A phase 1/2 study to assess the safety and efficacy of MB-102 in patients with relapsed or refractory BPDCN
The Phase 1 portion of the study will determine the maximum tolerated dose of MB-102. The Phase 2 portion of the trial will evaluate the efficacy of MB-102 in relapsed or refractory BPDCN.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
3
The study drug, MB-102 consists of adoptively transferred T cells that are genetically modified using a self-inactivating (SIN) lentiviral vector to express a CD123-specific, CD28-costimulatory chimeric antigen receptor (CAR) as well as a truncated human epidermal growth factor receptor (EGFRt) (CD123.CD28.CD3ζ.EGFRt+T cells) derived from autologous leukapheresis which is administered after a lymphodepletion regimen. Single dose of MB-102 up to 600 x 10 6 CART-T+ cells (Day 0) as defined by Phase 1 will be administered.
Fludarabine 30 mg/m2/day IV (3 days) on days -5, -4, and -3 * A 20% dose reduction (24 mg/m2/day IV (3 days) on days -5, -4, and -3) is required for patients with moderately impaired renal function (creatine clearance ≤ 70 mL/min).
City of Hope Medical Center
Duarte, California, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, United States
Duke University
Durham, North Carolina, United States
MD Anderson Cancer Center
Houston, Texas, United States
Phase 1: Safety and Tolerability as measured by the number of patients with treatment related adverse events
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 in Phase 1
Time frame: 28 Days
Phase 1: Maximum Tolerated Dose (MTD) and recommended Phase 2 dose
To determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose of MB-102
Time frame: 28 Days
Phase 2: Response Rate of patients with BPDCN
Relapsed or refractory Blastic Plasmacytoid Dendritic Cell Neoplasm is measured by a response rate which consists of Complete Response and clinical Complete Response and Complete Response with incomplete hematologic recovery (CR + CRc + CRi) at day 28 post infusion
Time frame: up to 3 years
Phase 2: BPDCN - DOR
Duration of Response
Time frame: up to 3 years
Phase 2: BPDCN - PFS
Progression-Free Survival
Time frame: up to 3 years
Phase 2: BPDCN - OS
overall survival
Time frame: up to 3 years
Phase 2: BPDCN - MRD
CR MRD- Response Rate for patients with CR and CRi
Time frame: up to 3 years
Phase 2 - Adverse events
Incidence of treatment-emergent AEs (TEAEs), including SAEs, therapy-related AEs or death.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Cyclophosphamide 300 - 500 mg/m2/day IV (3 days) on days -5, -4, and -3
Time frame: up to 3 years
Phase 2 -Change from Baseline in the European Organization for Research and Treatment (EORTC) QLQ-C 30 Version 3.0.
The European Organization for Research and Treatment (EORTC) QLQ-C 30 Version 3.0 is an integrated, modular approach for evaluating the quality of life of patients participating in international clinical trials. The questionnaire is designed to measure cancer patients' physical, psychological and social functions. The questionnaire is composed of 5 multi-item scales (physical, role, social, emotional and cognitive functioning) and 9 single items (pain, fatigue, financial impact, appetite loss, nausea/vomiting, diarrhea, constipation, sleep disturbance and quality of life). It utilizes a four-point scales for the first 28 questions which are coded with response categories as "Not at all", "A little", "Quite a bit" and "Very much.". the final two question consist of an overall physical condition questions which have employed a 7-point response scale where the higher number indicates a better overall health.
Time frame: up to 3 years
Phase 2 - Change from Baseline in the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Version 4.0.
The Functional Assessment of Cancer treatment for cancer, and a transplant-specific module, bone marrow transplant (BMT) concerns, that addresses disease and treatment-related questions specific to BMT. It utilizes a 5 point scale assessing physical, social, emotional, functional and other well-being concerns. Response categories are coded as "Not at all", "A little bit", "Somewhat", "Quite a bit" and "Very much.".
Time frame: up to 3 years
Phase 2 - Number of patients showing evidence of replication competent lentivirus
To confirm the absence of replication competent lentivirus
Time frame: up to 3 years