A Phase 1/2 interventional study of ATG 5.0 and Cyclophosphamide injection in Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndromes, sponsored by Instituto Nacional de Cancer, Brazil. Recruiting at 1 site in Brazil. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2025-02-25.
Sponsored by Instituto Nacional de Cancer, Brazil · Phase 1/2, Interventional, and Prevention
Hematopoietic stem cell transplantation is a curative treatment for a number of benign and malignant hematologic diseases. One of the key parts of hematopoietic stem cell transplantation is the prophylaxis of graft-versus-host disease. Since the end of the 1970s, with the introduction of cyclosporine, calcineurin inhibitors (cyclosporine and tacrolimus) have become part of almost all prophylactic regimens, even though they are a group of drugs with a poor toxicity profile that requires monitoring. constant serum level. Since 2008, post-transplant cyclophosphamide has been introduced with great success, associated with a calcineurin inhibitor and mycophenolate, in the prophylaxis of graft-versus-host disease in haploidentical transplantation (50% matched).
Since then, in view of this enormous success, efforts have been made to incorporate post-transplant cyclophosphamide in matched related and unrelated transplants, or with a mismatch.
This is a prospective, 2-arm, non-randomized study. Arm 1, with related donors, and arm 2, with unrelated donors. Patients will be allocated in these arms according to donor availability (patients with a matched-sibling donor will receive a matched-sibling transplant; patients with no related donors but with unrelated donors, an unrelated transplant).
Patients who are ready for transplantation with matched-sibling or unrelated donors will be recruited to participate in the study.
The stem cell collection target will be 5E6 CD34/kg recipient weight for peripheral source. If a quantity greater than this is collected, the remainder will be cryopreserved according to the institutional protocol.
Graft-versus-host disease prophylaxis will be performed on D+3 and D+4 with cyclophosphamide and with ATG on D-3 and D-2 for matched-sibling or unrelated donors transplants.
3,667 studies on the registry are indexed under Leukemia, Myeloid, Acute; 816 are open to participants now.
This study's planned enrollment of 50 is above the median of 40 across 3,005 interventional studies indexed under Leukemia, Myeloid, Acute.
Browse Leukemia, Myeloid, Acute studies →Instituto Nacional de Cancer, Brazil is the lead sponsor of 41 studies on the registry; 7 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Matched sibling transplants will receive PTCy + ATG4.0
Drug: Cyclophosphamide injection · Drug: ATG 4.0
Unrelated transplants will receive PTCy + ATG5.0
Drug: ATG 5.0 · Drug: Cyclophosphamide injection
ATG 2.5 mg/kg on days -3 and -2
Cyclophosphamide 50 mg/kg on days +3 and +4
ATG 2.5 mg/kg on day -2 + 1.5 mg/kg on day -3
Cumulative incidence of grades III-IV acute GVHD by the MAGIC criteria
Cumulative incidence of acute graft-versus-host disease, grades III-IV by the MAGIC criteria
Time frame: 6 months
Cumulative incidence of grades II-IV acute GVHD by the MAGIC criteria
Cumulative incidence of acute graft-versus-host disease, grades II-IV by the MAGIC criteria
Time frame: 6 months
Cumulative incidence of steroid-refractory acute GVHD as defined by Mohty et al PMID 32756949
Cumulative incidence of steroid-refractory graft-versus-host disease
Time frame: 6 months
Cumulative incidence of chronic GVHD as defined by the NIH criteria
Cumulative incidence of chronic graft-versus-host disease
Time frame: 3 years
Cumulative incidence of steroid-requiring chronic GVHD as defined by the NIH criteria
Cumulative incidence of steroid-requiring chronic graft versus host disease
Time frame: 3 years
Cumulative incidence of non-relapse mortality, i.e., death not following disease relapse
Cumulative incidence of death not caused by primary malignant disease or following relapse
Time frame: 3 years
Cumulative incidence of relapse, defined as > 5% blasts in bone marrow or 1% blasts in peripheral blood (acute leukemias/myelodysplasia) or biopsy proven relapse or positve PET-CT (lymphoma)
Cumulative incidence of relapse of primary malignant disease
Time frame: 3 years
Rate of overall survival
Death by any cause
Time frame: 3 years
Rate of disease-free survival (death or relapse)
Composite outcome of death or primary disease relapse
Time frame: 3 years
Cumulative incidence of clinically significant CMV reactivation (which led to antiviral treatment)
Incidence of cytomegalovirus reactivation
Time frame: 3 year
Cumulative incidence of posttransplant lymphoproliferative disorder (biopsy-proven or positive EBV PCR combined with clinical symptoms)
Incidence of posttransplant lymphoproliferative disorder
Time frame: 3 years
Cumulative incidence CMV disease (biopsy-proven CMV disease OR suggestive CMV+ BAL)
Cumulative incidence of cytomegalovirus disease
Time frame: 3 years
Measuremnt of quality of life using the FACT-BMT scale
Measurement quality of life
Time frame: 2 years
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Upon finalization of the study, data will be shared on reasonable requests.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report, Analytic code
Started Jun 2024 (actual). Primary completion is estimated for Jun 2031, 83 months after the start.
Of 134 non-industry phase 1/2 interventional studies under Leukemia, Myeloid, Acute, half reached primary completion within 76 months of starting (the median), counting those still running; a quarter within 49 and three quarters within 110. This study's planned 83 months is close to that.
Counting every sponsor, half of 226 studies reached it within 73 months; a quarter within 48 and three quarters within 106.
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Instituto Nacional de Cancer, Brazil