Hematopoietic cell transplantation using related donors for blood cancers
Nonmyeloablative Hematopoietic Stem Cell Transplantation (SCT) for High-Risk Hematologic Malignancies With Related, HLA-Haploidentical Donors: A Phase II Trial of Immunosuppression With Cyclophosphamide Administered Before and After SCT
PHASE2 · European Institute of Oncology · NCT01374841
This study is testing a new type of stem cell transplant using related donors to see if it can safely help patients with serious blood cancers who don't have a matched donor.
Quick facts
| Phase | PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | European Institute of Oncology (other) |
| Drugs / interventions | chemotherapy, cyclophosphamide |
| Locations | 1 site (Milan) |
| Trial ID | NCT01374841 on ClinicalTrials.gov |
What this trial studies
This study aims to evaluate the safety and effectiveness of non-myeloablative hematopoietic stem cell transplantation (HSCT) for patients with high-risk hematologic malignancies who lack HLA-matched donors. The approach involves using peripheral stem cells from haploidentical donors, combined with pre and post-transplant cyclophosphamide to enhance engraftment and reduce the risk of graft-versus-host disease (GVHD). By targeting patients with specific high-risk conditions, the study seeks to expand treatment options for those who would otherwise have limited alternatives.
Who should consider this trial
Good fit: Ideal candidates include patients aged 70 years or younger with specific high-risk hematologic malignancies such as AML, ALL, CML, MDS, or multiple myeloma.
Not a fit: Patients with low-risk hematologic malignancies or those over 70 years old may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could provide a viable treatment option for patients with high-risk blood cancers who do not have a fully matched donor.
How similar studies have performed: Previous studies have shown promise in using haploidentical donors for HSCT, but this specific approach with cyclophosphamide is still being evaluated.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients ≤70 years old * Eligible diagnoses: * CML in AP * AML with high-risk cytogenetics \[del(5q)/-5, del(7q)/-7, abnormal 3q, 9q, 11q, 20q, 21q, 17p, t(6:9), t(9;22), complex karyotypes (≥3 abnormalities)\] in CR1 * AML ≥ CR2; patients should have \<5% marrow blasts at the time of transplant * High-risk ALL defined as: CR1 with high-risk cytogenetics t(9;22), t(8;14), t(4;11), t(1;19) for adult patients \>4 wk to achieve CR1 ≥ CR2 Patients should have \<5% marrow blasts at the time of transplant * MDS (\>int-1 per IPSS) after ≥ 1 prior cycle of induction chemotherapy; should have\<5% marrow blasts at the time of transplant * MM Stage II or III patients who have progressed after an initial response to chemotherapy or autologous HSCT or MM patients with refractory disease who may benefit from tandem autologous-nonmyeloablative allogeneic transplant * CLL, NHL or HD who are ineligible for autologous HSCT or who have resistant/refractory disease and who may benefit from tandem autologous nonmyeloablative allogeneic transplant. * Patients who have received a prior allogeneic HSCT and who have either rejected their grafts or who have become tolerant of their grafts with no active GvHD requiring immunosuppressive therapy could be enrolled Exclusion Criteria: * Patients with suitably matched related or unrelated donors * Patients with conventional transplant options (a conventional transplant should be the priority for eligible patients ≤ 50 yr of age who have a related donor mismatched for a single HLA-A, -B or DRB1 antigen) * CNS involvement with disease refractory to intrathecal chemotherapy * Presence of active, serious infection (e.g., mucormycosis, uncontrolled aspergillosis, tuberculosis) * Karnofsky Performance Status \< 60% for adult patients (Appendix A) * Patients with the following organ dysfunction: * Left ventricular ejection fraction \<35% * DLCO \<35% and/or receiving supplemental continuous oxygen * Liver abnormalities: fulminant liver failure, cirrhosis of the liver with evidence of portal hypertension, alcoholic hepatitis, esophageal varices, hepatic encephalopathy, uncorrectable hepatic synthetic dysfunction as evidenced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal liver abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin \>3 mg/dL or symptomatic biliary disease. * HIV-positive patients * Women of childbearing potential who are pregnant (β-HCG+) or breast feeding * Fertile men and women unwilling to use contraceptives during and for 12 months post transplant * Life expectancy severely limited by diseases other than malignancy * Patients on any other investigational drug at time of enrolment
Where this trial is running
Milan
- European Institute of Oncology — Milan, Italy (RECRUITING)
Study contacts
- Principal investigator: Rocco Pastano, MD — European Institute of Oncology
- Study coordinator: Rocco Pastano, MD
- Email: rocco.pastano@ieo.it
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Hematologic Neoplasms, Hematopoietic Stem Cell Transplantation, High-Risk Hematologic Neoplasms, Haploidentical Donors, Cyclophosphamide