Venetoclax, SL-401, and Chemotherapy for the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm

Launched by M.D. ANDERSON CANCER CENTER · Dec 30, 2019

Keywords

ClinConnect Summary

This clinical trial is exploring a new treatment approach for a rare type of cancer called blastic plasmacytoid dendritic cell neoplasm (BPDCN). The study is testing how well three treatments work together: venetoclax, a medication that may help stop cancer cells from growing; SL-401, which targets cancer cells in a special way to deliver a toxic agent that kills them; and traditional chemotherapy drugs, which have various methods to attack cancer cells. The goal is to see if combining these treatments can be more effective for patients with BPDCN.

To participate in this trial, patients must be at least 18 years old and have a confirmed diagnosis of BPDCN. They may be newly diagnosed or have already received treatment that was not successful. Participants should also be able to understand the study and agree to follow the required visits and assessments. It’s important to note that certain health conditions, like active heart disease or infections, may prevent someone from joining. If eligible, participants can expect to receive the new treatment combination and be closely monitored by the healthcare team throughout the trial.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Treatment naïve or relapsed refractory patients with histologically confirmed diagnosis of blastic plasmacytoid dendritic cell neoplasm (BPDCN) per 2016 WHO criteria
• 2. Front line participants may have received emergent chemotherapy prior to study enrollment:
• • 1. One prior cycle of SL-401, or other BPDCN-directed therapy, will be allowed prior to entering the study.
• • 2. Prior or concomitant doses of ARA-C (cytarabine) or Hydroxyurea are allowed on before or during the study for proliferative disease due to BPDCN.
• • 3. Relapsed/refractory participants may have received at least one prior cycle of therapy.
• • 4. Age ≥ 18 years
• • 5. ECOG performance status 0, 1, or 2 (see APPENDIX B)
• 6. Adequate organ function as defined by:
• • Albumin ≥ 3.2 g/dL (in the absence of receipt of intravenous albumin in the previous 72 hours)
• • 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)
• • 7. Ability to understand and the willingness to sign a written informed consent document.
• • 8. Able to adhere to study visit schedule and other protocol requirements including follow-up for survival assessment
• 9. 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 VEN administration. Acceptable birth control methods allowed to be used while on study include:
• • Birth control pills or injections
• • Intrauterine devices (IUDs)
• • Double-barrier methods for example condom in combination with spermicide. Males should not donate sperm while on study and for at least 8 weeks after the last dose of SL-401.
• • 10. Left ventricular ejection fraction ≥ institutional lower limit of normal by MUGA scan or echocardiogram within 30 days of first protocol treatment.
• Exclusion Criteria:
• • 1. Participants is pregnant or breastfeeding
• • 2. Known active hepatitis B or C infection, or known seropositivity for human immunodeficiency virus (HIV)
• • 3. Major surgery or radiation therapy within 14 days prior to the first study dose
• • 4. Symptomatic or untreated leptomeningeal disease or spinal cord compression
• • 5. Participants with active heart disease (New York Heart Association (NYHA) class 3-4 as assessed by history and physical examination, unstable angina/stroke/myocardial infarction within the last 6 months)
• • 6. Malabsorption syndrome or other conditions that preclude enteral route of administration
• • 7. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that in the opinion of the investigator may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and/or would make the participants inappropriate for enrollment into this study