CD7-CAR-T Cells in Pediatric Relapsed/Refractory CD7+ T-ALL/LL

NCT06064903 | Recruiting Phase 1 DMC

• 1 other identifier: CD7-CAR01
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 1

Brief Summary

The main purpose of this study is to evaluate the safety, to establish the recommended dose, and to evaluate the antitumor effect of CD7-CART01 in pediatric patients with relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) or lymphoblastic lymphoma (T-LL).

Conditions

T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma

Keywords

T-ALL; T-LL; Relapsed; Refractory; anti-CD7 PEBL-CAR T

Outcome Measures

Primary Outcomes (2)

• Safety (Phase I) and definition of the recommended dose (Phase I)

  To evaluate the safety of the infusion of CD7-CART01, explore the dose-limiting toxicities (DLT) of the cellular product and define the recommended dose of CD7-CART01, defined as the maximum tolerated dose/recommended dose (MTD/RD), to be evaluated for efficacy in the phase II extension

  28 days

• Antitumor effect of CD7-CART01 (Phase II)

  To evaluate the portion of patients achieving either BM, PB and CSF morphological complete remission (CR) or CR with incomplete blood count recovery (CRi) and with minimal residual disease (MRD) negativity at day 28

  28 days

Secondary Outcomes (7)

• Number of participants with treatment-related adverse events as assessed by CTCAE v5.0

  28 days

• Antitumor effect of CD7-CART01 (Phase I and II)

  28 days

• In vivo persistence of CD7-CART01 (Phase I and II)

  3 years

• Relative proportion and phenotype of CD3+CAR+ T cells/CD3+CAR- T cells/NK cells (Phase I and II)

  3 years

• Assessment of cumulative incidence of relapse

  3 years

Study Arms (1)

CD7-CART01

EXPERIMENTAL

A single IV infusion of CD7-CART01 (CD7-directed chimeric antigen receptor T-cells) on Day 0 after lymphodepleting regimen. Patients will receive the following lymphodepleting regimen: * Fludarabine 30 mg/m2 per day over 1 hour on days -6, -5, -4 and -3 * Cyclophosphamide 1000 mg/m2 per day on days -4 and -3. CD7-CART01 will be infused at the following dose levels: * DL1: 0.5 x 10\^6 CAR+ cells/kg * DL2: 1 x 10\^6 CAR+ cells/kg If 2 DLTs are observed an additional DL0 of 0.25 x 106 CAR+ cells /kg will be explored.

Biological: CD7-CART01

Interventions

CD7-CART01 BIOLOGICAL

A single IV infusion of CD7-CART01 on Day 0

CD7-CART01

Eligibility Criteria

• Age: 6 Months - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis of CD7 expressing (\> 98% CD7 expression on blast cells) T-ALL or LL and one of the following:
• Patients in 1st or subsequent relapse, after at least one standard frontline chemotherapy with BM involvement (MRD \>1% in 2 consecutive determinations or evidence of morphological relapse, i.e. \>5% blasts in BM);
• Relapse after allogeneic HSCT, if at least 100 days post-transplant, if there is no evidence of active GVHD and if the patient is no longer taking immunosuppressive agents for at least 30 days prior to enrollment;
• CNS disease as defined as \> 5 WBCs/mcL in CSF with morphological/flow-cytometry evidence of blasts or biopsy proven recurrence in the eye or brain;
• Extramedullary relapse as defined by morphological evidence of blasts in the testis or any other extramedullary sites;
• Refractory disease, defined as MRD ≥ 1% or \<1% but persistently positive (i.e. a positive MRD value confirmed by PCR at 2 subsequent evaluations performed at least 2 weeks apart), at the end of consolidation blocks in newly diagnosed patients;
• Age: 6 months - 25 years.
• Adequate venous access for apheresis or eligible for appropriate catheter placement, and no other contraindications for leukapheresis.
• Voluntary informed consent is given. For subjects \<18-year-old, or below the age required by each Country regulation, their legal guardian must give informed consent. Pediatric subjects will be included in age-appropriate discussion and verbal assent will be obtained for those greater than or equal to 12 years of age, when appropriate, or to sign age-adapted informed consent, according to the regulatory requirement of each Country.
• Clinical performance status: Patients \> 16 years of age: Karnofsky greater than or equal to 60%; Patients \< 16 years of age: Lansky scale greater than or equal to 60%.

You may not qualify if:

• Severe, uncontrolled active intercurrent infections.
• HIV, or active HCV and/or HBV infection.
• Blast contamination in peripheral blood \>5%, by flow-cytometry, at the time of leukapheresis collection.
• Concurrent or recent prior therapies, before apheresis:
• Systemic chemotherapy in the 2 weeks preceding apheresis collection
• Nelarabine, daratumomab, clofarabine exposure in the 3 weeks preceding apheresis collection
• Anti-thymocyte globulin (ATG) or Alemtuzumab (Campath®) in the 8 weeks preceding apheresis collection
• Immunosuppressive agents in the 2 weeks preceding apheresis collection
• Radiation therapy must have been completed at least 2 weeks prior to apheresis
• Other anti-neoplastic investigational agents currently administered or within 30 days prior to apheresis (i.e. start of protocol therapy)
• Exceptions:
• There is no time restriction with regard to prior intrathecal chemotherapy, provided that there is complete recovery from any acute toxic effects of such chemotherapy;
• Patients who relapse while receiving standard ALL maintenance chemotherapy will not be required to have a waiting period before entry onto this study provided they meet all other eligibility criteria;
• Subjects receiving steroid therapy at physiologic replacement doses only are allowed provided there has been no increase in dose for at least 2 weeks prior to starting apheresis.
• Treatment eligibility

Sponsors & Collaborators

• Bambino Gesù Hospital and Research Institute: lead

Study Sites (1)

Ospedale Pediatrico Bambino Gesù

Rome, Rome, 00165, Italy

RECRUITING

MeSH Terms

Conditions

Precursor T-Cell Lymphoblastic Leukemia-Lymphoma; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Recurrence

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Franco Locatelli, MD, PhD

  Director Department of Hematology/Oncology and Cell and Gene Therapy

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Franco Locatelli, MD, PhD

CONTACT

+3966859; franco.locatelli@opbg.net

Francesca Del Bufalo, MD, PhD

CONTACT

+3966859; francesca.delbufalo@opbg.net

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 12, 2023
• First Posted: October 3, 2023
• Study Start: April 21, 2024
• Primary Completion (Estimated): September 30, 2028
• Study Completion (Estimated): September 30, 2040
• Last Updated: December 2, 2025

Record last verified: 2025-11

Locations

IT (1)