A Multi-Center Study Evaluating KTE-C19 in Pediatric and Adolescent Subjects With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia
Describes the nature of a clinical study. Types include:
- Observational study — observes people and measures outcomes without affecting results.
- Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
- Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.
- Rochester, Minnesota: 16-005582
NCT ID: NCT02625480
Sponsor Protocol Number: KTE-C19-104
About this study
This is a single arm, open-label, multi-center, phase 1/2 study, to determine the safety and efficacy of KTE-C19, an autologous anti-CD19 chimeric antigen receptor (CAR)-positive T cell therapy, in relapsed/refractory B-precursor acute lymphoblastic leukemia (ALL) in pediatric or adolescent subjects.
Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.See eligibility criteria
- Relapsed or refractory B-precursor ALL defined as one of the following:
- Primary refractory disease
- Relapsed or refractory disease after first or later salvage therapy
- Relapsed or refractory disease after allogeneic transplant provided subject is at least 100 days from stem cell transplant at the time of enrollment
- Morphological disease in the bone marrow (≥ 5% blasts)
- Subjects with Ph+ disease are eligible if they are intolerant to tyrosine kinase inhibitor (TKI) therapy, or if they have relapsed/refractory disease despite treatment with at least 2 different TKIs
- Ages 2 to 21 at the time of Assent or Consent per IRB guidelines
- Lansky (age < 16 years at the time of assent/consent) or Karnofsky (age ≥ 16 years at the time of assent/consent) performance status ≥ 80 at screening
- Adequate renal, hepatic, pulmonary and cardiac function defined as:
- Creatinine clearance ≥ 60 cc/min
- Serum ALT/AST ≤ 2.5 x ULN
- Total bilirubin ≤ 1.5 x ULN
- Cardiac ejection fraction ≥ 50% and no clinically significant ECG findings
- Baseline oxygen saturation > 92% on room air
- Diagnosis of Burkitt's leukemia/lymphoma according to WHO classification or chronic myelogenous leukemia lymphoid blast crisis
- History of malignancy other than non-melanoma skin cancer or carcinoma in situ (e.g. cervix, bladder, breast) unless disease free for at least 3 years
- Presence of CNS-3 disease and CNS-2 disease with neurological changes
- History of concomitant genetic syndrome such as Fanconi anemia, Kostmann syndrome, Shwachman-Diamond syndrome or any other known bone marrow failure syndrome
- History of myocardial infarction, cardiac angioplasty or stenting, unstable angina, or other clinically significant cardiac disease within 12 months of enrollment
- History of symptomatic deep vein thrombosis or pulmonary embolism within 6 months of enrollment.
- Primary immunodeficiency
- Known infection with HIV, hepatitis B (HBsAg positive) or hepatitis C virus (anti-HCV positive)
- Presence of fungal, bacterial, viral, or other infection that is uncontrolled or requiring IV antimicrobials for management.
- Prior medication:
- Prior CD19 directed therapy, including CAR+ T cell, BiTE, and antibody drug conjugate (ADC), with the exception of subjects who received KTE-C19 in this study and are eligible for re-treatment
- Treatment with alemtuzumab within 6 months prior to leukapheresis, or treatment with clofarabine or cladribine within 3 months prior to leukapheresis
- Donor lymphocyte infusion (DLI) within 28 days prior to enrollment
- Any drug used for GVHD within 4 weeks prior to enrollment
- Acute GVHD grade II-IV by Glucksberg criteria or severity B-D by IBMTR index; acute or chronic GVHD requiring systemic treatment within 4 weeks prior to enrollment
- Live vaccine ≤ 6 weeks prior to start of conditioning regimen
- Women of child-bearing potential who are pregnant or breastfeeding because of the potentially dangerous effects of the preparative chemotherapy on the fetus or infant. Females who have undergone surgical sterilization are not considered to be of childbearing potential
- Subjects of both genders of child-bearing potential who are not willing to practice birth control from the time of consent through 6 months after the completion of KTE-C19
Participating Mayo Clinic locations
Study statuses change often. Please contact us for help.
|Mayo Clinic Location
Mayo Clinic principal investigator
Richard Bram, M.D., Ph.D.
Open for enrollment
Cancer Center Clinical Trials Referral Office