every 6 hours for 8 doses on days -5 to -4. Patients also receive cyclophosphamide IV QD on days -3 and -2

TRANSPLANT: Patients undergo stem cell transplant on day 0.

GVHD PROPHYLAXIS: Patients receive cyclophosphamide QD on days 3 and 4, tacrolimus on days 5-180, and mycophenolate mofetil on days 5-35.

After completion of study treatment, patients are followed up periodically for 2 years.

Conditions

Graft Versus Host Disease; Hematopoietic/Lymphoid Cancer

Study Design

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

Study Groups

Treatment (stem cell transplant with GVHD prophylaxis)

PREPARATIVE REGIMEN: Patients receive fludarabine phosphate IV QD on days -6 to -2. Patients receiving myeloablative conditioning receive busulfan IV every 6 hours for 16 doses on days -7 to -4 and patients receiving reduced intensity conditioning receive busulfan IV every 6 hours for 8 doses on days -5 to -4. Patients also receive cyclophosphamide IV QD on days -3 and -2

TRANSPLANT: Patients undergo stem cell transplant on day 0.

GVHD PROPHYLAXIS: Patients receive cyclophosphamide QD on days 3 and 4, tacrolimus on days 5-180, and mycophenolate mofetil on days 5-35. Allogeneic hematopoietic stem cell transplantation

Group Type: EXPERIMENTAL

fludarabine phosphate

Intervention Type: DRUG

Given IV

busulfan

Intervention Type: DRUG

Given IV

cyclophosphamide

Intervention Type: DRUG

Given IV

allogeneic hematopoietic stem cell transplantation

Intervention Type: PROCEDURE

Undergo myeloablative or reduced intensity allogeneic stem cell transplant

tacrolimus

Intervention Type: DRUG

mycophenolate mofetil

Intervention Type: DRUG

Interventions

fludarabine phosphate

Given IV

Intervention Type: DRUG

busulfan

Given IV

Intervention Type: DRUG

cyclophosphamide

Given IV

Intervention Type: DRUG

allogeneic hematopoietic stem cell transplantation

Undergo myeloablative or reduced intensity allogeneic stem cell transplant

Intervention Type: PROCEDURE

tacrolimus

Intervention Type: DRUG

mycophenolate mofetil

Intervention Type: DRUG

Other Intervention Names

2-F-ara-AMP; Beneflur; Fludara; BSF; BU; Misulfan; Mitosan; Myeloleukon; CPM; CTX; Cytoxan; Endoxan; Endoxana; FK 506; Prograf; Cellcept; MMF

Eligibility Criteria

Inclusion Criteria

• Diagnosis of a hematological malignancy requiring an allogeneic stem cell transplant consistent with the standard of care
• Remission of any acute hematologic malignancy or adequate disease control for chronic malignancies.
• Ages 18-69 years old.
• Available familial haploidentical (4 to 6 out of 8 HLA loci-matched) donor

Exclusion Criteria

• Significant organ dysfunction defined as: LV EF < 50% (evaluated by echocardiogram or MRI), DLCO or FEV1 < 65% predicted, AST/ALT > 2.5 x ULN, Bilirubin > 1.5 x ULN, Serum creatinine > 2mg/dL, dialysis, or prior renal transplant
• HIV positive (Recipients who are positive for hepatitis B (HBV), hepatitis C (HCV) or human T-cell lymphotropic virus (HTLV-I/II) are not excluded from participation)
• Positive pregnancy test for women of childbearing age.
• Major anticipated illness or organ failure incompatible with survival form transplant.
• Severe psychiatric illness or mental deficiency sufficiently severe as to make compliance with the transplant treatment unlikely and informed consent impossible.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 69 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: collaborator

Wake Forest University Health Sciences

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Dianna S. Howard

Role: PRINCIPAL_INVESTIGATOR

Wake Forest University Health Sciences

Locations

Comprehensive Cancer Center of Wake Forest University

Winston-Salem, North Carolina, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

NCI-2014-01898

Identifier Type: REGISTRY

Identifier Source: secondary_id

CCCWFU 97214

Identifier Type: OTHER

Identifier Source: secondary_id

P30CA012197

Identifier Type: NIH

Identifier Source: secondary_id

View Link

IRB00029210

Identifier Type: -

Identifier Source: org_study_id