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Engineered immune cells take on rare leukemia

NCT ID NCT04815356

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Sep 17, 2026 · Updated 6 times

Summary

This early-phase trial tests a new treatment for hairy cell leukemia (HCL) that has come back or not responded to standard care. The treatment uses the patient's own immune cells, which are modified in a lab to recognize and attack cancer cells. Up to 27 adults will receive chemotherapy followed by an infusion of these modified cells, and researchers will monitor safety and whether the cancer shrinks or disappears.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Anti-CD22 CAR T cells (a type of immune cell therapy)
What this could lead to
If successful, this could provide a new treatment option for people with hairy cell leukemia who have not responded to standard therapies.
What could go wrong
This is an early Phase I trial with only 27 participants, so the treatment may not work or could cause serious side effects. It is too soon to know if it will be effective.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 27 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2022

Expected to finish

Dec 2036

An estimate. End dates often move.

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

* INCLUSION CRITERIA * Histologically confirmed diagnosis of HCL or HCLv according to morphological and immunophenotypic criteria of WHO classification \[WHO, 2008 revised 2016\] of lymphoid neoplasm. * Participants should have any of the following indications for therapy: * ANC \<1/nL, * Hemoglobin \<10g/dL, * Platelets\<100/nL, * Symptomatic splenomegaly, * HCL mass with short axis \> 2 cm outside or \>0.5 cm inside the CNS, * HCL/HCLv count \>5/nL in blood or \>25/mm\^3 in CSF, * HCL/HCLv count doubling time \<6 months and increasing lytic or blastic bone lesions Participants who have eligible blood counts within 4 weeks from the initiation of study will not be considered ineligible if subsequent blood counts prior to enrollment fluctuate and become ineligible up until the time of enrollment. * HCL/HCLv, after prior treatment with, ineligible for, refusal of, or inability to obtain 1)rituximab given concurrently with or sequentially after purine analog, 2) moxetumomab pasudotox-tdft, and 3) BRAF-inhibition. * CD22 expression must be detected on greater than 80% of malignant cells by flow cytometry. * Participants must have measurable or evaluable disease at the time of enrollment, which may include any evidence of disease including minimal residual disease (MRD) detected by flow cytometry or immunohistochemistry. * Age \>=18 years * ECOG performance \<=2 (Karnofsky \>=60%, see Appendix A), participants are exempt from this criterion if poor performance status is related to HCL. * Participants must have adequate organ function as defined below: Participants must have recovered from the acute side effects of their prior therapy, such that eligibility criteria are met. If participants exhibit minor lab abnormalities that are determined to be related to HCL (not therapy-related), then those participants will be allowed to participate * Total bilirubin \<= 3 ULN, unless consistent with Gilbert s (ratio between total and direct bilirubin \> 5) * Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \<= 3x upper limit of normal (ULN) * Alkaline phosphatase \< 2.5 ULN * Serum creatinine \<= 1.5 mg/dL or creatinine clearance \>= 60 mL/min/1.73 m\^2 for participants with creatinine levels above institutional normal calculated using eGFR or measured * Serum albumin \> 2 g/dL * Prothrombin time (PT)/International Normalized Ratio (INR) \< 2.5x ULN (if on warfarin, PT/INR \< 3.5x ULN; If on any other anticoagulation, PT \< 2.5x ULN * Fibrinogen \>= 0.5x lower limit of normal * Participants with CNS disease are eligible, with exceptions * Participants with history of allogeneic stem cell transplantation are eligible if at least 100 days post-transplant, if there is no evidence of active graft-versus-host disease (GVHD) and no longer taking immunosuppressive agents for at least 30 days prior to initiation of study intervention. * Women of childbearing potential (WOCBP) must agree to use effective contraception (barrier, hormonal, intrauterine device \[IUD\], abstinence, surgical sterilization) at the study entry and up to 12 months after the last dose of combined chemotherapy or 4 months after cells infusion, whichever is later. Men must agree to use an effective method of contraception (barrier, surgical sterilization, abstinence) at the study entry and up to 4 months after the last dose of study drug. * Breastfeeding participants must be willing to discontinue breastfeeding from study treatment initiation through 4 months after the last dose of study drug. * Ability of participant to understand and the willingness to sign a written informed consent document. EXCLUSION CRITERIA * Pregnancy * Systemic chemotherapy, immunotherapy, or radiation therapy \<= 2 weeks prior to apheresis with the following exception: * Participants receiving steroids may be enrolled, provided there has been no increase in dose for at least 1 week prior to starting apheresis; * For radiation therapy: Radiation therapy must have been completed at least 3 weeks prior to enrollment (including CNS radiation), with the exception that there is no time restriction if the volume of bone marrow treated is less than 10% and also the participant has measurable/evaluable disease outside the radiation port. * Other anti-neoplastic investigational agents, or antibody-based therapies currently or within 2 weeks prior to apheresis * Participants taking warfarin * Prior CAR therapy within 30 days prior to apheresis or prior CAR therapy at any time with evidence for persistence of CAR T cells in blood samples (circulating levels of genetically modified cells of \>= 5% by flow cytometry) * Seropositive for human immunodeficiency virus (HIV) antibody. (Participants with HIV are at increased risk of lethal infections when treated with marrow-suppressive therapy. Appropriate studies will be undertaken in participants receiving combination antiretroviral therapy in the future should study results indicate effectiveness.) * Seropositive for hepatitis C virus (HCV) or positive for hepatitis B surface antigen (HbsAG). Participants who convert to negative will not be excluded for history of positive test. * Uncontrolled, symptomatic, intercurrent illness including but not limited to infection, congestive heart failure, unstable angina pectoris, cardiac arrhythmia, asthma, chronic obstructive pulmonary disease, psychiatric illness, or social situations that would limit compliance with study requirements or in the opinion of the PI would pose an unacceptable risk to the subject * History of severe, immediate hypersensitivity reaction attributed to compounds of similar chemical or biologic composition to any agents used in study or in the manufacturing of the cells (i.e., gentamicin)

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

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