Single-center phase I/IIa study with CART cells produced using the anti-CD19 transposon technique (TranspoCART19) for patients with CD19+ acute lymphoblastic leukemia resistant or refractory to treatment

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Clinica Universidad De Navarra

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

17 Mar 2026 → ongoing

Decision date (initial)

2026-01-02

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Dose response, Efficacy

Phase 1:
● To determine the maximum tolerated dose (MTD) and/or recommended dose of TranspoCART19 cells in patients with treatment-resistant or refractory CD19+ acute lymphoblastic leukemia.
Phase 2:
● To evaluate the efficacy of TranspoCART19 cell infusion in patients with treatment-resistant or refractory CD19+ acute lymphoblastic leukemia.

Secondary objectives 5

1. To evaluate adverse events occurring after TranspoCART19 cell infusion at three months and one year.
2. To evaluate overall and progression-free survival after TranspoCART19 infusion.
3. To evaluate the duration of response after TranspoCART19 infusion.
4. To evaluate the expansion and persistence of TranspoCART19 cells in peripheral blood after administration.
5. To evaluate the effect of treatment with TranspoCART19 on the quality of life of patients

Conditions and MedDRA coding

refractory or resistant CD19+ acute lymphoblastic leukemia

Regulatory references

Scientific advice from competent authorities

Spanish Agency Of Medicines And Medical Devices

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Diagnosis of CD19+ acute lymphoblastic leukemia, with a prognosis of less than 2 years due to meeting one of the following conditions: ● Relapsed/refractory, not a transplant candidate (due to refractory disease or lack of a donor) ● Relapse after allogeneic transplant
2. Measurable disease, understood as the presence of at least residual measurable disease by flow cytometry in bone marrow or peripheral blood at the time of inclusion.
3. Age over 4 years and under 70 years.
4. ECOG performance status 0-1. Patients with ECOG 2 may be included if due to hematologic disease (Appendix 1).
5. Life expectancy greater than 3 months.
6. Adequate venous access for lymphapheresis. No contraindications.
7. Signing of informed consent (patient or legal guardian).

Exclusion criteria 17

1. Patients who, in the opinion of their physician, may benefit from other approved, potentially curative therapeutic options, including commercial CAR-T cells.
2. Treatment with any experimental or non-marketed substance within four weeks prior to enrollment, or actively participating in another therapeutic clinical trial.
3. Previous treatment with CART (commercial or experimental).
4. Diagnosis of another malignancy, past or present. Patients who have been in complete remission for more than 3 years, or with a history of non-melanoma skin cancer or completely resected carcinoma in situ, may be included.
5. Overt central nervous system involvement (CNS-3) at the time of inclusion. Patients with a lesser degree (CNS-2) or CNS-3 who have responded to intrathecal chemotherapy will be eligible for inclusion.
6. Isolated extramedullary involvement (i.e., in the absence of residual measurable disease in peripheral blood or bone marrow).
7. In the case of relapse after allogeneic transplantation, at least 3 months must have passed since the transplant for inclusion. Furthermore, the patient must have been off systemic immunosuppressants for at least 30 days to be eligible for inclusion.
8. Active immunosuppressive therapy for graft-versus-host disease and other conditions. The use of corticosteroids for leukemia control should be limited as much as possible at the time of enrollment and should be discontinued before the infusion of TranspoCART19 cells.
9. Active infection requiring systemic medical treatment.
10. HIV infecction
11. Concurrent and uncontrolled medical conditions including cardiac, renal, hepatic, gastrointestinal, endocrine, pulmonary, neurological, or psychiatric conditions that, in the opinion of the investigator, pose a risk to the patient.
12. Positive serology for hepatitis B, defined as a positive test for HBsAg. Additionally, if the patient is HBsAg negative but has anti-HBc antibodies, a hepatitis B virus DNA test will be required. If the result is positive, the patient will be excluded.
13. Positive serology for hepatitis C, defined as a positive test for anti-HCV antibodies confirmed by RIBA.
14. Severe organ involvement, defined as cardiac ejection fraction <40%; DLCO <40%; estimated glomerular filtration rate <30 ml/min; bilirubin >3 times the upper limit of normal (unless due to Gilbert's syndrome).
15. Pregnant or breastfeeding women. Women of childbearing age must have a negative pregnancy test during the screening phase.
16. Women of childbearing potential, including those whose last menstrual cycle was in the year prior to screening, who are unable or unwilling to use highly effective contraception* from baseline to completion.
17. Men who are unable or unwilling to use highly effective contraception* from the start of the study until its completion.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. To determine the maximum tolerated dose and evaluate the safety of TranspoCART19 cell infusion.
2. To determine the efficacy of TranspoCART19 cell infusion

Secondary endpoints 6

1. Toxicity assessment at 3 months and 1 year, defined as the number of grade II-IV adverse events using the CTC (Common Toxicity Criteria) version 5.0 (Annex 4) and the ASTCT classification.
2. Treatment-related mortality (TRM) at 1, 3, and 1 year, defined as any death not directly caused by leukemia. For the purpose of estimating TRM, disease relapse or progression will be considered a competing event.
3. Progression-free survival (PFS) at six months and one year after the procedure, defined as the time between TranspoCART19 infusion and disease progression or death. Patients alive and in complete remission will be censored at the time of last follow-up.
4. Overall survival (OS) at one year after infusion, defined as the time between TranspoCART19 infusion and the patient's death from any cause. Surviving patients will be censored at the time of last follow-up.
5. Response rate (overall and complete) at three months and one year.
6. Best response rate achieved during the first 3 months of follow-up after administration of the first fraction of TranspoCART19.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Clinica Universidad De Navarra

Sponsor organisation

Clinica Universidad De Navarra

Address

Pio XII Etorbidea 36

City

Pamplona

Postcode

31008

Country

Spain

Scientific contact point

Organisation

Clinica Universidad De Navarra

Contact name

Sara Villar

Public contact point

Organisation

Clinica Universidad De Navarra

Contact name

Sara Villar

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications