Recruiting
Phase 1

alloHCT & CAR-T

Sponsor:

National Cancer Institute (NCI)

Code:

NCT07162038

Conditions

Hematologic Malignancies

Hematologic Neoplasms

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

mCD19-CAR-CD28-CD3-zeta.(anti-CD19 CAR) retroviral vector-transduced allogeneic peripheral blood lymphocytes (PBL)

Fludarabine

Cyclophosphamide

Mycophenolate Mofetil

Sirolimus

Study Details

Brief summary:

Background:

High-risk blood cancers (leukemias and lymphomas) often come back after treatment, and many cannot be cured with chemotherapy alone. These cancers may be treated and potentially cured in 2 ways: (1) Bone marrow transplant (allogeneic hematopoietic cell transplantation, or alloHCT) gives immune and blood stem cells from a donor. These new cells can attack the cancer and also grow into healthy blood. (2) Chimeric antigen receptor (CAR) T-cell therapy takes immune cells and changes them in a lab to better recognize and target certain cancers. But these 2 treatments are not usually given at the same time.

Objective:

To test alloHCT and CAR-T cell therapy, used together, in people with high-risk blood cancers.

Eligibility:

People aged 18 to 75 years with an aggressive blood cancer that has a protein on the surface called CD19. A healthy related donor aged 12 years or older is also needed; this donor may be a parent or child or may be some siblings or even extended family members, but has to be half-matched at something called the HLA (human leukocyte antigen).

Design:

Participants will be screened. They will have imaging scans, blood tests, and tests of their heart and lung function. They will have eye and dental exams. They may have fluid drawn from around their spinal cord (spinal tap) and tissue taken from inside a bone (bone marrow biopsy).

Healthy donors will provide bone marrow, immune cells, and about 9 tablespoons of blood for both the recipient s treatment and for research. They will also provide stool, saliva, and oral swabs just for research.

Recipient participants will stay in the hospital for 4 to 6 weeks. They will be given drugs over 6 days to prepare for the cell therapies. Both the donor bone marrow cells and CAR-T-cells will be given through a tube inserted into a vein. They will receive drugs to reduce complications after the treatments.

Participants will remain within a 1-hour drive of the hospital for 2 to 3 months after they leave the hospital. They will have frequent visits during that time. They will continue to have periodic follow-up visits for 5 years.

...

Conditions

Hematologic Malignancies

Hematologic Neoplasms

Study ID

NCT07162038

Start date

Nov 14, 2025

Status verified date

Aug 19, 2026

Completion date

Oct 1, 2034

Anticipated

Primary completion date

Nov 1, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

-INCLUSION CRITERIA - Recipient

1. Participants with high or very high-risk hematologic malignancies, as defined by the revised Disease Risk Index (DRI), or malignancy that remains persistently MRD+ (by flow cytometry, cytogenetics, FISH, PCR, or NGS) on most recently assessed disease specimen (within 2 months of initiating conditioning).
2. Hematologic malignancy must be CD19+ (uniform expression on immunohistochemistry or >= 80% on flow cytometry) as confirmed by CD19 IHC assay (BT51E) or flow cytometry (BD QuantiBRITE(TM) Beads PE Fluorescence Quantitation Kit). (Participants do not have to have refused or lack access to commercial anti-CD19 CAR-T-cell therapies since this study focuses on the integration of CAR-T cells and HCT and not specifically the CAR-T cells themselves; furthermore the construct used to manufacture this product is the same as used in a current commercial product, but developed from a new batch and with a similar but not identical manufacturing process.)
3. Age 18-75
4. Karnofsky >= 60%.
5. Participants must have adequate organ and marrow function as defined below:

  • Cardiac ejection fraction >= 45% by 2D echocardiography;
  • Forced expiratory volume-1 (FEV-1) and diffusing capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin) all of >=50% predicted (this requirement would be waived in participants who are unable to properly perform pulmonary function tests - in such circumstances, participants must have pulse oximetry >=90% on room air and no dyspnea or obvious pulmonary restrictions);
  • Estimated serum creatinine clearance of >= 60 ml/minute/1.73m\^2 calculated using eGFR in the clinical lab (participants with estimated serum creatinine clearance less than 60 may have measured creatinine clearance performed and if >= 60 will be considered eligible);
  • Total bilirubin <= 2X the upper limit of normal (participants with documented or suspected Gilbert s are exempt from this requirement);
  • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) <= 5X the upper limit of normal.
6. At least one available HLA-haploidentical donor
7. Women of child-bearing potential (WOCBP) must agree to use a highly effective method of contraception (hormonal, intrauterine device (IUD), surgical sterilization, abstinence) at the study entry and for 1 year after transplant (restriction period).

Men must agree to use an effective method of contraception (barrier, surgical sterilization, abstinence) at the study entry and for 1 year after transplant. We also will recommend men with female partners of childbearing potential to ask female partners to be on highly effective birth control (hormonal, intrauterine device (IUD), surgical sterilization). Men must not freeze or donate sperm within the same period.
8. Breastfeeding participants must be willing to discontinue breastfeeding from study treatment initiation through 1 year after transplant.
9. Participants seropositive for human immunodeficiency virus (HIV) not due to intravenous immunoglobulin, must have adequate viral suppression (HIV viral load < 200 viral copies per ml of blood) prior to the beginning of conditioning.
10. For participants seropositive for hepatitis B virus (HBV) core antibody not due to intravenous immunoglobulin, a HBV viral load should be undetectable.
11. Participants seropositive for hepatitis C virus (HCV) not due to intravenous immunoglobulin must have been treated and cured. For participants with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load.
12. Ability of participant or legally authorized representative (LAR) to understand and the willingness to sign a written informed consent document.
13. Ability and willingness of participant to co-enroll on 20-C-0051: Gene Therapy Follow Up Protocol for Subjects Previously Enrolled in NCI for Immuno-Oncology Studies

14 Willingness to remain in the NIH hospital or, if discharged, stay close to the NIH (<60 minutes drive), for a minimum of 100 days after transplant or longer if there are complications. Participants must commit to having an adult caregiver with them during the first 100 days after transplant in case of discharging from the hospital before 100 days.

INCLUSION CRITERIA - Donor

1\. Related donor (age >=12) deemed suitable, eligible, and willing to donate, per clinical evaluations, who are additionally willing to donate blood, bone marrow, and stool for research. Related donors will be evaluated in accordance with existing Standard Policies and Procedures for determination of eligibility and suitability for clinical donation.

EXCLUSION CRITERIA - Recipient

1. Participants who are receiving any other investigational agents within 3 weeks prior to the beginning of conditioning.
2. Active CNS involvement of primary hematologic malignancy
3. Active malignancy of non-hematopoietic type (excluding non-melanoma skin cancers) which is metastatic, relapsed/refractory to treatment, or locally advanced and not amenable to intended curative treatment per standard of care.
4. Prior checkpoint inhibitor therapy within 6 weeks prior to the beginning of conditioning.
5. Prior history of seizure.
6. Uncontrolled infection.
7. History of allergic reactions attributed to compounds of similar chemical or biologic composition to study agents used in study.
8. Positive beta-HCG serum or urine pregnancy test performed in females of childbearing potential at screening. (A low positive test in a post-menopausal woman may not be exclusionary if deemed not indicative of pregnancy per gynecology.)
9. Uncontrolled intercurrent illness evaluated by medical history, physical exam, EKG, and laboratory testing (e.g., severe endocrinopathy, disseminated intravascular coagulation, profound electrolyte disturbance) that would make it unsafe to proceed with transplantation.

EXCLUSION CRITERIA - donor

1\. Pregnancy

Study Design

Enrollment

155 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

no intervention: Donor

Donors for Arms 1 and 2

experimental: Phase I: Dose Escalation

Participants receiving CAR-T cells at escalation/de-escalation dose levels to determine MTD

experimental: Phase I: Dose Expansion

Participants receiving CAR-T cells at MTD

Interventions

mCD19-CAR-CD28-CD3-zeta.(anti-CD19 CAR) retroviral vector-transduced allogeneic peripheral blood lymphocytes (PBL)

CAR-T cell infusion given at four escalating dose levels (DL1: 3 x 10\^4 cells/kg, DL2: 1 x 10\^5 cells/kg, DL3: 3 x 10\^5 cells/kg, DL4: 1 x 10\^6 cells/kg) with a dose de-escalation dose (DL-1: 1 x 10\^4 cells/kg), if needed.

Fludarabine

Pre-transplant: 30 mg/m\^2 IV infusion over 30-60 minutes once daily for 5 days from day -6 through day -2

Cyclophosphamide

Pre-transplant: 14.5 mg/kg/day IV daily for 2 days pre-transplant on day -6 and day -5.

Post-transplant: 25 mg/kg/day on day +3 and day +4.

Mycophenolate Mofetil

15 mg/kg orally or IV three times daily (max 1000 mg/dose) starting on day +5, continued through day +35 post-transplant.

Sirolimus

Loading dose of 6 mg orally given on day +5, then maintenance dose starting at 2 mg orally daily on day +6 with dose adjustments to maintain a trough of 5-12 ng/ml, continued through day +60 post-transplant.

CD19 Flow Cytometry Assay

Assay used to determine CD19+ status

CD19 Immunohistochemical Assay

Assay used to determine CD19+ status

Total Body Irradiation

400 centigray (cGy) to be delivered in 2 fractions as 200 cGy per fraction twice a day on Day -1 pre-transplant.

Primary outcome measure

  • Identify the safety of anti-CD19 CAR T-cell therapy in combination with HLA-haploidentical HCT in participants with high risk CD19+ hematologic malignancies [ Time Frame: 28 days (or 35 days for alternate dose levels) ]
  • Estimate the 1 year relapse and survival outcomes at the maximum tolerated dose [ Time Frame: 1 year ]

Central Contacts and Locations

Central contacts

Locations

National Institutes of Health Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

National Cancer Institute Referral Office

888-624-1937ncimo_referrals@mail.nih.gov

More Information

Sponsor

National Cancer Institute (NCI)

Last update posted

Aug 26, 2026

Last verified

Aug 19, 2026

Keywords

  • Chimeric-Antigen-Receptor T-cell Therapy
  • High Risk Hematologic Malignancy
  • CD19
  • Hematopoietic Cell Transplant

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by National Cancer Institute (NCI) on 2026-08-26.