Hematologic MalignancyPhase 1NCT06326463
CAR T-cell Therapy Directed to CD70 for Pediatric Patients With Hematological Malignancies
A Phase 1 study testing Fludarabine, Cyclophosphamide and CD70-CAR T cell infusion (Autologous) in people with Hematologic Malignancy and ALL, Childhood, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
The study participant has one of the following blood cancers: acute myelogenous leukemia (AML)/myelodysplastic syndrome (MDS), acute lymphoblastic leukemia (B-ALL, T-ALL) or Lymphoma. Your cancer has been difficult to treat (refractory) or has come back after treatment (relapse). Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Hematologic Malignancy, ALL, Childhood, AML, Childhood
- What is being tested?
- Fludarabine, Cyclophosphamide and CD70-CAR T cell infusion (Autologous)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Oct 2024to 1 Jul 2031
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age ≤21 years old
- Relapsed/refractory CD70+ hematological malignancy
- Relapsed disease: Patients developing recurrent disease after a prior complete remission (CR)
- Refractory disease: Patients with persistent disease despite 3 cycles of induction chemotherapy.
- Relapsed/refractory CD70+ AML or MDS:
- Relapsed disease that is CD70 positive
Show all 26Show fewer
- Refractory disease that is persistent despite 3 cycles of chemotherapy
- Relapsed/refractory CD70+ B-cell ALL:
- Relapsed disease that is CD70 positive and CD19 negative/dim or patients otherwise ineligible for CD19-directed therapies including:
- Patients in 2nd or greater relapse
- Patients with relapse after allogeneic HSCT
- Relapsed/refractory CD70+ T-cell ALL:
- Relapsed /refractory disease that is CD70 positive
- Mixed Phenotype Acute Leukemia (MPAL):
- Relapsed/refractory that is CD70 positive
- Relapsed/refractory CD70+ lymphoma:
- Relapsed disease that is CD70 positive and CD19 negative/dim or patients otherwise ineligible for CD19-directed therapies including:
- Patients in 2nd or greater relapse
- Patients with relapse after allogeneic HSCT
- Estimated life expectancy of >12 weeks
- Karnofsky or Lansky (age- dependent) performance score ≥50
- Patients with a history of prior allogeneic HCT must be clinically recovered from prior HCT therapy, have no evidence of active GVHD and have not received a donor lymphocyte infusion (DLI) within the 28 days prior to apheresis
- Patient must have an identified HCT donor
- For females of childbearing age:
- Not lactating with intent to breastfeed
- Not pregnant with negative serum or urine pregnancy test within 7 days prior to enrollment
Who cannot join
- Known primary immunodeficiency
- Known history of HIV positivity
- Severe intercurrent bacterial, viral or fungal infection
- History of hypersensitivity to cornstarch or hydroxyethyl starch
- Patients with acute promyelocytic leukemia (APL)
- Known contraindication to protocol defined lymphodepleting
- chemotherapy regimen of Fludarabine/cyclophosphamide
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Swati Naik, MD, 866-278-5833, referralinfo@stjude.org
About this study
The study participant has one of the following blood cancers: acute myelogenous leukemia (AML)/myelodysplastic syndrome (MDS), acute lymphoblastic leukemia (B-ALL, T-ALL) or Lymphoma. Your cancer has been difficult to treat (refractory) or has come back after treatment (relapse).
Primary Objective
Read more
To determine the safety and maximum tolerated dose of intravenous infusions of escalating doses of CD70-CAR T cells in patients (≤21 years) with recurrent/refractory CD70+ hematological malignancies after lymphodepleting chemotherapy.
Secondary Objectives
To evaluate the antileukemic activity of CD70-CAR T cells. We will determine the anti- leukemic activity of the CD70-CAR T cells in the bone marrow and in the treatment of extramedullary disease.
Read the detailed description
The primary interventions are a lymphodepleting chemotherapy regimen (fludarabine and cyclophosphamide), followed by a single autologous infusion of CD70-CAR T cells.
Phase I study evaluating three (3) dose levels of CD70-CAR T cells.
- Conditions
- Hematologic Malignancy, ALL, Childhood, AML, Childhood, Lymphoma, MDS
- Being tested
- Fludarabine, Cyclophosphamide, CD70-CAR T cell infusion (Autologous), Mesna
- Medical subject headings
- Hematologic Neoplasms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FludarabineDrug
Also called Fludara
40mg/m2, Day -4, -3 and -2
CyclophosphamideDrug
Also called Cytoxan
Day -3 and Day-2
REST DAY, -1
CD70-CAR T cell infusion (Autologous)Drug
Day 0 or +1
MesnaDrug
Also called Mesnex
Mesna is generally dosed at approximately 25% of the cyclophosphamide dose. It is generally given intravenously prior to and again at 3, 6 and 9 hours following each dose of cyclophosphamide
| Study group | Role | What they receive |
|---|---|---|
| CD70- CAR T cell Therapy | Experimental | Patients will receive autologous (their own) cells. Receives: Drug: Fludarabine; Drug: Cyclophosphamide; Drug: CD70-CAR T cell infusion (Autologous); Drug: Mesna |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose of CD70-CAR T cells
Phase I design to determine the maximum tolerated dose (MTD) of autologous, CD70-CAR T cells. Three (3) dose levels will be evaluated (1x10e6, 3x10e6, and 1x10e7 cells/kg).
Measured: 28 days after CD70-CAR T-cell infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2024
- Study start16 Oct 2024
- Primary completion1 Jul 2030 (estimated)
- Study completion1 Jul 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic MalignancyStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Swati Naik, MBBS, Principal investigator, St. Jude
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06326463
- Sponsor's protocol ID
- DIRECT70
- Other IDs
- NCI-2024-03240
- Status last verified
- Jan 2026
- First submitted
- 15 Mar 2024
- Last updated
- 23 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.
Study at a glance
From the registry record, last updated by the sponsor on 23 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- CAR T-cell Therapy Directed to CD70 for Pediatric Patients With Hematological Malignancies
- ClinicalTrials.gov ID
- NCT06326463
- Acronym / sponsor ID
- DIRECT70
- Conditions
- Hematologic Malignancy, ALL, Childhood, AML, Childhood, Lymphoma, MDS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- 16 Oct 2024 (actual)
- Primary completion
- 1 Jul 2030 (estimated)
- Study completion
- 1 Jul 2031 (estimated)
- First posted
- 22 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study participant has one of the following blood cancers: acute myelogenous leukemia (AML)/myelodysplastic syndrome (MDS), acute lymphoblastic leukemia (B-ALL, T-ALL) or Lymphoma. Your cancer has been difficult to treat (refractory) or has come back after treatment (relapse).
Primary Objective
To determine the safety and maximum tolerated dose of intravenous infusions of escalating doses of CD70-CAR T cells in patients (≤21 years) with recurrent/refractory CD70+ hematological malignancies after lymphodepleting chemotherapy.
Secondary Objectives
To evaluate the antileukemic activity of CD70-CAR T cells. We will determine the anti- leukemic activity of the CD70-CAR T cells in the bone marrow and in the treatment of extramedullary disease.
Detailed description
The primary interventions are a lymphodepleting chemotherapy regimen (fludarabine and cyclophosphamide), followed by a single autologous infusion of CD70-CAR T cells.
Phase I study evaluating three (3) dose levels of CD70-CAR T cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CD70- CAR T cell Therapy | Experimental | Drug: Fludarabine, Drug: Cyclophosphamide, Drug: CD70-CAR T cell infusion (Autologous), Drug: MesnaPatients will receive autologous (their own) cells. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (26 listed)
- Age ≤21 years old
- Relapsed/refractory CD70+ hematological malignancy
- Relapsed disease: Patients developing recurrent disease after a prior complete remission (CR)
- Refractory disease: Patients with persistent disease despite 3 cycles of induction chemotherapy.
- Relapsed/refractory CD70+ AML or MDS:
Key exclusion criteria (7 listed)
- Known primary immunodeficiency
- Known history of HIV positivity
- Severe intercurrent bacterial, viral or fungal infection
- History of hypersensitivity to cornstarch or hydroxyethyl starch
- Patients with acute promyelocytic leukemia (APL)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FludarabineAlso: Fludara40mg/m2, Day -4, -3 and -2 | Drug | CD70- CAR T cell Therapy |
| CyclophosphamideAlso: CytoxanDay -3 and Day-2 REST DAY, -1 | Drug | CD70- CAR T cell Therapy |
| CD70-CAR T cell infusion (Autologous)Day 0 or +1 | Drug | CD70- CAR T cell Therapy |
| MesnaAlso: MesnexMesna is generally dosed at approximately 25% of the cyclophosphamide dose. It is generally given intravenously prior to and again at 3, 6 and 9 hours following each dose of cyclophosphamide | Drug | CD70- CAR T cell Therapy |
Primary outcome
Maximum tolerated dose of CD70-CAR T cells
Time frame: 28 days after CD70-CAR T-cell infusion
Phase I design to determine the maximum tolerated dose (MTD) of autologous, CD70-CAR T cells. Three (3) dose levels will be evaluated (1x10e6, 3x10e6, and 1x10e7 cells/kg).
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2026.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Swati Naik, MBBS principal investigator
St. Jude
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Fludarabine, Cyclophosphamide and CD70-CAR T cell infusion (Autologous) in people with Hematologic Malignancy and ALL, Childhood, run by St. Jude Children's Research Hospital 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 26 inclusion and 7 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: looking for participants now, as of the sponsor's last update on 23 Jan 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 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?
Contact Swati Naik, MBBS (866-278-5833, referralinfo@stjude.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum tolerated dose of CD70-CAR T cells (time frame: 28 days after CD70-CAR T-cell infusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Jan 2026.