Clinical trial · Interventional
Microtransplantation to Treat Refractory or Relapsed Hematologic Malignancies in Younger Patients
A Phase II Study of Microtransplantation in Patients With Refractory or Relapsed Hematologic Malignancies
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): The study was closed due to poor accrual and because of competing protocols.
Summary
Brief summary (as posted)
Allogeneic transplant can sometimes be an effective treatment for leukemia. In a traditional allogeneic transplant, patients receive very high doses of chemotherapy and/or radiation therapy, followed by an infusion of their donor's bone marrow or blood stem cells. The high-dose chemotherapy drugs and radiation are given to remove the leukemia cells in the body. The infusion of the donor's bone marrow or blood stem cells is given to replace the diseased bone marrow destroyed by the chemotherapy and/or radiation therapy. However, there are risks associated with allogeneic transplant. Many people have life-threatening or even fatal complications, like severe infections and a condition called graft-versus-host disease, which is caused when cells from the donor attack the normal tissue of the transplant patient. Recently, several hospitals around the world have been using a different type of allogeneic transplant called a microtransplant. In this type of transplant, the donor is usually a family member who is not an exact match. In a microtransplant, leukemia patients get lower doses of chemotherapy than are used in traditional allogeneic transplants. The chemotherapy is followed by an infusion of their donor's peripheral blood stem cells. The objective of the microtransplant is to suppress the bone marrow by giving just enough chemotherapy to allow the donor cells to temporarily engraft (implant), but only at very low levels. The hope is that the donor cells will cause the body to mount an immunologic attack against the leukemia, generating a response called the "graft-versus-leukemia" effect or "graft-versus-cancer" effect, without causing the potentially serious complication of graft-versus-host disease. With this research study, the investigators hope to find out whether or not microtransplantation will be a safe and effective treatment for children, adolescents and young adults with relapsed or refractory hematologic malignancies
Conditions
Conditions (2)
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 Myeloid Leukemia (AML) | Acute Myeloid Leukemia | CURATED_BROADER | 0.80 |
| Myelodysplastic Syndrome (MDS) | Myelodysplastic Syndrome | CURATED_BROADER | 0.80 |
Interventions
Interventions (3)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Cytarabine | Drug | Cytarabine | ALIAS |
| HPC-A | Biological | — | UNRESOLVED |
| Intrathecal Triples | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Myeloid Malignancies
- description
- Includes participants with AML and MDS. Participants receive chemotherapy based on diagnosis followed by infusion of donor HPC-A. Participants with CNS disease receive weekly age-adjusted intrathecal triples therapy until the cerebrospinal fluid becomes free of leukemia (minimum of 4 doses). Interventions: * Cycle 1: cytarabine, HPC-A donor infusion * Cycle 2: Participants who have at least a partial response to Cycle 1 are eligible to receive Cycle 2: cytarabine, HPC-A infusion
- interventionNames
- Drug: Cytarabine
- Drug: Intrathecal Triples
- Biological: HPC-A
Primary outcomes (2)
- measure
- Number of Participants by Stratum Who Complete 2 Cycles of Therapy
- timeFrame
- At the end of therapy cycle 2 (approximately 2-3 months)
- description
- If two or more patients die from causes other than leukemia progression or experience ≥ Grade 3 GVHD that is associated with detectable donor chimerism due to this protocol, or demonstrate persistent engraftment defined as \>5% donor chimerism at the time of count recovery (ANC \> 0.3 x 10\^9/L and platelet count \> 30 x 10\^/L), then the cohort will close due to intolerability. Any subject who transfers to transplant prior to completion of two courses without experiencing an unacceptable toxicity is considered inevaluable for purposes of evaluating tolerability. Accrual will be halted for intolerability if there are two or more failures in tolerability among the first six subjects who are evaluable for tolerability.
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 21 Years
Show eligibility criteria text
INCLUSION CRITERIA - AML and MDS PARTICIPANTS * Participants must have a diagnosis of AML or myelodysplastic syndrome (MDS), ALL, and must have disease that has relapsed or is refractory to chemotherapy, or that has relapsed after HSCT. * Refractory disease is defined as persistent disease after at least two courses of induction chemotherapy. * Patients with AML must have ≥ 5% leukemic blasts in the bone marrow or have converted from negative minimal residual disease (MRD) status to positive MRD status in the bone marrow as assessed by flow cytometry. If an adequate bone marrow sample cannot be obtained, patients may be enrolled if there is unequivocal evidence of leukemia in the peripheral blood. * Participant is ≤ 21 years of age (i.e., has not reached 22nd birthday). * Adequate organ function defined as the following: * Total bilirubin ≤ upper limit of normal (ULN) for age, or if total bilirubin is \> ULN, direct bilirubin is ≤ 1.5 mg/dL * AST (SGOT)/ALT (SGPT) \< 5 x ULN * Calculated creatinine clearance \> 50 ml/min/1.73m\^2 as calculated by the Schwartz formula for estimated glomerular filtration rate \> * Left ventricular ejection fraction ≥ 40% or shortening fraction ≥ 25%. * Has an available HPC-A donor. * Performance status: Lansky ≥ 50 for patients who are ≤ 16 years old and Karnofsky ≥ 50% for patients who are \> 16 years old. * Does not have an uncontrolled infection requiring parenteral antibiotics, antivirals, or antifungals within one week prior to first dose. Infections controlled on concurrent anti-microbial agents are acceptable, and anti-microbial prophylaxis per institutional guidelines is acceptable. * Patient has fully recovered from the acute effects of all prior therapy and must meet the following criteria. * At least 14 days must have elapsed since the completion of myelosuppressive therapy. * At least 24 hours must have elapsed since the completion of hydroxyurea, low-dose cytarabine (up to 200 mg/m\^2/day), and intrathecal chemotherapy. * At least 30 days must have elapsed since the use of investigational agents. * For patients who have received prior HSCT, there can be no evidence of GVHD and greater than 60 days must have elapsed since the HSCT. Patients cannot be receiving therapy, including steroids, for GVHD. * Post-menarchal female has had negative serum pregnancy test within 7 days prior to enrollment. * Male or female of reproductive potential has agreed to use effective contraception for the duration of study participation. * Not breastfeeding INCLUSION CRITERIA - HPC-A CELL DONOR * At least 18 years of age. * Family member (first degree relatives). * Not pregnant as confirmed by negative serum or urine pregnancy test within 7 days prior to enrollment (if female). * Not breast feeding. * Meets donation eligibility requirements as outlined by 21 CFR 1271.
References
Publications (0)
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