Status:
TERMINATED
Trial to Evaluate the Safety and Efficacy of MB-102 in Patients With BPDCN.
Lead Sponsor:
Mustang Bio
Conditions:
Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
A phase 1/2 study to assess the safety and efficacy of MB-102 in patients with relapsed or refractory BPDCN
Detailed Description
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.
Eligibility Criteria
Inclusion
- Blastic Plasmacytoid Dendritic Cell Neoplasm
- Patients with a diagnosis of BPDCN according to WHO classification (Arber et al., 2016) confirmed by hematopathology and histological/cytological evidence of BPDCN in the peripheral blood, bone marrow, spleen, lymph nodes, skin and/or other sites who have failed one prior therapy.
- General Inclusion Criteria
- Male and female patients ≥ 18 years of age at the time of consent.
- Written informed consent in accordance with federal, local, and institutional guidelines.
- Must be able to adhere to the study visit schedule and other protocol requirements.
- Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
- Meet the following laboratory criteria:
- Absolute lymphocyte count (ALC) \> 100/mm3
- ALT/SGPT and AST/SGOT \< 2.5x the upper limit of normal (ULN) unless due to underlying disease state
- Calculated creatinine clearance ≥ 45.0 mL/min as estimated by Cockcroft Gault and dialysis independent
- Total bilirubin ≤ 3.0 mg/dL
- Patients with Gilbert's Syndrome must have a total bilirubin \< 5.0 mg/dL.
- Serum albumin ≥ 3.2 g/dL
- Cardiac ejection fraction ≥ 45%, with no evidence of pericardial effusion as determined by an echocardiogram (ECHO) or if not available, a multigated acquisition scan (MUGA).
- Females participants of childbearing potential must have a negative serum test.
- Patients must agree to use a highly effective method of contraception if procreative potential exists from the start of the study until one year after the completion of lymphodepletion for females and 4 months after completion of lymphodepletion for males.
- Patients with a previously treated malignancy if treatment of that malignancy was completed greater than 2 years before screening and the patient has no evidence of disease at the time of screening.
- Patients who have previously undergone allogenic or autologous bone marrow transplants are allowed.
- Centrally confirmed CD-123 positivity on the bone marrow, or for patients without bone marrow involvement local pathology assessments within 28 days from Screening, showing evidence of CD-123 positivity of skin/lymph node biopsy.
Exclusion
- Patients with a corticosteroid dependence on doses greater than physiological replacement i.e., prednisone no more than 7.5 mg/day or hydrocortisone less than 12mg/m2/day.
- Contraindication or hypersensitivity to fludarabine or cyclophosphamide.
- Hypersensitivity or known history of allergic reactions attributed to tocilizumab, Cetuximab, or other anti-EGFR -monoclonal antibodies.
- Immunotherapy treatments within 28 days prior to leukapheresis.
- Previous treatment with anti-CD123 CAR-T treatment.
- Previous treatment with non-CAR-T anti-CD123 agents is allowed e.g. tagraxofusp-erzs.
- Previous treatment with any other antileukemic or investigational agent within 7 days of leukapheresis.
- Hydroxyurea is allowed up to 3 days prior to leukapheresis.
- Patients with history or active seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease or any autoimmune disease with CNS involvement.
- Patients with known CNS leukemic involvement that are refractory to intrathecal chemotherapy and/or cranio-spinal radiation that have NOT been effectively treated to complete remission (defined as \< 5 WBC/mm3 and no blasts in CSF).
- Patients with active Graft versus Host Disease (GVHD).
- Acute active infection
- Patients being administered prophylactic antibiotics, antivirals, or antifungals are permitted.
- Patients who have any form of primary immunodeficiency, such as severe combined immunodeficiency disease, human immunodeficiency virus (HIV), or acquired immune deficiency syndrome (AIDS).
- Active infection with hepatitis B or C.
- Patients requiring supplemental oxygen or mechanical ventilation or oxygen saturation \< 92% on room air.
- Patients with an oxygen saturation \< 92%, a pulmonary function test with a result of Diffusing capacity of the lungs for carbon monoxide (DLCO) of ≥ 40% of predicted and a forced expiratory volume in one second (FEV1) \> 45% predicted will be accepted.
- Patients with decompensated hepatic cirrhosis/liver failure.
- Pregnant or lactating females.
- Any other clinically significant medical disease or condition that, in the investigator's opinion, may interfere with protocol adherence or a patient's ability to give informed consent.
Key Trial Info
Start Date :
February 17 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 17 2023
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT04109482
Start Date
February 17 2020
End Date
May 17 2023
Last Update
July 22 2024
Active Locations (4)
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1
City of Hope Medical Center
Duarte, California, United States, 91010
2
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
3
Duke University
Durham, North Carolina, United States, 27710
4
MD Anderson Cancer Center
Houston, Texas, United States, 77030