Safety and efficacy of hCD1a-CAR T (OC-1) therapy, in patients with relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LL)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Onechain Immunotherapeutics S.L.

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

31 Jan 2023 → ongoing

Decision date (initial)

2024-07-17

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

OneChain Immunotherapeutics

External identifiers

EU CT number

2024-514591-40-00

EudraCT number

2021-002333-42

ClinicalTrials.gov

NCT05679895

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To assess the safety of OC-1 in patients with primary relapsed/refractory CD1a-positive T-ALL/LL.

Secondary objectives 4

1. To describe the response to OC-1.
2. To describe the duration of the response after the administration of OC-1.
3. To describe the overall survival after the administration of OC-1
4. To describe the persistence of OC-1 cells in peripheral blood after administration

Conditions and MedDRA coding

T-cell Acute Lymphoblastic Leukemia

Study design 1 period

Regulatory references

Scientific advice from competent authorities

Agencia Espanola De Medicamentos Y Productos Sanitarios

Plan to share IPD

No

IPD plan description

NA

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Children older than 2 years or adults, male and female in both groups.
2. Patients CD1a antigen blast expression ≥20% at inclusion, either immunophenotypically (flow cytometry) or histologically confirmed.
3. R/R CD1a-positive T-ALL/LL patients, including morphologic or MRD-detectable (≥1x10-4) bone marrow and/or extramedullary relapses after 2 therapy lines: ­ Relapse after allogeneic haematopoietic stem cell transplantation (allo-HSCT) ­ Primary refractoriness, defined as either morphologic persistence or detectable MRD (≥1x10-4) after two standard therapy lines, making the patient not candidate for allo-HSCT. ­ Refractory first relapse. ­ Second or further relapse.
4. Patient without reproductive capacity or else, commitment to the use of a highly effective method of contraception during the study.

Exclusion criteria 10

1. Limiting organ dysfunction, such as uncontrolled cardiac (e.g., depressed left ventricular ejection fraction (LVEF), <45%), pulmonary, liver, renal or CNS dysfunction.
2. Allo-HSCT within a time frame <3 months, or requiring continued immunosuppressive treatment for graft versus host disease (GvHD).
3. Uncontrolled epilepsy or underlying central nervous system (CNS) severe disease.
4. Active bacterial, fungal or viral infection not controlled by adequate treatment.
5. Known HIV, active hepatitis B (HBV), or hepatitis C virus (HCV) infection.
6. Women who are pregnant (urine/blood pregnancy test positive) or lactating.
7. Severe illness or medical condition, which would not permit the patient to be managed according to the protocol.
8. Suffering from a serious autoimmune disease or immunodeficiency disease
9. The patient participated in other experimental drug clinical trial within 6 weeks prior to OC-1 infusion.
10. Other non-controlled concomitant neoplasms.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 7

1. Number of adverse events grade III-IV using Common Toxicity Criteria for Adverse Events (CTCAE) version 5.
2. Incidence of severe Cytokine release syndrome (CRS) # grade III and Immune effector cell-associated neurotoxicity syndrome (ICANS) # grade II
3. Proportion of patients with non-relapse, treatment-related mortality (NRM)
4. Number of adverse events of special interest (AESI)
5. Assessment of the immunological homeostasis, through the description of lymphocytes subpopulations at each study timepoint.
6. Incidence of severe (#3) treatment-related dermatological events.
7. Number of patients developing dose limiting toxicity (DLT)

Secondary endpoints 6

1. Remission rate: Percentage of patients presenting complete response (CR) or incomplete count recovery (CRi) at any point after treatment
2. Duration of remission: The duration of the remission will be assessed from the first documented date of remission status until progression (in days)
3. Minimal residual disease (MRD) response by flow cytometry: blast count among patients presenting bone marrow complete response (sensitivity 10-4).
4. Progression-free survival: time since the first infusion to the documented loss of response. In patients not presenting a CR or CRi progression free survival will be zero
5. Overall survival time since first infusion to date of death
6. Persistence of OC-1, as determined by flow cytometry and quantitative analysis by qPCR

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Onechain Immunotherapeutics S.L.

Sponsor organisation

Onechain Immunotherapeutics S.L.

Address

Carrer De Muntaner 383 3rd-2nd

City

Barcelona

Postcode

08021

Country

Spain

Scientific contact point

Organisation

Onechain Immunotherapeutics S.L.

Contact name

Medical Department

Public contact point

Organisation

Onechain Immunotherapeutics S.L.

Contact name

Medical Department

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

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

Application history

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