Safety and Efficacy of GYA01 (CART84) in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and Acute Lymphoblastic T Leukemia Patients (T-ALL).

NCT07471789 | Recruiting Phase 1 DMC

• 1 other identifier: GYA-CART84-AL-001
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 2

Brief Summary

This Phase I/IIa clinical study is testing an experimental treatment called GYA01 (CART84) for people with acute myeloid leukemia (AML) or T-cell acute lymphoblastic leukemia (T-ALL) whose disease has come back after treatment (relapsed) or did not respond to treatment (refractory). GYA01 (CART84) is a type of CAR T-cell therapy. In this approach, a participant's own T cells (a type of immune cell) are collected and changed in a laboratory to help them better recognize and attack leukemia cells. The modified cells GYA01 (CART84) are then given back to the participant through an infusion into a vein. The study is being done to: Find a dose that can be given safely (Phase I) by treating small groups of participants with increasing dose levels and carefully monitoring side effects. Look for early signs that GYA01 (CART84) may help control AML or T-ALL (Phase IIa). Participants will be closely monitored for side effects and for changes in their leukemia after the infusion, and followed over time to understand safety and possible benefit.

Conditions

Acute Myeloblastic Leukaemia; Leukemia, T-Cell

Keywords

CART84; CART; AML; T-ALL; CD84; CAR-T; Cell Therapy; Gene Therapy; GYA01

Outcome Measures

Primary Outcomes (2)

• Number of Participants with Adverse Events.

  Primary Outcome Measure Phase I and Secondary Outcome Measure Phase II: Frequency and severity of adverse events (AEs) and serious AEs (SAEs) occurring after CART84 infusion using the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 and American Society for Transplantation and Cellular Therapy (ASTCT) grading for CRS/ICANS.

  2 years

• Proportion of patients achieving ORR after first infusion of CART84

  Primary Outcome Measure Phase II and Secondary Outcome Measure Phase I: Proportion of patients achieving ORR at day 30 after first infusion of CART84 as assessed by European Leukemia Net (ELN) 2022 guidelines

  30 days

Secondary Outcomes (9)

• Proportion of patients achieving late ORR

  day 90

• Proportion of patients achieving measurable residual disease (MRD)-negative remission in bone marrow

  day 30

• Proportion of CART84-infused patients undergoing allo-HSCT

  6 months

• Proportion of patients with detectable CART84 cells

  Day 30 and Day 90

• Proportion of enrolled patients for whom a CART84 product can be manufactured and administered as per protocol.

  3 months

Study Arms (1)

GYA01 (CART84)

EXPERIMENTAL

Drug: CART84

Interventions

CART84 DRUG

GYA01 (CART84): autologous cell-based product containing CD3+ T cells transduced with a lentiviral vector expressing an anti-CD84 chimeric antigen receptor (CAR).

GYA01 (CART84)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18 years or older at the time of signing the informed consent.
• Willing and able to give written, informed consent to the current study.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
• Diagnosed with AML or T-ALL with ≥5% blasts in BM and/or PB at screening, without any approved therapeutic alternative and one of the following:
• Primary refractory disease (not achieving CR/CRi after more than two cycles of induction chemotherapy).
• Second relapse or beyond.
• Refractory relapse after at least 1 line of salvage therapy.
• Relapsed or refractory disease after allogeneic transplant provided the CART84 infusion occurs at least 3 months after the stem cell transplant.
• Documentation of CD84 expression on leukemic blasts in the BM and in peripheral blood, or other tissues if blasts are present, as assessed by flow cytometry at screening.
• For T-ALL patients: diagnosed with T-ALL exhibiting a double-negative (CD4- CD8-) immunophenotype, or patients with CD4+ and/or CD8+ T-ALL with no detectable blasts in peripheral blood.
• Availability of an appropriate HSCT donor, either related (haploidentical HLA matching or HLA identical sibling donor) or unrelated, if available within the required timeframe (days 30-90 post-CART84 infusion). If an unrelated donor is selected, it is highly recommended to have an haploidentical HLA matched donor identified and evaluated as a backup.
• For females of childbearing potential (defined as \<24 months after last menstruation or not surgically sterile), a negative serum or urine pregnancy test must be documented at screening, prior to pre-conditioning and confirmed before receiving the first dose of study treatment.
• For females who are not postmenopausal (\<24 months of amenorrhea) or who are not surgically sterile (absence of ovaries and/or uterus), commitment to the use of 2 methods of contraception, comprising of one highly effective method of contraception together with a barrier method, during the treatment period and for at least 12 months after the last dose of study treatment.
• Male participants must agree to use 2 acceptable methods of contraception (one by the patient - usually a barrier method), and one highly effective method by the patient's partner during the treatment period and for at least 12 months after the last dose of study treatment.
• Adequate renal, hepatic, pulmonary, and cardiac function defined as:

You may not qualify if:

• Isolated extramedullary (EM) disease.
• Females who are pregnant or lactating.
• For T-ALL patients: Patients with T-ALL exhibiting CD4+ and/or CD8+ immunophenotypes with detectable blasts in peripheral blood.
• History or presence of clinically relevant CNS pathology, such as epilepsy, paresis, aphasia, stroke within 3 months prior to enrollment, severe brain injuries, dementia, Parkinson's disease, cerebellar disease, organic brain syndrome, uncontrolled mental illness, or psychosis.
• Clinically significant, uncontrolled heart disease (New York Heart Association Class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, sick-sinus syndrome, or electrocardiographic evidence of acute ischemia or Grade 3 conduction system abnormalities, unless the patient has a pacemaker) or a recent (within 12 months) cardiac event.
• Patients with active, life-threatening bleeding.
• Presence of uncontrolled fungal, bacterial, viral, or other infection requiring systemic antimicrobials for management.
• Positive serological testing for human immunodeficiency virus antibody, hepatitis B surface antigen, hepatitis B core antibody (anti-HBc), and hepatitis C virus antibody. Patients who are positive for anti-HBc or hepatitis C antibody may be included if they have a negative PCR test within 6 weeks prior to initial IMP administration.
• History of autoimmune disease (e.g., Crohn's disease, rheumatoid arthritis, systemic lupus) resulting in organ injury or requiring systemic immunosuppression/systemic disease modifying agents within the last 24 months, or any autoimmune disease with CNS involvement.
• History of other malignant neoplasms unless disease-free for at least 12 months (carcinoma in situ, non-melanoma skin cancer, breast or prostate cancer on hormonal therapy allowed).
• Known history of concomitant genetic syndromes such as Fanconi anemia, Schwachman-Diamond syndrome, Kostmann syndrome, or any other known BM failure syndrome.
• Patients who have received a prior stem cell transplant less than 3 months prior to CART84 infusion.
• Active significant (overall Grade ≥II, Seattle criteria) acute graft-versus-host disease (GvHD) or moderate/severe chronic GvHD (NIH consensus criteria) requiring systemic steroids or other immunosuppressants within 4 weeks of consent.
• The following medications are excluded:
• Steroids: Therapeutic doses of corticosteroids (greater than 10 mg daily of prednisone or its equivalent) within 7 days of leukapheresis or 72 hours prior to CART84 administration. However, physiological replacement, topical, and inhaled steroids are permitted.

Sponsors & Collaborators

• Gyala Therapeutics: lead
• Astrum CRO, S.L.: collaborator

Study Sites (2)

Hospital Clínic de Barcelona

Barcelona, Barcelona, 08036, Spain

RECRUITING

Hospital Universitario y Politécnico La Fe

Valencia, Valencia, 46026, Spain

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Leukemia, T-Cell; Precursor T-Cell Lymphoblastic Leukemia-Lymphoma

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Lymphoid; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Precursor Cell Lymphoblastic Leukemia-Lymphoma

Central Study Contacts

Claudio Santos, PhD

CONTACT

+34 684 765 219; claudio.santos@gyalatx.com

Teresa Galera, PhD, MBA

CONTACT

teresa.galera@gyalatx.com

Study Design

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

Model Details: Open-label, single arm, dose escalation Phase I/IIa study to evaluate the safety and efficacy of CART84 in adult patients with R/R AML/T-ALL. Phase I (dose escalation with a standard 3 + 3 patients cohort design). The Maximum Tolerated Dose in Phase I will be the recommended phase 2 dose. The clinical trial will have 7 stages: 1. Screening 2. Leukapheresis 3. Bridging Stage 4. Lymphodepletion 5. Treatment 6. Consolidation 7. Follow-up

Study Record Dates

• First Submitted: March 10, 2026
• First Posted: March 13, 2026
• Study Start: February 4, 2026
• Primary Completion (Estimated): May 31, 2029
• Study Completion (Estimated): February 4, 2031
• Last Updated: March 13, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: SAP, CSR, ANALYTIC CODE
• Time Frame: At the end of the study
• Access Criteria: The documents will be uploaded to this website

Locations

ES (2)