Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Authorised, recruiting
Participants planned
20
Countries
1
Sites
2
T-cell acute lymphoblastic Lymphoma
To monitor patients with primary relapsed/refractory (R/R) CD1a-positive T-ALL/LL who received OC-1 cells, for 15 years following CAR-T infusion to assess the risk of delayed adverse events (AEs).
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] - Neoplasms [C04], Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Trial duration
- 21 May 2025 → ongoing
- Decision date (initial)
- 2025-04-11
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- OneChain Immunotherapeutics
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety
To monitor patients with primary relapsed/refractory (R/R) CD1a-positive T-ALL/LL who received OC-1 cells, for 15 years following CAR-T infusion to assess the risk of delayed adverse events (AEs).
Secondary objectives 2
- To assess the long-term efficacy of the product.
- Monitor the persistence of OC-1 cells
Conditions and MedDRA coding
T-cell acute lymphoblastic Lymphoma
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.1 | LLT | 10066110 | T-cell lymphoblastic leukemia acute | 10029104 |
| 21.1 | LLT | 10025245 | Lymphoblastic lymphoma (Precursor T-lymphoblastic lymphoma/leukaemia) recurrent | 10029104 |
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | This is a multi-center, long-term follow-up study (LTFU). Per Health Authorities guidelines (FDA 2006, EMA 2009) for gene therapy medicinal products that utilize integrating vectors (e.g. lentiviral vectors), long term safety and efficacy follow-up of treated patients is required for 15 years post exposure.
|
Not Applicable | None |
Regulatory references
- Plan to share IPD
- No
- IPD plan description
- NA
| EU CT number | Title | Sponsor |
|---|---|---|
| 2024-514591-40-00 | 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)_CARxALL | Onechain Immunotherapeutics S.L. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 3
- Patients previously treated with at least one fraction of OC-1 cell investigational product
- Patient or patient’s legal representative has provided signed and dated informed consent.
- Patient is able to comply with the study requirements.
Exclusion criteria 1
- None. All patients who have received prior treatment with OC-1 cell investigational product are eligible for this long-term follow up (LTFU) study.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 2
- Number of adverse events grade III-IV, related or possibly related, using Common Toxicity Criteria for Adverse Events (CTCAE) version 5
- Proportion of patients with: - New malignancies. - Incidence/exacerbation of pre-existing neurologic disorder. - New incidence or exacerbation of a prior rheumatologic or other ATMP-CLINICAL TRIAL PROTOCOL OC-01-23002 EU CT Number: 2024-516617-20-00 Page 6 de 39 Protocol Version 1, 4 november 2024 autoimmune disorder. - New incidence of a hematologic disorder. - New incidence of infection (potentially product-related)- - New incidence of skin disorder
Secondary endpoints 5
- Overall survival following OC-1 cell therapy infusion
- Progression-free survival
- Progression-free survival in patients who did not received HSCT after OC-1 infusion
- Proportion of patients who received an HSCT post OC-1 infusion
- Persistence of OC-1, as determined by flow cytometry and quantitative analysis by qPCR. Genomic copy number integrations of the CAR in peripheral blood (PB) T cells and percentage of CD1a CAR-expressing T cells.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD11346828 · Product
- Active substance
- Autologous T-Cells Ex Vivo Modified with a Lentiviral Vector Encoding a Chimeric Antigen Receptor Specific for CD1A
- Substance synonyms
- OC-1
- Other product name
- humanised CD1a-CAR T
- Pharmaceutical form
- SUSPENSION FOR IV INFUSION
- Route of administration
- INTRAVASCULAR USE
- Authorisation status
- Not Authorised
- MA holder
- ONECHAIN IMMUNOTHERAPEUTICS
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/21/2535
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
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Spain | Authorised, recruiting | 20 | 2 |
| Rest of world | — | 0 | — |
Investigational sites
Hospital Clinic De Barcelona
Hematology, Calle Villarroel 170, 08036, Barcelona
Hospital Sant Joan De Deu Barcelona
Hematology, Passeig De Sant Joan De Deu 2, 08950, Esplugues De Llobregat
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Spain | 2025-05-21 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-01-13 | Spain | Acceptable with conditions 2025-04-11
|
2025-04-11 |