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A Study of BGB-11417 in Participants With Myeloid Malignancies

Phase 1
Recruiting
Conditions
Acute Myeloid Leukemia
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Neoplasm
Interventions
Registration Number
NCT04771130
Lead Sponsor
BeiGene
Brief Summary

The study will determine the safety, tolerability, recommended Phase 2 dose (RP2D) and preliminary efficacy of BGB-11417 as monotherapy and in combination with azacitidine in participants with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)or MDS/myeloproliferative neoplasm (MPN) .

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
260
Inclusion Criteria
  1. Confirmed diagnosis of one of the following by 2016 World Health Organization criteria:

    • AML, nonacute promyelocytic leukemia
    • MDS
    • MDS/MPN
  2. Eastern Cooperative Oncology Group performance status of 0 to 2.

  3. Adequate organ function defined as:

    • Creatinine clearance ≥ 50 milliliters/minute (mL/min) (or between 30 and 49 mL/min in unfit AML cohort)
    • Adequate liver function
  4. Life expectancy of > 12 weeks.

  5. Ability to comply with the requirements of the study.

Key

Exclusion Criteria
  1. A diagnosis of acute promyelocytic leukemia.
  2. History of prior malignancy, with the exception of either a history of MDS or MDS/MPN that has transformed to AML, or other prior malignancy that was treated with a full curative intent and no evidence of recurrence within the past 2 years (eg, localized skin cancer, superficial bladder cancer, carcinoma in situ of the cervix or breast, or localized Gleason score ≤ 6 prostate cancer)
  3. Antecedent MPN including myelofibrosis, essential thrombocytosis, polycythemia vera, or chronic myelogenous leukemia with or without BCR-ABL1 translocation and AML with BCR-ABL1 translocation.
  4. Prior therapy with a B-cell lymphoma-2 inhibitor
  5. Known central nervous system involvement by leukemia.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Part 3: AML and MDS CohortBGB-11417Participants with MDS and R/R AML (China only) will receive BGB-11417 on a 28-day cycle.
Parts 1 and 2: AML CohortsBGB-11417Participants with AML will receive BGB-11417 and azacitidine on a 28-day cycle.
Parts 1 and 2: AML CohortsAzacitidineParticipants with AML will receive BGB-11417 and azacitidine on a 28-day cycle.
Parts 1 and 2: MDS CohortsAzacitidineParticipants with MDS will receive BGB-11417 and azacitidine on a 28-day cycle.
Parts 1 and 2: MDS CohortsBGB-11417Participants with MDS will receive BGB-11417 and azacitidine on a 28-day cycle.
Part 3: AML and MDS CohortsBGB-11417Participants with AML and MDS will receive BGB-11417 and azacitidine on a 28-day cycle. A subset of the participants will receive a modified second cycle of treatment to explore drug-drug interactions (DDI) with posaconazole.
Part 3: AML and MDS CohortsAzacitidineParticipants with AML and MDS will receive BGB-11417 and azacitidine on a 28-day cycle. A subset of the participants will receive a modified second cycle of treatment to explore drug-drug interactions (DDI) with posaconazole.
Part 3: AML and MDS CohortsPosaconazoleParticipants with AML and MDS will receive BGB-11417 and azacitidine on a 28-day cycle. A subset of the participants will receive a modified second cycle of treatment to explore drug-drug interactions (DDI) with posaconazole.
Primary Outcome Measures
NameTimeMethod
Part 1 And 2: Number Of Participants Experiencing Dose-limiting Toxicities (DLTs)Cycle 1 (Up to 28 days for non-hematologic DLTs and up to 42 days for hematologic DLTs)
Part 1 And 2: Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)Approximately 24 months
Part 3 AML Cohort: Complete Remission (CR) Plus CR With Partial Hematologic Recovery (CRh) RateApproximately 24 months

CR plus CRh will be defined as the percentage of participants whose best overall response (BOR) is CR plus CRh. BOR will be defined as the best response recorded from the first dose of study drug until data cut or the initiation of new anticancer treatment.

Part 3 MDS Cohort: Modified Overall Response (mOR) RateApproximately 24 months

The mOR will be defined as the percentage of participants whose BOR is achieving CR, marrow complete remission (mCR), or partial remission (PR) at any time point during the study for myelodysplastic/myeloproliferative neoplasm (MDS/MPN).

Part 3 AML Cohort (DDI Sub-cohort): Area Under Plasma Concentration-time Curve (AUC) from time 0 to the last quantifiable timepoint (t) (AUC0-t) Of BGB-11417 When Administered Alone and when Co-administered With PosaconazoleCycle 2 Day 12 and Cycle 2 Day 20 (predose and 1, 2, 4, 6, 8, and 24 hours postdose)
Part 3 AML Cohort (DDI Sub-cohort): Maximum Observed Plasma Concentration (Cmax) Of BGB-11417 When Administered Alone and When Co-administered With PosaconazoleCycle 2 Day 12 and Cycle 2 Day 20 (predose and 1, 2, 4, 6, 8, and 24 hours postdose)
Part 3 AML Cohort (DDI Sub-cohort): Area Under Plasma Concentration-time Curve (AUC) from time 0 to infinity (AUC0-infinity) Of BGB-11417 When Administered Alone and When Co-administered With PosaconazoleCycle 2 Day 12 and Cycle 2 Day 20 (predose and 1, , 4, 6, 8, and 24 hours postdose)
Part 3 AML and MDS Cohorts (Treated with Monotherapy): Number Of Participants Experiencing DLTsCycle 2
Part 3 AML and MDS Cohorts (Treated with Monotherapy): Number of Participants Experiencing TEAEsApproximately 24 months
Secondary Outcome Measures
NameTimeMethod
Parts 1 And 2: Cmax Of Azacitidine When Coadministered With BGB-11417Day 0 and 4 (Cycle 1) predose and at multiple time points up to 4 hours postdose
Parts 1 And 2: Terminal Half-life (t1/2) Of Azacitidine When Coadministered With BGB-11417Day 0 and 4 (Cycle 1) predose and at multiple time points up to 4 hours postdose
Parts 1 And 2: AUC From Time Zero To Time t (AUC0-t) Of Azacitidine When Coadministered With BGB-11417Day 0 and 4 (Cycle 1) predose and at multiple time points up to 4 hours postdose
Parts 1 And 2: AUC From Time Zero To Infinity (AUC0-inf) Of Azacitidine When Coadministered With BGB-11417Day 0 and 4 (Cycle 1) predose and at multiple time points up to 4 hours postdose
Parts 1 And 2: Apparent Total Clearance Of Azacitidine From Plasma (CL/F) When Coadministered With BGB-11417Day 0 and 4 (Cycle 1) predose and at multiple time points up to 4 hours postdose
Parts 1 And 2: Apparent Volume Of Distribution (Vz/F) Of Azacitidine When Coadministered With BGB-11417Day 0 and 4 (Cycle 1) predose and at multiple time points up to 4 hours postdose
Parts 1 And 2 AML Cohort: Complete remission (CR) + Morphologic CR With Partial Hematologic Recovery (CRh)Approximately 24 months
Parts 1 And 2 MDS Cohort: mOR RateApproximately 24 months
Parts 1 And 2: Steady-state AUC From Time Zero To Time Of Last Measurable Concentration (AUClast,ss) Of BGB-11417 When Coadministered With AzacitidineDay 1-4 and 28 (Cycle 1) Day 5 (Cycle 2) predose and at multiple time points up to 8 hours postdose
Parts 1 And 2: Steady-state Cmax (Cmax,ss) Of BGB-11417 When Coadministered With AzacitidineDay 1-4 and 28 (Cycle 1) Day 5 (Cycle 2) predose and at multiple time points up to 8 hours postdose
Parts 1 And 2: Steady-state Trough Plasma Concentration (Ctrough,ss) Of BGB-11417 When Coadministered With AzacitidineDay 1-4 and 28 (Cycle 1) Day 5 (Cycle 2) predose and at multiple time points up to 8 hours postdose
Parts 1 And 2: Steady-state Time To Maximum Observed Plasma Concentration (tmax,ss) Of BGB-11417 When Coadministered With AzacitidineDay 1-4 and 28 (Cycle 1) Day 5 (Cycle 2) predose and at multiple time points up to 8 hours postdose
Part 3: Number Of Participants Experiencing TEAEsApproximately 24 months
Part 3: Complete Response RateApproximately 24 months

CR will be defined as the percentage of participants whose BOR is CR. BOR will be defined as the best response recorded from the first dose of study drug until data cut or the initiation of new anticancer treatment.

Part 3 AML Cohort: CR With Incomplete Hematologic Recovery (CRi) RateApproximately 24 months

CRi will be defined as the percentage of participants whose BOR is CRi.

Part 3 AML Cohort: Overall Response Rate (ORR)Approximately 24 months

The ORR will include participants whose BOR include CR, CRi, PR, and morphologic leukemia-free state.

Part 3 AML Cohort: Duration Of Response (DOR)Approximately 24 months

DOR will be defined as the time from the first response to disease progression documented after treatment initiation or death, whichever occurs first. DOR will include CR, CR plus CRi, overall response (OR), and CR plus CRh.

Part 3 AML Cohort: Time To Response (TTR)Approximately 24 months

TTR will be defined as the time from treatment initiation to the first documented response and will include CR, CR plus CRi, OR, and CR plus CRh.

Part 3 Event-free Survival (EFS)Approximately 24 months

EFS will be defined as the time from treatment initiation to disease progression, treatment failure, relapse (hematologic relapse for AML) for those who have treatment success, or death due to any cause, whichever happens first.

Part 3 Overall Survival (OS)Approximately 24 months

OS will be defined as the time from treatment initiation to death. OS will be analyzed using the same methods as the EFS analysis except for the censoring rules.

Part 3 AML Cohort: Number of Participants with Transfusion IndependenceApproximately 24 months

Transfusion independence will be defined as the proportion of participants whose BOR is transfusion independence. Transfusion independence will need to last for at least 56 consecutive days postbaseline.

Part 3 MDS Cohort: Number Of Participants With Hematological Improvement-erythroid (HI-E)Approximately 24 months

The proportion of participants whose BOR is HI-E

Part 3 MDS Cohort: Proportion Of Participants With Hematological Improvement-platelet (HI-P)Approximately 24 months

The proportion of participants whose BOR is HI-P

Part 3 MDS Cohort: Proportion Of Participants With Hematological Improvement-neutrophil (HI-N)Approximately 24 months

The proportion of participants whose BOR is HI-N will be reported.

Part 3 MDS Cohort: Number of participants with Transfusion IndependenceApproximately 24 months

Transfusion independence will be defined as the percentage of participants whose BOR is transfusion independence. Transfusion independence will need to last for at least 56 consecutive days postbaseline.

Part 3: Ctrough,ss Of BGB-11417 When Coadministered With AzacitidineCycle 1 Day 1 and Cycle 2 Day 5 (predose and 4 and 6 hours postdose)
Part 3 MDS cohort: Partial Hematologic Recovery CRhApproximately 24 months

Percentage of participants with partial hematologic recovery will be reported

Part 3 AML (Treated with Monotherapy): Complete Response + Morphologic complete Remission with Partial Hematologic RecoveryApproximately 24 months

Percentage of participants with Complete Response + Morphologic complete Remission with Partial Hematologic Recovery will be reported.

Part 3 AML (Treated with Monotherapy): Steady State trough plasma concentration of BGB-11417Cycle 2 Day 12 and Cycle 2 Day 20 (predose and 1, 4, 6, 8, and 24 hours postdose)
Part 3 MDS (Treated with Monotherapy): Modified Overall ResponseApproximately 24 months

Percentage of participants with modified overall response including CR, marrow CR (mCR) or partial remission (PR)

Part 3 MDS (Treated with Monotherapy): Steady State trough plasma concentration of BGB-11417Cycle 2 Day 12 and Cycle 2 Day 20 (predose and 1, 4, 6, 8, and 24 hours postdose)

Trial Locations

Locations (49)

Hopital Saint Louis

🇫🇷

Paris, France

Universitaetsklinikum Leipzig Aor

🇩🇪

Leipzig, Germany

Universitaetsklinikum Ulm

🇩🇪

Ulm, Germany

Policlinico Sorsola Malpighi, Aou Di Bologna

🇮🇹

Bologna, Italy

Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori Irst

🇮🇹

Meldola, Italy

Niguarda Cancer Center Division of Hematology

🇮🇹

Milano, Italy

Hopital Larchet

🇫🇷

Nice, France

Hopital Claude Huriez Chu Lille

🇫🇷

Lille, France

Gold Coast University Hospital

🇦🇺

Southport, Queensland, Australia

City of Hope National Medical Center

🇺🇸

Duarte, California, United States

Tampa General Hospital

🇺🇸

Tampa, Florida, United States

Upmc Hillman Cancer Center(Univ of Pittsburgh)

🇺🇸

Pittsburgh, Pennsylvania, United States

Md Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Medical College of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

Concord Repatriation General Hospital

🇦🇺

Concord, New South Wales, Australia

St George Hospital

🇦🇺

Kogarah, New South Wales, Australia

Orange Health Hospital

🇦🇺

Orange, New South Wales, Australia

Monash Health

🇦🇺

Clayton, Victoria, Australia

St Vincents Hospital Melbourne

🇦🇺

Fitzroy, Victoria, Australia

Austin Health

🇦🇺

Heidelberg, Victoria, Australia

The Alfred Hospital

🇦🇺

Melbourne, Victoria, Australia

Fiona Stanley Hospital

🇦🇺

Murdoch, Western Australia, Australia

Linear Clinical Research

🇦🇺

Nedlands, Western Australia, Australia

One Clinical Research

🇦🇺

Nedlands, Western Australia, Australia

Peking University Peoples Hospital

🇨🇳

Beijing, Beijing, China

The First Hospital of Lanzhou University

🇨🇳

Lanzhou, Gansu, China

Guangdong Provincial Peoples Hospital

🇨🇳

Guangzhou, Guangdong, China

Nanfang Hospital of Southern Medical University

🇨🇳

Guangzhou, Guangdong, China

The Second Peoples Hospital of Shenzhen

🇨🇳

Shenzhen, Guangdong, China

Henan Cancer Hospital

🇨🇳

Zhengzhou, Henan, China

Union Hospital of Tongji Medical College, Huazhong University of Science and Technology

🇨🇳

Wuhan, Hubei, China

The First Affiliated Hospital of Soochow University

🇨🇳

Suzhou, Jiangsu, China

The First Affiliated Hospital of Nanchang University Branch Donghu

🇨🇳

Nanchang, Jiangxi, China

West China Hospital, Sichuan University

🇨🇳

Chengdu, Sichuan, China

Tianjin Medical University Cancer Institute and Hospital

🇨🇳

Tianjin, Tianjin, China

The First Affiliated Hospital, Zhejiang University School of Medicine

🇨🇳

Hangzhou, Zhejiang, China

Samsung Medical Center

🇰🇷

GangnamGu, Seoul Teugbyeolsi, Korea, Republic of

Severance Hospital Yonsei University Health System

🇰🇷

SeodaemunGu, Seoul Teugbyeolsi, Korea, Republic of

Seoul National University Hospital

🇰🇷

Seoul, Seoul Teugbyeolsi, Korea, Republic of

Asan Medical Center

🇰🇷

SongpaGu, Seoul Teugbyeolsi, Korea, Republic of

North Shore Hospital

🇳🇿

Auckland, New Zealand

Aotearoa Clinical Trials

🇳🇿

Auckland, New Zealand

Wellington Regional Hospital (Ccdhb)

🇳🇿

Wellington, New Zealand

Hospital de La Santa Creu I Sant Pau

🇪🇸

Barcelona, Spain

Hospital Universitario de Salamanca

🇪🇸

Salamanca, Spain

Hospital Universitario Virgen Del Rocio

🇪🇸

Sevilla, Spain

Hospital Universitari I Politecnic La Fe

🇪🇸

Valencia, Spain

Edinburgh Cancer Centre

🇬🇧

Edinburgh, United Kingdom

The Christie Hospital

🇬🇧

Greater Manchester, United Kingdom

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