Clinical trial · Interventional
Azacitidine in Haploidentical Donor Hematopoietic Cell Transplantation
A Phase I/II Study of Azacitidine in Haploidentical Donor Hematopoietic Cell Transplantation
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): Toxicity. Only enrolled patients in phase I portion of trial.
Summary
Brief summary (as posted)
Allogeneic hematopoietic cell transplantation (allo-HCT) is a potentially curative therapy for patients with hematologic malignancies including acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and acute lymphoblastic leukemia (ALL); however, human leukocyte antigen (HLA)-matched donor availability continues to be a major hurdle. Historically, HLA haploidentical donor hematopoietic cell transplantation (haplo-HCT) was associated with high incidences of graft rejection and excessive non-relapse mortality (NRM), but recent advances utilizing post-transplant cyclophosphamide (PT-Cy) have revolutionized haplo-HCT and the outcomes are now comparable to allo-HCT using more traditional HLA matched related and unrelated donors. However, graft-versus-host disease (GvHD) continues to be a problem and is associated with significant morbidity and mortality in allo-HCT patients including those who receive haplo-HCT on PT-Cy platform. The aim of this early phase study is to investigate the safety and overall efficacy of azacitidine in reducing the incidence and severity of GvHD when added to PT-Cy based haplo-HCT platform for patients with AML, ALL, or advanced MDS.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Acute Lymphocytic Leukemia | Acute Lymphoblastic Leukemia | ALIAS | 0.90 |
| Acute Myeloid Leukemia | Acute Myeloid Leukemia | CURATED_BROADER | 0.80 |
| Myelodysplastic Syndrome | Myelodysplastic Syndrome | CURATED_BROADER | 0.80 |
Interventions
Interventions (9)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Azacitidine | Drug | Azacitidine | ALIAS |
| Busulfan | Drug | Busulfan | ALIAS |
| Cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| Fludarabine | Drug | Fludarabine | ALIAS |
| Fractionated total body irradiation | Radiation | — | UNRESOLVED |
| Granulocyte-colony stimulating factor | Drug | — | UNRESOLVED |
| Melphalan | Drug | Melphalan | ALIAS |
| Single dose total body irradiation | Radiation | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Arm 1: Azacitidine
- description
- * Treating physician must choose from one of these conditioning regimens (will be given per standard of care) * fludarabine and fractionated total body irradiation (Flu/FrTBI) * fludarabine and busulfan (Flu/Bu4) * fludarabine, cyclophosphamide, and single dose total body irradiation (Flu/Cy/sdTBI) * fludarabine and melphalan (Flu/Mel) * reduced-intensity fludarabine and busulfan (Flu/Bu2) * G-CSF from Day -5 through Day -1 per standard of care * On Day 0, the allograft will be infused per standard of care. * Azacitidine will be administered on Day +1 and +2 post-stem cell transfusion days * Cyclophosphamide on Days +3 and +4 post-transplant
- interventionNames
- Drug: Fludarabine
- Radiation: Fractionated total body irradiation
- Drug: Busulfan
- Drug: Cyclophosphamide
- Radiation: Single dose total body irradiation
- Drug: Melphalan
- Drug: Granulocyte-colony stimulating factor
- Procedure: Stem cell transplant
- Drug: Azacitidine
Primary outcomes (3)
- measure
- Safety of azacitidine (Phase I only) as measured by frequency and grade of adverse events
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Diagnosis of acute leukemia (AML/ALL) or advanced MDS (INT-2 or high risk) in complete remission (CR/CRc/CRi) documented by bone marrow biopsy done within 30 days prior to the initiation of conditioning regimen. * Available HLA-haploidentical donor that meets the following criteria: * Immediate family member (sibling, offspring, or parent) * At least 18 years of age * HLA-haploidentical donor/recipient match by class I serologic typing at the A\&B locus. * In the treating physician's opinion, is in general good health, and medically able to tolerate leukapheresis required for harvesting HSC * No active hepatitis (B, C), HTLV, and HIV infections * Not pregnant * Karnofsky performance status ≥ 70 % * Adequate organ function as defined below: * Total bilirubin ≤ 2.5 mg/dl (unless the patient has a history of Gilbert's syndrome) * AST(SGOT) and ALT(SGPT) ≤ 3.0 x IULN * Creatinine ≤ 2.0 x IULN OR estimated creatinine clearance ≥ 30 mL/min/1.73 m\^2 by Cockcroft-Gault Formula * Oxygen saturation ≥ 90% on room air * LVEF ≥ 40% * FEV1 and FVC ≥ 50% predicted, corrected DLCO ≥ 40% predicted * At least 18 years of age at the time of study registration * Able to understand and willing to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable) Exclusion Criteria: * Recipients with donor sensitive antibodies (DSA), defined by 2000 or higher MFI against one or more class I or II antigens * Known HIV or active Hepatitis B or C infection * Underwent a previous related or unrelated allogeneic transplant * Known hypersensitivity to one or more of the study agents * Currently receiving or has received any investigational drugs within the 14 days prior to the first dose of the conditioning regimen. * Pregnant and/or breastfeeding * Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or unstable cardiac arrhythmias. * Presence of a readily available 6/6 matched sibling donor who is a candidate for donation
References
Publications (0)
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