Tagraxofusp in Treating Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm After Stem Cell Transplant

NCT04317781 | Terminated Phase 2 Results FDA Drug

• 2 other identifiers: 2018-0646NCI-2019-03832
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

This phase II trial studies the side effects of tagraxofusp in treating patients with blastic plasmacytoid dendritic cell neoplasm after stem cell transplant. Tagraxofusp is a type of immunotoxin that is made by linking a protein called IL-3 to a toxic substance. Tagraxofusp may help find cancer cells that express IL-3 and kill them without harming normal cells.

Conditions

Blastic Plasmacytoid Dendritic Cell Neoplasm

Outcome Measures

Primary Outcomes (1)

• Number of Participants That Received Planned Tagraxofusp Post Transplant

  Participants completed at least 75% of planned tagraxofusp doses in at least 4 cycles of therapy.

  Up to 1 year

Secondary Outcomes (2)

• Progression Free Survival (PFS)

  From treatment start date to date of disease progression or death, assessed up to 1 year

• Overall Survival (OS)

  From treatment start date to death, assessed up to 1 year

Study Arms (1)

Treatment (tagraxofusp-erzs)

EXPERIMENTAL

Within day 45 and 180 after stem cell transplant, patients receive tagraxofusp-erzs IV over 15 minutes on days 1-3 of cycles 1-4 and days 1-2 of subsequent cycles. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.

Biological: Tagraxofusp-erzs

Interventions

Tagraxofusp-erzs BIOLOGICAL

Given IV

Also known as: Diphtheria Toxin(388)-Interleukin-3 Fusion Protein, DT(388)-IL3 Fusion Protein, DT388IL3 fusion protein, Elzonris, IL3R-targeting Fusion Protein SL-401, SL-401, Tagraxofusp, Tagraxofusp ERZS

Treatment (tagraxofusp-erzs)

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Eligible patients will be aged \>= 18 years. Pediatric patients age 2 years and older will be considered on a case by case basis.
• Diagnosis of blastic plasmacytoid dendritic cell neoplasm (BPDCN) according to World Health Organization (WHO) classification or confirmed by hematopathology
• The patients must be in partial response or better
• \> 30 days post-transplant without active or chronic infections
• Karnofsky performance status \>= 60%; Lansky \>= 60
• Left ventricular ejection fraction (LVEF) \>= institutional lower limit of normal by multigated acquisition (MUGA) scan or echocardiogram within 30 days of first protocol treatment
• Diffusion capacity of the lung for carbon monoxide (DLCO) \> 40% of predicted value (corrected for hemoglobin) within 3 months of registration
• Forced expiratory volume in 1 second (FEV1) \> 40% of predicted value within 3 months of registration
• Forced vital capacity (FVC) \> 40% of predicted value within 3 months of registration
• Serum creatinine =\< 1.5 mg/dL (133 mmol/L)
• Serum albumin \>= 3.2 g/dL (or \>= 32 g/L) without IV albumin within the previous 72 hours
• Bilirubin =\< 1.5 x the upper limit of normal (\[ULN\] except patients with Gilbert syndrome in whom bilirubin level of \> 1.5 x ULN will be allowed)
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =\< 2.5 times ULN
• Hemoglobin \>= 8 g/dL with or without transfusion in the last 7 days
• Absolute neutrophil count (ANC) \>= 1000 without granulocyte colony stimulating factor (GCSF) or granulocyte-macrophage colony-stimulating factor (GMCSF) in the last 2 weeks prior to screening

You may not qualify if:

• The patient has persistent clinically significant non-hematologic toxicities \>= grade 2 (excluding alopecia, nausea, and fatigue)
• Evidence of central nervous system (CNS) involvement
• Uncontrolled and active pulmonary disease
• Requirement for oxygen treatment
• Receiving chemotherapy, radiotherapy or other anti-cancer therapy within 14 days of first dose of study drug. There must be at least a 6-week interval from the last immunotherapy therapy
• Uncontrolled infection
• Human immunodeficiency virus (HIV)/hepatitis B and/or C
• Any history of invasive malignancy in the last 2 years excluding any malignancy such as cervical cancer or skin cancer (excluding melanoma) that is considered cured at the time of screening
• Pregnant or breast-feeding woman
• Patient has uncontrolled intercurrent illness or medical/psychiatric condition that would limit compliance with study requirements or that would in the investigator's opinion place the patient at an unacceptably high risk for toxicities
• Clinical significant cardiopulmonary disease including uncontrolled or New York Heart Association (NYHA) class 3 or 4 congestive heart failure, uncontrolled angina, uncontrolled hypertension, uncontrolled arrhythmia, myocardial infarction or stroke within 6 months of first protocol treatment or corrected QT (QTc) \> 480 ms

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Publications (1)

• Wang SY, Thomassen K, Kurch L, Opitz S, Franke GN, Bach E, Platzbecker U, Kayser S. Combination of Tagraxofusp and Azacitidine Is an Effective Option for Relapsed Blastic Plasmacytoid Dendritic Cell Neoplasm After Allogeneic Hematopoietic Stem-Cell Transplantation. Clin Lymphoma Myeloma Leuk. 2021 Jul;21(7):e579-e582. doi: 10.1016/j.clml.2021.02.008. Epub 2021 Mar 2. No abstract available.

  PMID: 33795208 DERIVED

Related Links

• MD Anderson Cancer Center

MeSH Terms

Conditions

Blastic Plasmacytoid Dendritic Cell Neoplasm

Interventions

tagraxofusp

Condition Hierarchy (Ancestors)

Histiocytic Disorders, Malignant; Neoplasms by Histologic Type; Neoplasms; Leukemia; Lymphoma; Hematologic Neoplasms; Neoplasms by Site; Skin Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Limitations and Caveats

The protocol was terminated early due to slow participant accrual and the sponsor not willing to provide the study drug. Stemline is the funder, not the sponsor.

Results Point of Contact

• Title: Qaiser Bashir, M.D. / Stem Cell Transplantation and Cellular Therapy Department
• Organization: The University of Texas MD Anderson Cancer Center

qbashir@mdanderson.org

713-794-4422

Study Officials

• Qaiser Bashir, MS

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 19, 2020
• First Posted: March 23, 2020
• Study Start: March 27, 2020
• Primary Completion: July 11, 2023
• Study Completion: July 11, 2023
• Last Updated: July 31, 2024
• Results First Posted: July 16, 2024

Record last verified: 2024-07

Locations

US (1)