NCT03088709

Haploidentical Stem Cell Transplantation Using Post-Transplant Cyclophosphamide

Safety, Efficacy and Feasibility of Haploidentical Stem Cell Transplantation (Haplo-SCT) Using Post-Transplant Cyclophosphamide (PTCy) as an Alternative Donor Source for Patients Who Lack a Matched Sibling/Unrelated Donor Options

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Loyola University · Academic / Other
• Sex: All
• Age: 16 Years – 90 Years
• Healthy volunteers: Not accepted

Summary

Historically, the best results of allogeneic SCT have been obtained when the stem cell donor is a human leukocyte antigen (HLA)-matched sibling, however, this is only available for approximately 30 percent of patients in need for SCT. Alternative donor sources include matched unrelated donor utilizing the donor registry, cord blood transplant and mismatched donor transplant. A human leukocyte antigen (HLA)-haploidentical donor is one who shares, by common inheritance, exactly one HLA haplotype with the recipient, and includes the biologic parents, biologic children and full or half siblings. There is strong body of evidence supporting the use of haplo-SCT in patient who lack a matched sibling or unrelated donor with high rates of successful engraftment, effective Graft Versus Host Disease (GVHD) control and favorable outcomes comparative to those seen using other allograft sources, including HLA-matched sibling SCT. Furthermore, it provides a cost-efficient donor option in a timely manner especially for patients who need to proceed quickly to transplant due to concern of disease relapse/progression.

Detailed description

An open label, single-arm, single-center study to evaluate the safety, efficacy and feasibility of haplo-SCT as an alternative donor source for patients who lack a matched sibling/unrelated donor options. The choice of the chemotherapy treatment for transplantation will be up to the investigator. Post-transplant cyclophosphamide will serve as the backbone of the immunosuppression treatment to prevent GVHD. GVHD Prevention Treatment: Cyclophosphamide will be administered IV on Day 3 and Day 5 post transplant. Tacrolimus will be administered IV until patient can take it by mouth starting on day of transplant and continue approximately 100 days post-transplant. Mycophenolate mofetil will be administered IV until patient can take it by mouth starting on Day 1 post transplant until 28 days.

Conditions

• Acute Myeloid Leukemia
• Acute Lymphocytic Leukemia
• Myelodysplastic Syndrome
• Non-hodgkin Lymphoma
• Chronic Lymphocytic Leukemia

Interventions

Type	Name	Description
DRUG	Cyclophosphamide	IV medication given for prevention of graft versus host disease.
DRUG	Tacrolimus	IV medication given for prevention of graft versus host disease.
DRUG	Mycophenolate mofetil	IV medication given for prevention of graft versus host disease.
OTHER	Haploidentical Stem Cell Transplantation	A stem cell transplant that involves matching a patient's tissue type, specifically their human leukocyte antigen (HLA) tissue type, with that of a related donor.

Timeline

• Start date: 2017-01-18
• Primary completion: 2022-01-31
• Completion: 2022-01-31
• First posted: 2017-03-23
• Last updated: 2024-07-10

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study