Trial to Evaluate the Safety and Efficacy of MB-102 in Patients With BPDCN.
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|ClinicalTrials.gov Identifier: NCT04109482|
Recruitment Status : Recruiting
First Posted : September 30, 2019
Last Update Posted : February 15, 2021
|Condition or disease||Intervention/treatment||Phase|
|Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)||Biological: MB-102 Drug: Fludarabine Drug: Cyclophosphamide||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||44 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1/2, Open Label, Multicenter Trial to Assess the Safety and Efficacy of MB-102 in Patients With Relapsed or Refractory Blastic Plasmacytoid Dendritic Cell Neoplasm|
|Actual Study Start Date :||February 17, 2020|
|Estimated Primary Completion Date :||March 2023|
|Estimated Study Completion Date :||April 2026|
Experimental: Relapsed or Refractory BPDCN
Treatment with MB-102.
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.
Other Name: CD123 CAR-T
Fludarabine 30 mg/m2/day IV (3 days) on days -5, -4, and -3
Other Name: Fludara
Cyclophosphamide 300 - 500 mg/m2/day IV (3 days) on days -5, -4, and -3
Other Name: Cytoxan
- Phase 1: Safety and Tolerability as measured by the number of patients with treatment related adverse events [ Time Frame: 28 Days ]Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 in Phase 1
- Phase 1: Maximum Tolerated Dose (MTD) and recommended Phase 2 dose [ Time Frame: 28 Days ]To determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose of MB-102
- Phase 2: Response Rate of patients with BPDCN [ Time Frame: up to 3 years ]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
- Phase 2: BPDCN - DOR [ Time Frame: up to 3 years ]Duration of Response
- Phase 2: BPDCN - PFS [ Time Frame: up to 3 years ]Progression-Free Survival
- Phase 2: BPDCN - OS [ Time Frame: up to 3 years ]overall survival
- Phase 2: BPDCN - MRD [ Time Frame: up to 3 years ]CR MRD- Response Rate for patients with CR and CRi
- Phase 2 - Adverse events [ Time Frame: up to 3 years ]Incidence of treatment-emergent AEs (TEAEs), including SAEs, therapy-related AEs or death.
- Phase 2 -Change from Baseline in the European Organization for Research and Treatment (EORTC) QLQ-C 30 Version 3.0. [ Time Frame: up to 3 years ]
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.
- Phase 2 - Change from Baseline in the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Version 4.0. [ Time Frame: up to 3 years ]
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.".
- Phase 2 - Number of patients showing evidence of replication competent lentivirus [ Time Frame: up to 3 years ]To confirm the absence of replication competent lentivirus
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04109482
|Contact: Kerry Bironfirstname.lastname@example.org|
|United States, California|
|City of Hope Medical Center||Recruiting|
|Duarte, California, United States, 91010|
|Contact: Lihua E Budde, MD 626-218-1133 email@example.com|
|United States, Massachusetts|
|Dana-Farber Cancer Institute||Recruiting|
|Boston, Massachusetts, United States, 02215|
|Contact: Michael Rocchio MichaelJ_Rocchio@DFCI.Harvard.edu|
|Principal Investigator: Marlise Luskin, MD|
|United States, North Carolina|
|Durham, North Carolina, United States, 27710|
|Contact: Hematology Malignancy Research Nurse Office 919-681-4769|
|Principal Investigator: David Rizzieri, MD|
|United States, Texas|
|MD Anderson Cancer Center||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Lenart Vega 713-563-4754 firstname.lastname@example.org|
|Principal Investigator: Naveen Pemmaraju, MD|
|Principal Investigator:||Lihua E Budde, MD||City of Hope Medical Center|