Clinical trial NCT05679895: Safety and Efficacy of OC-1 Therapy in Patients With R/R T-ALL/LL
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: T-cell Acute Lymphoblastic Leukemia
Lymphoblastic T-Cell Lymphoma
Keywords: CAR-T-based therapies
CD1a
Lead sponsor: OneChain Immunotherapeutics
Sponsor class: INDUSTRY
Collaborators: name: BioClever 2005 S.L.
name: Hospital Clinic of Barcelona
name: Hospital Sant Joan de Deu
First posted: 2023-01-11
Enrollment: 12
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: First in humans, exploratory, open-label, single-arm, multicentre, non-competitive, dose escalation study to assess the safety and efficacy of CD1a-CAR T therapy in patients with relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LL)


Eligibility criteria: Inclusion Criteria:

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:

   1. Relapse after allogeneic haematopoietic stem cell transplantation (allo-HSCT)
   2. 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.
   3. Refractory first relapse.
   4. 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:

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 timeframe \<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 (positive urine/blood pregnancy test) or lactating.

Primary outcomes:
measure: Number of adverse events grade III-IV
timeFrame: 1 year particularly the first 28 days after infusion
measure: Incidence of severe Cytokine release syndrome (CRS) and Immune effector cell-associated neurotoxicity syndrome (ICANS)
timeFrame: 1 year particularly the first 28 days after infusion
measure: Non-relapse treatment-related mortality (NRM)
timeFrame: 1 year
measure: Number of adverse events of special interest (AESI)
timeFrame: 1 year
measure: Assessment of the immunological homeostasis
timeFrame: 1 year
measure: Incidence of the treatment-related dermatological events
timeFrame: 1 year
measure: Number of patients developing dose limiting toxicity (DLT)
timeFrame: first 28 days after infusion
Secondary outcomes:
measure: Remission rate
timeFrame: 1 year
measure: Response rates
timeFrame: 1 year
measure: Duration of remission
timeFrame: 1 year
measure: Minimal residual disease (MRD) response
timeFrame: 1 year
measure: Progression-free survival (PFS)
timeFrame: 1 year
measure: Overall survival
timeFrame: 1 year
measure: Persistence of OC-1
timeFrame: 1 year
Other outcomes:
Locations:
city: Barcelona
country: Spain
facility: Hospital Clínic
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Déu
Study officials:
References: