CAR-T Cell Therapy for ALL
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age5-50
SponsorDr. Zaineb Akram
About this trial
Acute lymphoblastic leukemia (ALL) is the most common malignancy in children and the second most frequent acute leukemia in adults. B-cell ALL constitutes approximately 85% of all ALL diagnoses. In Pakistan, ALL represents the most prevalent haematological malignancy presenting to tertiary centres, with AFBMTC receiving the largest national referral volume for haematological malignancies and transplantation.
First-line combination chemotherapy achieves complete remission (CR) in \>95% of paediatric patients; however, 15-20% relapse. Outcomes following first relapse are substantially inferior: second-line salvage chemotherapy achieves CR2 in 30-50% of patients, and long-term event-free survival (EFS) after conventional chemotherapy alone is \<10%. Outcomes in adult ALL are even more dismal, with OS at 5 years below 40% even in first CR without allogeneic transplant.
Patients with primary refractory ALL or multiply relapsed ALL have an unmet medical need for novel therapeutic approaches. The classical paradigm of chemotherapy followed by allogeneic haematopoietic stem cell transplantation (allo-HSCT) is limited by donor availability, conditioning-related mortality, and inability to achieve remission before transplant.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Inclusion Criteria
1. Age ≥5 years and ≤50 years at the time of consent
a. Second or subsequent bone marrow relapse (BM ≥2nd relapse)
b. Any BM relapse following prior allogeneic HSCT, provided ≥6 months have elapsed from SCT to planned CAR-T infusion
Disqualifiers
1. Isolated extra-medullary (CNS-only or testicular-only) disease relapse without bone marrow involvement
2. Active CNS involvement by ALL, defined as CNS-3 status per NCCN criteria (CSF blasts on cytospin, cranial nerve palsy, or brain parenchymal disease) at time of screening. Patients with prior CNS disease that has been effectively treated and cleared are eligible
3. Burkitt's lymphoma/leukemia (mature B-ALL with sIg positive, FAB L3 morphology and/or MYC translocation)
4. T-cell ALL or ambiguous lineage leukemia
Trial design
Design model
Single group
Treatments tested in this trial
CAR-T cell infusion
Biological/VaccineCAR-T Cell infusion therapy will be given
Treatment groups
10 Participants
are divided into 1 treatment groupGroup A: CAR-T armOther 1 intervention
Trial outcomes
Primary outcomes
1
To evaluate the safety and feasibility of autologous anti-CD19 CAR-T cell therapy manufactured at AFBMTC using the BIOCCUS lentiviral vector and CELLBRI automated expansion system in patients aged 5-50 years with relapsed or refractory B-cell ALL.
Primary Safety Incidence/grade of CRS (ASTCT) Day 0-28
Time frame
1 year after infusion of CAR-TSponsors and contacts
Click on the lead sponsor to view all of their trials.
Dr. Zaineb Akram
Lead sponsor
National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Sponsor institution
Source ClinicalTrials.gov