Clinical trial NCT02248597: Donor Stem Cell Transplant Followed by Cyclophosphamide in Treating Patients With Hematological Diseases
Official title: Haploidentical Stem Cell Transplant Using Post Transplant Cyclophosphamide for GvHD Prophylaxis: A Pilot Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Graft Versus Host Disease
Hematopoietic/Lymphoid Cancer
Lead sponsor: Wake Forest University Health Sciences
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
First posted: 2014-09-25
Enrollment: 27
Sex: ALL
Minimum age (years): 18
Maximum age (years): 69
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: 12 Month Disease Free Survival Probability
timeFrame: At 12 months
Secondary outcomes:
measure: Rate of Acute GvHD
timeFrame: 12 months
measure: Overall Survival
timeFrame: At 12 months
measure: Progression Free Survival
timeFrame: At 12 months
measure: Relapse-free Mortality
timeFrame: At 12 months
Other outcomes:
Locations:
city: Winston-Salem
country: United States
facility: Comprehensive Cancer Center of Wake Forest University
state: North Carolina
Study officials:
affiliation: Wake Forest University Health Sciences
name: Dianna S. Howard
role: PRINCIPAL_INVESTIGATOR
References: