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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03485547
Recruitment Status : Active, not recruiting
First Posted : April 2, 2018
Last Update Posted : February 13, 2024
Sponsor:
Collaborator:
AbbVie
Information provided by (Responsible Party):
Andrew Lane, Dana-Farber Cancer Institute

Study Description
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Brief Summary:

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

The intervention involved in this study is: Venetoclax


Condition or disease Intervention/treatment Phase
Blastic Plasmacytoid Dendritic Cell Neoplasm Drug: Venetoclax Phase 1

Detailed 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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 1 Study of Venetoclax, a BCL2 Antagonist, for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
Actual Study Start Date : August 23, 2018
Estimated Primary Completion Date : January 19, 2025
Estimated Study Completion Date : January 19, 2025

Resource links provided by the National Library of Medicine

Drug Information available for: Venetoclax

Arms and Interventions
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Arm Intervention/treatment
Experimental: Venetoclax
  • Venetoclax is administered on a daily basis orally.
  • The investigators will use a modified 3+3 with a de-escalation dose level design to establish the appropriate and tolerable dose of venetoclax.
 Drug: Venetoclax
VENCLEXTA targets BCL-2 in order to help restore the process of apoptosis. Through apoptosis, your body allows cancer cells and normal cells to self-destruct
Other Name: Venclexta


Outcome Measures
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Primary Outcome Measures :
  1. Dose Limiting Toxicity [ Time Frame: 2 years ]

Secondary Outcome Measures :
  1. Overall Response Rate [ Time Frame: 2 years ]
  2. Complete Response [ Time Frame: 2 years ]
  3. Partial Response [ Time Frame: 2 years ]
  4. Overall Survival [ Time Frame: 2 years ]
  5. Progression free survival [ Time Frame: 2 years ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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-naïve BPDCN, and age > 75 years; or treatment-naïve 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.
Contacts and Locations
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Information from the National Library of Medicine

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): NCT03485547


Locations
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United States, Massachusetts
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02215
United States, Texas
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
Dana-Farber Cancer Institute
AbbVie
Investigators
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Principal Investigator: Andrew Lane, MD Dana-Farber Cancer Institute
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Andrew Lane, Principal Investigator, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT03485547    
Other Study ID Numbers: 18-045
First Posted: April 2, 2018    Key Record Dates
Last Update Posted: February 13, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Andrew Lane, Dana-Farber Cancer Institute:
Blastic Plasmacytoid Dendritic Cell Neoplasm
BPDCN
Additional relevant MeSH terms:
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Neoplasms
Venetoclax
Antineoplastic Agents