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A Study of Venetoclax in Combination With Cobimetinib and Venetoclax in Combination With Idasanutlin in Patients With Relapsed or Refractory Acute Myeloid Leukemia Who Are Not Eligible for Cytotoxic Therapy

Phase 1
Completed
Conditions
Leukemia, Myeloid, Acute
Interventions
Registration Number
NCT02670044
Lead Sponsor
Hoffmann-La Roche
Brief Summary

The primary objective for this study is to assess the safety and tolerability as well as preliminary efficacy of venetoclax in combination with cobimetinib, and venetoclax in combination with idasanutlin in patients with relapsed or refractory acute myeloid leukemia (R/R) AML who are not eligible for cytotoxic therapy.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
88
Inclusion Criteria
  • Histological confirmation of relapsed or refractory AML after prior anti-leukemic therapy by WHO classification

  • Ineligible for cytotoxic therapy defined by the following:

    a. Age (>/=) 75 years or b. age 18- 74 years with at least one of the following comorbidities: i. Eastern Cooperative Oncology Group (ECOG) Performance Status of 2 or 3 ii. Cardiac history of congestive heart failure requiring treatment or ejection fraction (</=) 50% or chronic stable angina iii. Diffusing capacity of the lungs for carbon monoxide (</=) 65% or forced expiratory volume in the first second of expiration (</=) 65% iv. Creatinine clearance (>/=) 30 mL/min to< 45 mL/min v. any other comorbidity that the physician judges to be incompatible with intensive chemotherapy must be reviewed and approved by the Medical Monitor before screening and study enrollment.

  • Life expectancy of at least 12 weeks

  • Eastern Cooperative Oncology Group (ECOG) Performance Status 0-2

  • Adequate liver and renal function

Exclusion Criteria
  • Patients with acute promyelocytic leukemia (French-American-British [FAB] class M3 AML)
  • Known active central nervous system (CNS) involvement with AML at study entry
  • ECOG Performance Status (>/=) 3 in patients who are (>/=) 75 years old or ECOG Performance Status of 4, regardless of age
  • Prior exposure to Bcl-2 inhibitors, murine double minute 2 (MDM2) antagonists or prior exposure to experimental treatment targeting Raf, mitogen-activated protein kinase (MEK), or the mitogen-activated protein kinase (MAPK) RAS/RAF/MEK/ERK MAPK pathway
  • Positive for hepatitis C virus (HCV), hepatitis B surface antigen (HBsAg) and known history of HIV, malignancy, active infection and cardiovascular diseases (CVs)
  • Received strong cytochrome (CYP) 3A inhibitors, moderate CYP3A inhibitors, strong CYP3A inducers and moderate CYP3A inducers within 7 days prior to initiation of study treatment
  • History of symptomatic Clostridium difficile infection within 1 month prior to dosing

Additional arm specific exclusion criteria:

Dose Escalation Arm A (Venetoclax and Cobimetinib):

  • History or evidence of retinal pathology on ophthalmologic examination that is considered a risk factor for neurosensory retinal detachment/central serous chorioretinopathy (CSCR), retinal vein occlusion (RVO), or neovascular macular degeneration
  • Left ventricular ejection fraction (LVEF) below institutional lower limit of normal (LLN) or below 50%, whichever is lower

Arm B (Venetoclax and Idasanutlin):

  • Received the following within 7 days prior to the initiation of study treatment: Strong CYP2C8 inhibitors or CYP2C8 substrates, OATP1B1/3 substrates
  • Received the following within 14 days prior to the initiation of study treatment: Strong CYP2C8 inducers
  • History of liver cirrhosis by radiologic, clinical or laboratory data, or biopsy despite normal liver function tests
  • Received treatment with oral or parenteral anticoagulants/anti-platelet agents within 7 days prior to initiation of study treatment.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Dose Escalation: Arm A (Venetoclax 800mg + Cobi 40mg)VenetoclaxParticipants received Venetoclax 800mg daily on Days 1-28 of each 28 day treatment cycle and Cobimetinib 40mg daily on Days 1-21 of each 28-day treatment cycle.
Dose Escalation: Arm A (Venetoclax 400mg + Cobi 40mg)CobimetinibParticipants received Venetoclax 400mg daily on Days 1-28 of each 28 day treatment cycle and Cobimetinib 40mg daily on Days 1-21 of each 28-day treatment cycle.
Dose Escalation: Arm A (Venetoclax 400mg + Cobi 40mg)VenetoclaxParticipants received Venetoclax 400mg daily on Days 1-28 of each 28 day treatment cycle and Cobimetinib 40mg daily on Days 1-21 of each 28-day treatment cycle.
Dose Escalation: Arm A (Venetoclax 600mg + Cobi 40mg)CobimetinibParticipants received Venetoclax 600mg daily on Days 1-28 of each 28 day treatment cycle and Cobimetinib 40mg daily on Days 1-21 of each 28-day treatment cycle.
Dose Escalation: Arm A (Venetoclax 600mg + Cobi 40mg)VenetoclaxParticipants received Venetoclax 600mg daily on Days 1-28 of each 28 day treatment cycle and Cobimetinib 40mg daily on Days 1-21 of each 28-day treatment cycle.
Dose Escalation: Arm A (Venetoclax 800mg + Cobi 40mg)CobimetinibParticipants received Venetoclax 800mg daily on Days 1-28 of each 28 day treatment cycle and Cobimetinib 40mg daily on Days 1-21 of each 28-day treatment cycle.
Dose Escalation: Arm A (Venetoclax 400mg + Cobi 60mg)CobimetinibParticipants received Venetoclax 400mg daily on Days 1-28 of each 28 day treatment cycle and Cobimetinib 60mg daily on Days 1-21 of each 28-day treatment cycle.
Dose Escalation: Arm A (Venetoclax 400mg + Cobi 60mg)VenetoclaxParticipants received Venetoclax 400mg daily on Days 1-28 of each 28 day treatment cycle and Cobimetinib 60mg daily on Days 1-21 of each 28-day treatment cycle.
Dose Escalation: Arm B (Venetoclax 400mg + Ida 200mg)VenetoclaxParticipants received Venetoclax 400mg daily on Days 1-28 of each 28 day treatment cycle and Idasanutlin 200mg daily or twice daily on Days 1-5 of each 28 day treatment cycle.
Dose Escalation: Arm B (Venetoclax 600mg + Ida 200mg)IdasanutlinParticipants received Venetoclax 600mg daily on Days 1-28 of each 28 day treatment cycle and Idasanutlin 200mg daily or twice daily on Days 1-5 of each 28 day treatment cycle.
Dose Escalation: Arm B (Venetoclax 600mg + Ida 150mg)VenetoclaxParticipants received Venetoclax 600mg daily on Days 1-28 of each 28 day treatment cycle and Idasanutlin 150mg daily or twice daily on Days 1-5 of each 28 day treatment cycle.
Dose Escalation: Arm B (Venetoclax 600mg + Ida 200mg)VenetoclaxParticipants received Venetoclax 600mg daily on Days 1-28 of each 28 day treatment cycle and Idasanutlin 200mg daily or twice daily on Days 1-5 of each 28 day treatment cycle.
Dose Escalation: Arm B (Venetoclax 400mg + Ida 400mg)VenetoclaxParticipants received Venetoclax 400mg daily on Days 1-28 of each 28 day treatment cycle and Idasanutlin 400mg daily or twice daily on Days 1-5 of each 28 day treatment cycle.
Dose Escalation: Arm B (Venetoclax 400mg + Ida 400mg)IdasanutlinParticipants received Venetoclax 400mg daily on Days 1-28 of each 28 day treatment cycle and Idasanutlin 400mg daily or twice daily on Days 1-5 of each 28 day treatment cycle.
Dose Optimisation: Arm B (Ven 600mg (Day 1 to 21) + Ida 150mg)VenetoclaxParticipants received Venetoclax 600mg daily on Days 1-21 of each 28 day treatment cycle and Idasanutlin 150mg daily on Days 1-5 of each 28 day treatment cycle.
Dose Escalation: Arm B (Venetoclax 400mg + Ida 200mg)IdasanutlinParticipants received Venetoclax 400mg daily on Days 1-28 of each 28 day treatment cycle and Idasanutlin 200mg daily or twice daily on Days 1-5 of each 28 day treatment cycle.
Dose Escalation: Arm B (Venetoclax 600mg + Ida 150mg)IdasanutlinParticipants received Venetoclax 600mg daily on Days 1-28 of each 28 day treatment cycle and Idasanutlin 150mg daily or twice daily on Days 1-5 of each 28 day treatment cycle.
Dose Optimisation: Arm B (Ven 600mg (Day 1 to 21) + Ida 150mg)IdasanutlinParticipants received Venetoclax 600mg daily on Days 1-21 of each 28 day treatment cycle and Idasanutlin 150mg daily on Days 1-5 of each 28 day treatment cycle.
Primary Outcome Measures
NameTimeMethod
Number of Participants with Dose Limiting Toxicities (DLTs)From Cycle 1 Day 1 to Cycle 2 Day 1 for a minimum of 28 days
Number of Participants with Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)Baseline up until 30 days after the last dose of study drug (up to a maximum of 4 years, 9 months)
Secondary Outcome Measures
NameTimeMethod
Overall Survival (OS)Up to 2 years
CR + Complete Remission with Partial Hematologic Recovery (CRh) RateUp to 2 years
Duration of Response (DOR)Up to 2 years
Progression-Free Survival (PFS)Up to 2 years
Event-Free Survival (EFS)Up to 2 years
Time to Progression (TTP)Up to 2 years
Leukemia-Free Survival (LFS)Up to 2 years
Overall Response Rate (ORR) (Complete Remission (CR) + Complete Remission with Incomplete Blood Recovery (CRi) + Incomplete Platelet Count Recovery (CRp) + Partial Remission/Partial Response [PR])Up to 2 years
Complete Remission/complete response (CR) + Complete Remission with Incomplete Blood Recovery (CRi) + Incomplete Platelet Count Recovery (CRp)Up to 2 years
Pharmacokinetics of Venetoclax Area Under the Concentration-Time Curve from Time Zero to Last Measurable Concentration (AUC0-24hr)Up to 6 months
Pharmacokinetics of Venetoclax Maximum Observed Concentration (Cmax)Up to 6 months
Pharmacokinetics of Cobimetinib Area Under the Concentration-Time Curve from Time Zero to Last Measurable Concentration (AUC0-24hr)Up to 6 months
Pharmacokinetics of Cobimetinib Maximum Observed Concentration (Cmax)Up to 6 months
Pharmacokinetics of Idasanutlin Area Under the Concentration-Time Curve from Time Zero to Last Measurable Concentration (AUC0-24hr)Up to 6 months
Pharmacokinetics of Idasanutlin Maximum Observed Concentration (Cmax)Up to 6 months
Number of Participants Reporting Symptoms in Patient-Reported Outcomes of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) QuestionnaireUp to 2 years
Rate of Transfusion IndependenceUp to 2 years
Duration Of Transfusion Independence, Defined As The Number Of Consecutive Days Of Transfusion Independence, Measured From 1 Day After Last Transfusion To Disease Progression Or Subsequent TransfusionUp to 2 years
Minimal Residual Disease (MRD) In The Bone Marrow To Evaluate The Depth Of ResponseUp to 2 years

Trial Locations

Locations (17)

Weill Cornell Medical College

🇺🇸

New York, New York, United States

University of Alberta Hospital

🇨🇦

Edmonton, Alberta, Canada

Institut Paoli Calmettes

🇫🇷

Marseille, France

Jewish General Hospital

🇨🇦

Montreal, Quebec, Canada

University of Bologna

🇮🇹

Bologna, Emilia-Romagna, Italy

USC Norris Cancer Center

🇺🇸

Los Angeles, California, United States

UC Davis; Comprehensive Cancer Center

🇺🇸

Sacramento, California, United States

MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Univ of Calif, San Francisco

🇺🇸

San Francisco, California, United States

University of Colorado

🇺🇸

Aurora, Colorado, United States

Dana Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

Princess Margaret Cancer Center

🇨🇦

Toronto, Ontario, Canada

Hopital Avicenne, Paris

🇫🇷

Bobigny, France

CHU de Bordeaux

🇫🇷

Pessac, France

Presidio san salvatore muraglia

🇮🇹

Pesaro, Emilia-Romagna, Italy

Universita di Roma

🇮🇹

Roma, Emilia-Romagna, Italy

Wake Forest Baptist Medical Center

🇺🇸

Winston-Salem, North Carolina, United States

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