Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02248597
Recruitment Status : Recruiting
First Posted : September 25, 2014
Last Update Posted : November 26, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Wake Forest University Health Sciences

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.

Condition or disease Intervention/treatment Phase
Graft Versus Host Disease Hematopoietic/Lymphoid Cancer Drug: fludarabine phosphate Drug: busulfan Drug: cyclophosphamide Procedure: allogeneic hematopoietic stem cell transplantation Drug: tacrolimus Drug: mycophenolate mofetil Phase 2

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.


Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Haploidentical Stem Cell Transplant Using Post Transplant Cyclophosphamide for GvHD Prophylaxis: A Pilot Study
Actual Study Start Date : February 25, 2015
Estimated Primary Completion Date : February 2020
Estimated Study Completion Date : February 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: 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

Drug: fludarabine phosphate
Given IV
Other Names:
  • 2-F-ara-AMP
  • Beneflur
  • Fludara

Drug: busulfan
Given IV
Other Names:
  • BSF
  • BU
  • Misulfan
  • Mitosan
  • Myeloleukon

Drug: cyclophosphamide
Given IV
Other Names:
  • CPM
  • CTX
  • Cytoxan
  • Endoxan
  • Endoxana

Procedure: allogeneic hematopoietic stem cell transplantation
Undergo myeloablative or reduced intensity allogeneic stem cell transplant

Drug: tacrolimus
Other Names:
  • FK 506
  • Prograf

Drug: mycophenolate mofetil
Other Names:
  • Cellcept
  • MMF




Primary Outcome Measures :
  1. 12 month disease free survival probability [ Time Frame: At 12 months ]
    The proportion of patients who experience death or disease relapse by one year will be calculated and a corresponding 95% confidence interval will be constructed using the normal approximation for binomial proportions. The survival function will be estimated and plotted using the method of Kaplan and Meier.


Secondary Outcome Measures :
  1. Rate of acute GvHD [ Time Frame: At 12 months ]
    Calculating the rates of acute GvHD in the study population at twelve months with a 95% confidence interval.

  2. Rate of chronic GvHD [ Time Frame: At 12 months ]
    Calculating the rates of acute GvHD in the study population at twelve months with a 95% confidence interval.

  3. Overall survival [ Time Frame: At 12 months ]
    The survival function will be estimated and plotted using the method of Kaplan and Meier.

  4. Relapse (or death) [ Time Frame: At 12 months ]
    Calculating the proportion of patients who experience death by one year and construct a corresponding 95% confidence interval using the normal approximation for binomial proportions.

  5. Relapse-free mortality [ Time Frame: At 12 months ]
    The cumulative incidence function in the presence of relapse will be estimated.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 69 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02248597


Locations
Layout table for location information
United States, North Carolina
Comprehensive Cancer Center of Wake Forest University Recruiting
Winston-Salem, North Carolina, United States, 27157
Contact: Brittany Mabe, RN    336-713-5440      
Principal Investigator: Dianna S. Howard         
Sponsors and Collaborators
Wake Forest University Health Sciences
National Cancer Institute (NCI)
Investigators
Layout table for investigator information
Principal Investigator: Dianna S. Howard Wake Forest University Health Sciences

Layout table for additonal information
Responsible Party: Wake Forest University Health Sciences
ClinicalTrials.gov Identifier: NCT02248597     History of Changes
Other Study ID Numbers: IRB00029210
NCI-2014-01898 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
CCCWFU 97214 ( Other Identifier: Comprehensive Cancer Center of Wake Forest University )
P30CA012197 ( U.S. NIH Grant/Contract )
First Posted: September 25, 2014    Key Record Dates
Last Update Posted: November 26, 2019
Last Verified: November 2019

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Graft vs Host Disease
Immune System Diseases
Mycophenolic Acid
Cyclophosphamide
Busulfan
Fludarabine
Fludarabine phosphate
Tacrolimus
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Calcineurin Inhibitors
Enzyme Inhibitors
Antimetabolites, Antineoplastic
Antimetabolites
Antibiotics, Antineoplastic
Antibiotics, Antitubercular
Antitubercular Agents
Anti-Bacterial Agents
Anti-Infective Agents