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Blood CancerPhase 2NCT05873205

Open-Label, Phase II Trial of Isatuximab for Patients With Refractory Immune Cytopenias After Allogeneic Hematopoietic Cell Transplantation

A Phase 2 study testing Isatuximab in people with Blood Cancer and Refractory Immune Cytopenias, run by Memorial Sloan Kettering Cancer Center at 7 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 purpose of this study is to find out whether isatuximab is an effective treatment for people who developed immune cytopenias/ICs after allogeneic hematopoietic cell transplant/allo-HCT. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Blood Cancer, Refractory Immune Cytopenias
What is being tested?
Isatuximab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
7 sitesUnited States
Study dates
21 Jul 2023to 29 Jun 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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age ≥ 18 years (There are no dosing/AE data for isatuximab in children).
  • Disease for which patient underwent an allo-HCT is in documented remission.
  • Patients must have previously had documented primary neutrophil and platelet engraftment, defined as:
  • Neutrophil engraftment: the first of 3 successive days with an absolute neutrophil count of ≥500/μL after post-transplantation nadir.
  • Platelet engraftment: the first of 3 consecutive days with a platelet count of 20,000/μL or higher in the absence of platelet transfusion for 7 consecutive days.
  • Patients must be at least 45 days post allogeneic HCT to enroll.
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  • Patients must be diagnosed with IC(s) based on the following criteria:
  • o For AIHA: Positive (abnormal) DAT test and decreasing hemoglobin of ≥2 g/dL from a stable baseline (i.e., from the patients typical hemoglobin value prior to AIHA) and at least grade 2 (i.e., hemoglobin <10 g/dL) due to evidence of hemolytic anemia with ≥2 of the following tests: increased reticulocyte count (>ULN), increased lactate dehydrogenase (LDH) (>ULN), decreased haptoglobin (<LLN), increased unconjugated bilirubin (>ULN).
  • DAT negative AIHA may be included providing exclusion of alternative etiology of hemolytic anemia.
  • For ITP: decreasing thrombocytopenia from baseline (i.e., from the patients typical platelet count prior to ITP) and platelet count ≤30 K/µL or requiring platelet transfusions in the absence of other causes of thrombocytopenia (including drug-induced thrombocytopenia), and with normal or increased bone marrow megakaryocytes.
  • For PRCA: severe anemia (hemoglobin <8 g/dL without transfusions) with reticulocytopenia (reticulocyte percentage <1% and/or absolute reticulocyte count <10,000/µL) after exclusion of obvious causes of anemia.
  • Patients with concomitant ICs can be enrolled on the study.
  • Patient must have responded incompletely to their previous treatment, defined as:
  • Corticosteroid refractoriness: defined as a clear progression or minimal responsiveness of IC(s) after ≥7 days of treatment with prednisone equivalent of ≥1 mg/kg/day.
  • Corticosteroid dependence: defined as dependence on prednisone equivalent of ≥0.5 mg/kg/day to maintain hemoglobin level ≥2 g/dL nadir level (for AIHA and/or PRCA), and/or platelet count ≥30 x 109/L or ≥2-fold increase from nadir level (for ITP).
  • Refractory IC(s) after ≥2 treatment lines including corticosteroids (≥0.5 mg/kg/day prednisone equivalent), IVIG (400 mg/kg/day for 2 to 5 days), and/or rituximab, etc.
  • For rituximab treated patients, refractoriness will be defined as no or minimal response within 2 weeks of completing ≥4 doses of rituximab.
  • Absolute neutrophil count (ANC) ≥ 1.0 x 109/L.
  • o Growth factors, including granulocyte colony stimulating factors and erythropoietin are allowed, but should be administered at a stable dose.
  • No active hepatitis viral infection or on active treatment for hepatitis infection.
  • Female patients of childbearing potential are eligible if the patient has had a negative serum or urine pregnancy test within 10-14 days prior to starting isatuximab therapy.
  • They must also agree to avoid pregnancy by using an adequate method of contraception (2 barrier method or 1 barrier method with a spermicide or intrauterine device) for 2 weeks prior to screening, during and 5 months after the last dose of trial medication. Adequate methods of contraception are provided as examples. Other acceptable and effective methods of birth control are also permitted (e.g., abstinence).
  • Male patients must agree to not donate sperm while on the study and for at least 5 months after the last dose of study drug. They must agree to use contraception during the intervention period and for at least 5 months after the last dose of isatuximab treatment.
  • Subjects must be able to give informed consent.

Who cannot join

  • Presence of relapse/progression of malignant disease for which the patient underwent allo-HCT
  • Patients with anemia and/or thrombocytopenia related to transplant-associated thrombotic microangiopathy.
  • Patients with active GVHD requiring therapy may be eligible if the GVHD is responsive to treatment (< grade 4 in severity), and after agreement between the sponsor and principal investigator.
  • Organ insufficiency based on above criteria.
  • Pregnancy or unwillingness to agree to birth control as noted above.
  • Known to be HIV+ or to have active hepatitis A, B, or C infection (i.e., with viremia).
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  • Of note:
  • Patients can be eligible if anti-HBc seropositive (with or without positive anti-HBs), but HBsAg and HBV DNA are negative.
  • Patients with antiviral therapy for HCV started before initiation of treatment and positive Hep C antibodies are eligible. The antiviral therapy for Hep C should continue throughout the treatment period until seroconversion. Patients with positive anti-Hep C and undetectable Hep C RNA without antitviral therapy for Hep C are eligible.
  • Any clinically significant, uncontrolled medical condition(s), including infection(s) that, in the Investigator's opinion, would expose the patient to excessive risk or may interfere with compliance or interpretation of the study results.
  • Hypersensitivity or history of intolerance to steroids, mannitol, pregelatinized starch, sodium stearyl fumarate, histidine (as base and hydrochloride salt), arginine hydrochloride, poloxamer 188, sucrose, prior anti-CD38 moAb such as daratumumab, or any of the other components of study intervention that are not amenable to premedication with steroids and H2 blockers or would prohibit further treatment with these agents.
  • Received any investigational drug within 14 days or 5 half-lives of the investigational drug prior to initiation of study intervention, whichever is longer. In case of very aggressive disease (e.g., acute leukemia) delay could be shortened after agreement between sponsor and principal investigator, in absence of residual toxicities from previous therapy.
  • Patients on post-HCT maintenance therapy to reduce the risk of relapse (for patients with hematologic malignancies) or GVHD (e.g., FLT3 inhibitors, etc.) may be eligible after agreement between the sponsor and principal investigator.
  • Contraindication to any concomitant medication, including pre-medications or hydration given prior to therapy
  • Participants who are unable to consent to the study or comply with the study procedures.

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

United States7 sites

  • Memorial Sloan Kettering Basking Ridge (Limited Protocol Activities)

    Basking Ridge, New Jersey, 07920

  • Memorial Sloan Kettering Suffolk-Commack (All Protocol Activities )

    Commack, New York, 11725

  • Memorial Sloan Kettering Westchester (Limited Protocol Activities)

    Harrison, New York, 10604

  • Memorial Sloan Kettering Monmouth (Limited Protocol Activities)

    Middletown, New Jersey, 07748

  • Memorial Sloan Kettering Bergen (Limited protocol activities)

    Montvale, New Jersey, 07645

  • Memorial Sloan Kettering Cancer Center (All Protocol Activities)

    New York, New York, 10065

  • Memorial Sloan Kettering Nassau (All protocol activities)

    Rockville Centre, New York, 11553

Other trials in United States

About this study

The purpose of this study is to find out whether isatuximab is an effective treatment for people who developed immune cytopenias/ICs after allogeneic hematopoietic cell transplant/allo-HCT.

Being tested
Isatuximab
Keywords
Refractory Immune Cytopenias, Blood Cancer, Isatuximab, Allogeneic Hematopoietic Cell Transplantation, 23-119, Memorial Sloan Kettering
Medical subject headings
Hematologic Neoplasms

What participants receive

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

  • IsatuximabBiological

    All participants enrolled on the study will receive isatuximab intravenously as a single agent

Study groups in NCT05873205
Study groupRoleWhat they receive
Participants with Refractory Immune CytopeniasExperimentalParticipants will be adults who develop Immune Cytopenias/ICs after Allogeneic Hematopoietic Cell Transplantation/allo-HCT and who did not respond to initial immunosuppressive therapy.
Receives: Biological: Isatuximab

What is being measured

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

  1. Overall response rate

    To estimate the overall response rate (ORR; complete response [CR] + response)

    Measured: Up to 4 years

Timeline

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

  1. First posted24 May 2023
  2. Study start21 Jul 2023
  3. Primary completion29 Jun 2027 (estimated)
  4. Study completion29 Jun 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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05873205
Sponsor's protocol ID
23-119
Status last verified
Apr 2026
First submitted
15 May 2023
Last updated
2 Apr 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 beginning 12 months after publication and for up to 36 months post publication. 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.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Isatuximab in people with Blood Cancer and Refractory Immune Cytopenias, run by Memorial Sloan Kettering Cancer Center at 7 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 24 inclusion and 15 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 2 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 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 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?

Overall response rate (time frame: Up to 4 years). 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 NCT05873205.

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 2 Apr 2026.

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