Phase 2
N=27
Donor Stem Cell Transplant Followed by Cyclophosphamide in Treating Patients With Hematological Diseases
Graft Versus Host Disease · Hematopoietic/Lymphoid Cancer
Bottom Line
View on ClinicalTrials.gov: NCT02248597 ↗Enrolled (actual)
27
Serious AEs
100.0%
Results posted
Oct 2023
Primary outcome: Primary: 12 Month Disease Free Survival Probability — 51.8 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- fludarabine phosphate (Drug); busulfan (Drug); cyclophosphamide (Drug); allogeneic hematopoietic stem cell transplantation (Procedure); tacrolimus (Drug); mycophenolate mofetil (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Wake Forest University Health Sciences
- Primary completion
- Jan 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 12 Month Disease Free Survival Probability |
51.8 | — |
| SECONDARY Rate of Acute GvHD |
51.8 | — |
| SECONDARY Overall Survival |
66.7 | — |
| SECONDARY Progression Free Survival |
51.8 | — |
| SECONDARY Relapse-free Mortality |
86.8 | — |
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.
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 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.
Data sourced from ClinicalTrials.gov (NCT02248597). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.