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Acute Myeloid Leukemia (AML)Phase 2NCT04872595

A Modified Dose of Rabbit Anti-thymocyte Globulin (rATG) in Children and Adults Receiving Treatment to Help Prepare Their Bodies for a Bone Marrow Transplant

A Phase 2 study testing Personalized rATG (P-rATG), Hyper fractionated total body irradiation and Thiotepa in people with Acute Myeloid Leukemia (AML) and Acute Lymphoid Leukemia (ALL), run by Memorial Sloan Kettering Cancer Center at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The purpose of this study is to see if conditioning regimens that include personalized rabbit ATG (P-rATG) help the immune system recover sooner and decrease the chances of transplant-related side effects. Read more about this study

Who is it for?
Ages 4 years and older
What is being tested?
Personalized rATG (P-rATG), Hyper fractionated total body irradiation and Thiotepa
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
59 participants
Where
1 siteUnited States
Study dates
30 Apr 2021to Apr 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 4 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Patients receiving first peripheral blood mobilized ex-vivo CD34-selected T cell depleted allo-HCT for the following hematologic malignant conditions:
  • Acute myeloid leukemia (AML) with intermediate or high-risk features in CR1 or Relapse AML in ≥ CR2.
  • Must have MRD <5% (flow cytometry, molecular and/or cytogenetics accepted).
  • Acute leukemias of ambiguous lineage in ≥ CR1.
  • Must have MRD <5% (flow cytometry, molecular and/or cytogenetics accepted).
  • Acute lymphoid leukemia (ALL) in CR1 with clinical, flow cytometric, or molecular features indicating a high risk for relapse, or ALL in ≥ CR2.
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  • Adult Patients - recommended but not required to be MRDnegative (by flow cytometry, molecular and/or cytogenetics).
  • Pediatric Patients - Must be MRD-negative by flow cytometry, molecular and/or cytogenetics.
  • Myelodysplastic syndromes (MDS) with least one of the following:
  • Revised International Prognostic Scoring System risk score of intermediate or higher at the time of transplant evaluation.
  • Life-threatening cytopenia.
  • Karyotype or genomic changes that indicate high risk for progression to acute myelogenous leukemia, including abnormalities of chromosome 7 or 3, mutations of TP53, or complex or monosomal karyotype.
  • Therapy related disease or disease evolving from other malignant processes.
  • Able to tolerate cytoreduction
  • Patients age:
  • Regimen A: 4 - 60 years
  • Regimen B - no age restriction
  • Adequate organ function is required, defined as follows:
  • Hepatic: Serum bilirubin ≤ 2 mg/dL, unless benign congenital hyperbilirubinemia. Patients with hyperbilirubinemia related to paroxysmal nocturnal hemoglobinuria or other hemolytic disorders are eligible with PI approval.
  • Hepatic: AST, ALT, and alkaline phosphatase < 2.5 times the upper limit of normal unless thought to be disease-related.
  • Renal: serum creatinine <1.5x normal for age. If serum creatinine is outside the normal range, then CrCl > 50 mL/min/1.73m2 (calculated or estimated) or GFR (mL/min/1.72m2) >30% of predicted normal for age.
  • Normal GFR by Age
  • 1 week 40.6 + / - 14.8
  • 2 - 8 weeks 65.8 + / - 24.8
  • °> 8 weeks 95.7 +/- 21.7
  • 2 - 12 years 133 +/- 27
  • 13 - 21 years (males) 140 +/- 30
  • 13 - 21 years (females) 126.0 + / - 22.0
  • Cardiac: LVEF ≥ 50% by MUGA or resting echocardiogram.
  • Pulmonary: Pulmonary function testing (FEV1 and corrected DLCO) ≥ 50% predicted (pediatric patients unable to complete PFTs will need oxygen saturation as recorded by pulse oximetry of ≥92% on room air).
  • Adequate performance status:
  • Age ≥ 16 years: ECOG ≤ 1 or Karnofsky 70%
  • Age < 16 years: Lansky 70%
  • Each patient must be willing to participate as a research subject and must sign an informed consent form or legal guardian with assent as appropriate.

Who cannot join

  • Patients with active extramedullary disease.
  • Patients with active central nervous system malignancy.
  • Uncontrolled infection at the time of allo-HCT.
  • Patients who have undergone previous allo-HCT.
  • Patient seropositivity for HIV I/II and/or HTLV I/II.
  • Females who are pregnant or breastfeeding.
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  • Patients unwilling to use contraception during the study period.
  • Patient or parent or guardian unable to give informed consent or unable to comply with the treatment protocol including research tests.
  • Donor Inclusion Criteria:
  • Related or Unrelated Donors:
  • °8/8 HLA matched at A, B, C, and DRB1 loci, as tested by DNA analysis.
  • Able to provide informed consent for the donation process per institutional standards.
  • Meet standard criteria for donor collection (e.g. National Marrow Donor Program Guidelines or collecting center guidelines as approved by treating physician).
  • Provide GSCF mobilized peripheral blood stem cells

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

Other trials in United States

About this study

Official title: Phase 2 Study of Personalized r-ATG Dosing to Improve Survival Through Enhanced Immune Reconstitution in Pediatric and Adult Patients Undergoing Ex-vivo CD34-Selected Allogeneic-HCT (PRAISE-IR)

The purpose of this study is to see if conditioning regimens that include personalized rabbit ATG (P-rATG) help the immune system recover sooner and decrease the chances of transplant-related side effects. Participants in this study will be children and adults who have acute leukemia or myelodysplastic syndrome (MDS), and will receive a standard conditioning regimen to prepare the body for an allogeneic hematopoietic cell transplant (allo-HCT). The conditioning regimen will include r-ATG, one of two combinations of chemotherapy, and possibly total body irradiation (TBI).

Being tested
Personalized rATG (P-rATG), Hyper fractionated total body irradiation, Thiotepa, Cyclophosphamide, GCSF
Keywords
personalized rabbit ATG (P-rATG), ex-vivo CD34+ T cell depleted, total body irradiation, thiotepa, cyclophosphamide, busulfan, melphalan, fludarabine, 21-193
Medical subject headings
Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Personalized rATG (P-rATG)Other

    P-rATG days (always starting on Day -12 to -10)

  • Hyper fractionated total body irradiationRadiation

    (1375 - 1500cGy*) Day -9 to -6

    *TBI dose in 125cGy fractions (with lung shielding) and total dose to be determined by treating physician/radiation oncology and is based off age, stage of disease, and anesthesia requirements.

  • ThiotepaDrug

    (5mg/kg/day x 2 day) Day -5 to -4

  • CyclophosphamideDrug

    (60mg/kg/day x 2 days) Day -3 to -2

  • GCSFDrug

    Day +7

  • BusulfanDrug

    Day -9 to -7 doses 2-3 to be adjusted per PK for target cumulative exposure of 65 mg*h/L

  • MelphalanDrug

    (70mg/m2/day x 2 days) Day -6 to -5

  • FludarabineDrug

    (25mg/m2/day x 5 days) Day -6 to -2

Study groups in NCT04872595
Study groupRoleWhat they receive
P-rATG with total body irradiation, thiotepa, cyclophosphamideExperimentalP-rATG days (always starting on Day -12 to -10) * Hyper fractionated total body irradiation (1375 - 1500cGy*) Day -9 to -6 * Thiotepa (5mg/kg/day x 2 day) Day -5 to -4 * Cyclophosphamide (60mg/kg/day x 2 days) Day -3 to -2 * GCSF Day +7 *TBI dose in 125cGy fractions (with lung shielding) and total dose to be determined by treating physician/radiation oncology and is based off age, stage of disease, and anesthesia requirements.
Receives: Other: Personalized rATG (P-rATG); Radiation: Hyper fractionated total body irradiation; Drug: Thiotepa; Drug: Cyclophosphamide; Drug: GCSF
P-rATG with busulfan, melphalan and fludarabineExperimentalP-rATG days (Appendix A - always starting on Day -12 to -10) * Busulfan -Day -9 to -7 * Initial dose per table in Appendix B; doses 2-3 to be adjusted per PK for target cumulative exposure of 65 mg*h/L Melphalan (70mg/m2/day x 2 days) Day -6 to -5 * Fludarabine (25mg/m2/day x 5 days) Day -6 to -2 * GCSF Day +7
Receives: Other: Personalized rATG (P-rATG); Drug: GCSF; Drug: Busulfan; Drug: Melphalan; Drug: Fludarabine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. proportion of patients who achieve CD4+IR

    is defined at CD4+ > 50u/L at two consecutive measures within 100 days post allo-HCT.

    Measured: within 100 days of HCT

Secondary outcomes (1)

  1. Overall Survival (OS)

    The duration of time between HCT and death due to any cause.

    Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 May 2021
  2. Study start30 Apr 2021
  3. Primary completionApr 2027 (estimated)
  4. Study completionApr 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Model description
This phase 2 study is to assess the effects of personalized rabbit ATG (P-rATG) dosing on CD4+ immune reconstitution (CD4+IR) based on a pharmacokinetic/pharmacodynamic (PK/PD) model in patients with hematologic malignancies undergoing peripheral blood mobilized, ex-vivo CD34+ T cell depleted, allogeneic, hematopoietic cell transplantation (CD34+/TCD allo-HCT)1.
Masking
None (open label)
Enrolment
59 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04872595
Sponsor's protocol ID
21-193
Status last verified
May 2026
First submitted
30 Apr 2021
Last updated
6 May 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made following one year after publication and for up to 36 months later. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

Publications

  • Scordo M, Perales MA, Mauguen A, Lin A, Kunvarjee B, Paes Pena M, Mcavoy D, Nguyen LK, Hogan M, Chapman N, Bieler J, Cho C, Gyurkocza B, Harris AC, Spitzer B, O'Reilly RJ, Jakubowski AA, Lin RJ, Papadopoulos EB, Politikos I, Ponce DM, Shaffer BC, Shah GL, Tamari R, Giralt SA, Boelens JJ, Curran KJ. Model-based antithymocyte globulin dosing in ex vivo CD34+ selected allogeneic haematopoietic cell transplantation: a single-centre, single-arm, phase 2 study. Lancet Haematol. 2025 Dec;12(12):e956-e965. doi: 10.1016/S2352-3026(25)00293-5.

    PubMed 41338864

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Personalized rATG (P-rATG), Hyper fractionated total body irradiation and Thiotepa in people with Acute Myeloid Leukemia (AML) and Acute Lymphoid Leukemia (ALL), run by Memorial Sloan Kettering Cancer Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 34 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 6 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

proportion of patients who achieve CD4+IR (time frame: within 100 days of HCT). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04872595, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 May 2026.

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