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AzTrials

Myeloid DiseasesPhase 1NCT03531736

T Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation Conditioned With a Reduced Intensity Regimen in Patients With Hematologic Malignancies and Aplastic Anemia

A Phase 1 study testing Antithymocyte globulin (Rabbit), fludarabine and total body irradiation in people with Myeloid Diseases, run by Memorial Sloan Kettering Cancer Center at 2 sites 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 main purpose of this study is to learn if a new combination of chemotherapy, in combination with low-dose radiation, will be safe for the patient, and at the same time provide the best opportunity to cure the bone marrow cancer. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myeloid Diseases
What is being tested?
Antithymocyte globulin (Rabbit), fludarabine and total body irradiation
Could I receive a placebo?
No, everyone receives an active treatment
How many people
17 participants
Where
2 sitesUnited States
Study dates
9 May 2018to May 2027
Phase
Phase 1
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients with one of the high risk myeloid diseases as outlined below. Patients must have ≤ 5% blasts on the last BM evaluation prior to starting the conditioning regimen. Diseases included on this protocol include:
  • Acute Myeloid Leukemia (AML) in CR1 with intermediate or high risk features as defined below:
  • °Cytogenetic abnormalities which are not considered "good risk" cytogenetic features (i.e t(8:21), t(15:17), inv 16 without c-kit mutations.
  • And/or
  • Therapy related AML with history of antineoplastic therapy (radiation and/or chemotherapy) And/or
  • Normal karyotype with mutations of FLT3, RUNX1, TP53 mutation, ASXL1 or any others that are considered to be high risk
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  • AML in ≥ 2nd remission
  • Myelodysplastic syndrome, myeloproliferative neoplasms, or MDS/MPN overlap syndrome with:
  • °International prognostic scoring system risk score INT-2 or high risk at the time of transplant evaluation.
  • And/or
  • Any risk category if life-threatening cytopenia exists And/or
  • 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.
  • Chronic myelomonocytic leukemia (CMML)
  • Chronic myeloid leukemia (CML) with the following features:
  • °Patients who have failed or are intolerant to BCR-ABL tyrosine kinase inhibitors.
  • And/or
  • °CML with BCR-ABL mutation consistent with poor response to tyrosine kinase inhibition (e.g T351l mutation)
  • Patients with severe aplastic anemia
  • Chronic lymphocytic leukemia (CLL) with high risk disease as defined by the EBMT consensus criteria.
  • Non-Hodgkin lymphoma meeting both of the following criteria:
  • Responding to therapy prior to enrollment.
  • Relapse after prior autologous bone marrow transplant or are ineligible for autologous bone marrow transplant.
  • Multiple Myeloma with disease in the following categories:
  • Patients with relapsed multiple myeloma following autologous stem cell transplantation who have achieved at least partial response following additional chemotherapy
  • Patients with high risk cytogenetics at diagnosis must have achieved at least a partial response following autologous stem cell transplantation. Patients must have complex karyotype, del17p, t4;14, and/or t14;16 by FISH and/or del13 by karyotyping.
  • Each patient must be willing to participate as a research participant and must sign an informed consent form.
  • Organ Function and Performance Status Criteria:
  • Patients be ≥ 18 years old.
  • Patients must have a Karnofsky (adult) or Performance Status ≥ 70%.
  • Patients must have adequate organ function measured by:
  • Cardiac: asymptomatic or if symptomatic, then LVEF at rest must be ≥ 40% and must improve with exercise.
  • Hepatic: < 5x ULN ALT and < 2x ULN total serum bilirubin, unless there is congenital benign hyperbilirubinemia.
  • Renal: CrCl >30ml/min (measured or calculated/estimated).
  • Pulmonary: asymptomatic or if symptomatic, DLCO > 50% of predicted (corrected for hemoglobin)

Who cannot join

  • Prior allogenic hematopoietic stem cell transplantation
  • Prior radiation therapy with 400cGY or more of TBI
  • BM with increased fibrosis (Reticulin stain > 1/3)
  • Active and uncontrolled infection at time of transplantation
  • HIV infection
  • Seropositivity for HTLV-1
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  • Inadequate performance status/ organ function
  • Pregnancy or breast feeding
  • Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up, and research tests.
  • Donor Inclusion and Exclusion Criteria:
  • Must be a 10/10 HLA genotypically match related or unrelated donor at all A, B, C, DRB1, and DQB1 loci, as tested by DNA analysis
  • Able to provide informed consent for the donation process per institutional standards
  • Meet standard criteria for donor collection as defined by the National Marrow Donor Program Guidelines

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • Memorial Sloan Kettering Monmouth (Limited Protocol Activities)

    Middletown, New Jersey, 07748

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

Other trials in United States

About this study

Official title: Allogeneic Hematopoietic Stem Cell Transplantation of α/β T-Lymphocyte Depleted Graft Conditioned With a Reduced Intensity Regimen in Patients With Hematologic Malignancies and Aplastic Anemia

The main purpose of this study is to learn if a new combination of chemotherapy, in combination with low-dose radiation, will be safe for the patient, and at the same time provide the best opportunity to cure the bone marrow cancer. The combination of chemotherapy and radiation described in the study is considered 'low intensity.' Although the chemotherapy agents used in this study and for transplant are FDA approved, the chemotherapy treatment and conditioning regimens or combinations listed in this consent are not yet FDA approved.

The CliniMACS device is FDA approved for one type of T cell depletion (positive selection of the stem cells) but not approved yet for other type of T cell depletion, which is being studied on this protocol. This pilot study, along with other studies will serve as the basis for FDA approval, if outcomes are favorable.

Being tested
Antithymocyte globulin (Rabbit), fludarabine, total body irradiation, cyclophosphamide, Rituxan
Keywords
Allogeneic Hematopoietic Stem Cell Transplantation, intensity conditioning regimen, 17-639

What participants receive

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

  • Antithymocyte globulin (Rabbit)Drug

    Also called ATG

    ATG (2 mg/kg/d IV on days-8 through -7)

  • fludarabineDrug

    fludarabine (30 mg/m2/d on days -5 through -2)

  • total body irradiationRadiation

    Also called TBI

    TBI 200 cGy (days -2 and -1) given post stem cell infusion

  • cyclophosphamideDrug

    cyclophosphamide given post stem cell infusion (50 mg/kg on days +3 and +4)

  • RituxanDrug

    Rituxan (200 mg/m2) will be given to reduce the risk of EBV viremia

  • Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery

    Allogeneic Hematopoietic Stem Cell Transplantation

Study groups in NCT03531736
Study groupRoleWhat they receive
Patients with Myeloid Malignancies & Aplastic AnemiaExperimentalTransplant conditioning will consist of: ATG (2 mg/kg/day IV on days-8 through-6), fludarabine (30 mg/m^2/d on days -5 through -2), TBI 400 cGy in 2 divided doses (days -2 and -1) and high dose cyclophosphamide given post stem cell infusion (50 mg/kg on days +3 and +4). One dose of Rituxan (200 mg/m^2) will be given to reduce the risk of EBV viremia. The donor stem cell product will be derived from the peripheral blood with a target cell infusion of ≥8X10^6 CD34 cells per recipient kg. Patients will receive post-transplant G-CSF starting on day +7. Patients will undergo donor/recipient bone marrow and peripheral chimerism studies at 30 and 100, and 6, 12, 18 and 24 months post allo HCT and thereafter, at the discretion of the treating clinician. Immune function and disease restaging will be performed at day 100 and 6, 12, 18, and 24 months and as otherwise clinically indicated by the treating physician.
Receives: Drug: Antithymocyte globulin (Rabbit); Drug: fludarabine; Radiation: total body irradiation; Drug: cyclophosphamide; Drug: Rituxan; Procedure: Allogeneic Hematopoietic Stem Cell Transplantation

What is being measured

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

  1. Rate of donor Neutrophil Engraftment

    Neutrophil engraftment (recovery of ANC) defined by an ANC ≥ 500/mm^3 for 3 consecutive days

    Measured: 30 days post-transplant

Timeline

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

  1. First posted22 May 2018
  2. Study start9 May 2018
  3. Primary completionMay 2027 (estimated)
  4. Study completionMay 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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
This is a pilot study to assess engraftment of a T cell depleted (TCD) graft following a reduced intensity conditioning regimen (RIC). The conditioning regimen will include total body irradiation (TBI), Fludarabine, anti-thymocyte globulin (ATG) and post transplant cyclophosphamide (PT-Cy). The graft will be TCD and will be composed of a TCR-α/β+ lymphocyte depletion stem cells and CD34+ selected stem cells.
Masking
None (open label)
Enrolment
17 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03531736
Sponsor's protocol ID
17-639
Status last verified
Jul 2026
First submitted
9 May 2018
Last updated
24 Jul 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Antithymocyte globulin (Rabbit), fludarabine and total body irradiation in people with Myeloid Diseases, run by Memorial Sloan Kettering Cancer Center at 2 sites 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 13 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 24 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Rate of donor Neutrophil Engraftment (time frame: 30 days post-transplant). 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 NCT03531736.

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 24 Jul 2026.

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