Study of Venetoclax a BCL2 Antagonist for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

  • STATUS
    Recruiting
  • End date
    Apr 30, 2025
  • participants needed
    26
  • sponsor
    Dana-Farber Cancer Institute
Updated on 16 May 2021

Summary

This research study is studying a drug as a possible treatment for BPDCN.

The intervention involved in this study is: Venetoclax

Description

This research study is a Phase I clinical trial, which tests the safety of an investigational drug and also tries to define the safest dose of the investigational drug to use for further studies. "Investigational" means that the drug is being studied.

The FDA (the U.S. Food and Drug Administration) has not approved venetoclax for this specific disease but it has been approved for other uses.

Based on laboratory data where it was found that BPDCN cells die after treatment with Venetoclax, the investigators believe that this drug will be effective in treating patients with BPDCN.

Details
Condition Blastic Plasmacytoid Dendritic Cell Neoplasm
Treatment venetoclax
Clinical Study IdentifierNCT03485547
SponsorDana-Farber Cancer Institute
Last Modified on16 May 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Histologically confirmed diagnosis of blastic plasmacytoid dendritic cell neoplasm (BPDCN) per 2016 WHO criteria
Age > 18 years
In Stage 1 (modified 3+3): BPDCN relapsed after or refractory to at least one prior treatment regimen (hydroxyurea is not considered a prior treatment regimen)
In Stage 2 (expansion)
(A) BPDCN relapsed after or refractory to at least one prior treatment regimen (hydroxyurea is not considered a prior treatment regimen)
\---OR
(B) Treatment-nave BPDCN, and age > 75 years; or treatment-nave BPDCN, and age > 18 years and who decline intensive induction chemotherapy or who are unfit due to co-morbidity or other factors (see APPENDIX A for unfitness definitions)
ECOG performance status 0, 1, or 2
Adequate organ function as defined by
Serum creatinine < 1.5x ULN
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 2.5x ULN
Total bilirubin < 1.5x ULN (if total bilirubin is > 1.5x but < 3x ULN, and thought to be elevated due to Gilbert's disease or the patient's BPDCN, the subject may be eligible but must discuss with the PI)
Ability to understand and the willingness to sign a written informed consent document
Able to adhere to study visit schedule and other protocol requirements including follow-up for survival assessment
Women of child-bearing potential and men enrolled on this protocol must agree to use adequate contraception for the duration of study participation and for 2 months after completion venetoclax administration

Exclusion Criteria

Prior treatment with venetoclax
Received treatment with chemotherapy, radiation, or biologic cancer therapy within 14 days of first protocol treatment. Prior and concurrent hydroxyurea is permitted during the first cycle
Hematopoietic stem cell transplantation (HSCT) within 60 days of first protocol treatment, or receipt of immunosuppressive therapy for graft-versus-host disease treatment or prophylaxis within 14 days of first protocol treatment, or active graft-versus-host-disease
Known active CNS involvement by BPDCN
Known positive status for HIV infection; known active hepatitis B or hepatitis C infection
Clinically significant cardiopulmonary disease including uncontrolled or 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
Patients with known active advanced malignant solid tumors are excluded (except for basal or squamous skin cancers, or carcinomas in situ). Patients with additional hematologic malignancies that require treatment are excluded
Patients with gastrointestinal (GI) tract disease causing the inability to take oral medication, malabsorption syndrome, a requirement for intravenous (IV) alimentation, prior surgical procedures affecting absorption, uncontrolled inflammatory GI disease (e.g., Crohn's disease, ulcerative colitis)
Pregnant women are excluded from this study because there is an unknown but potential risk for adverse events in the developing fetus with venetoclax (negative urine or serum pregnancy test required within 14 days of Cycle 1, Day 1). Because nursing infants have unknown potential for adverse events secondary to treatment of the mother, breastfeeding should be discontinued if the mother is treated with venetoclax
Infection is a common feature of BPDCN and acute leukemias. Patients with active infection are permitted to enroll provided that the infection is controlled (patients on IV or PO antibiotics are allowed). Patients with uncontrolled infection shall not be enrolled until infection is treated and brought under control
Subject has received the following within 7 days prior to the initiation of study
treatment
Strong and moderate CYP3A inducers
Strong and moderate CYP3A inhibitors
Subject has consumed grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges), or Star fruit within 3 days prior to the initiation of study treatment
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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