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Study to Evaluate Adverse Events and Movement of Lemzoparlimab in Body When Used Intravenously (IV) With Azacitidine Subcutaneously or IV and Venetoclax Orally in Participants With Acute Myeloid Leukemia and With Azacitidine With or Without Venetoclax in Participants With Myelodysplastic Syndrome

Phase 1
Terminated
Conditions
Myelodysplastic Syndrome (MDS)
Acute Myeloid Leukemia (AML)
Interventions
Registration Number
NCT04912063
Lead Sponsor
AbbVie
Brief Summary

Acute myeloid leukemia (AML) is one of the most aggressive blood cancers, with a very low survival rate and few options for participants who are unable to undergo intensive chemotherapy, the current standard of care. This study is to evaluate how safe lemzoparlimab is and how it moves within the body when used along with azacitidine and/or venetoclax in adult participants with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Adverse events and maximum tolerated dose (MTD) of lemzoparlimab will be assessed.

Lemzoparlimab (TJ011133) is being evaluated in combination with azacitidine and venetoclax for the treatment of acute myeloid leukemia (AML) and with azacitidine with/without venetoclax for myelodysplastic syndrome (MDS). Study doctors place the participants in 1 of 5 groups, called treatment arms. Each group receives a different treatment. Adult participants with a diagnosis of AML or MDS will be enrolled. Around 80 participants will be enrolled in the study in approximately 50 sites worldwide.

Participants will receive lemzoparlimab (IV) once weekly (Q1W), venetoclax oral tablets once daily (QD) for 28 days (AML participants) or 14 days (MDS participants) and Azacitidine by SC or IV route QD for 7 days of each 28-day cycle.

There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests and checking for side effects.

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
40
Inclusion Criteria
  • Documented confirmation of acute myeloid leukemia (AML) according to the World Health Organization (WHO) criteria, previously untreated [OR]

  • Documented diagnosis of previously untreated de novo myelodysplastic syndrome (MDS) according to the 2017 WHO classification with presence of < 20% bone marrow blasts per marrow biopsy/aspirate.

  • Participants with documented MDS must meet the following disease activity criteria:

    • Overall revised international prognostic scoring system (IPSS-R) score > 3 (intermediate, high, or very high);
    • Eastern cooperative oncology group (ECOG) performance status of 0 to 2;
    • Hematopoietic stem cell transplant (HSCT) ineligible, or participant who chooses not to undergo HSCT.
  • Participants with documented AML with adverse cytogenetic and/or molecular risk, and must be considered ineligible for induction therapy defined by the following:

    • >= 75 years of age; [OR]

    • >= 18 to 74 years of age with at least one of the following comorbidities: --- Eastern cooperative oncology group (ECOG) performance status of 2 to 3; --- Cardiac history of congestive heart failure requiring treatment or ejection fraction <= 50% or chronic stable angina;

      • Diffusion capacity of lung (DLCO) <= 65% or forced expiratory volume during the first second (FEV1) <= 65%;
      • Creatinine clearance >= 30 mL/min to < 45 mL/min;
      • Moderate hepatic impairment with total bilirubin > 1.5 to <= 3.0 × upper limit of normal (ULN);
      • Any other comorbidity that the physician judges to be incompatible with intensive chemotherapy or the participant declines to receive intensive chemotherapy.

Japan Safety Lead-In Phase:

  • Documented confirmation of AML according to WHO criteria, relapsed or refractory (R/R) disease without other standard of care treatments.

  • Documented diagnosis of MDS according to the 2017 WHO classification with presence of < 20% bone marrow blasts per marrow biopsy/aspirate, with intermediate- and high-risk relapsed/refractory MDS.

  • Documented MDS must meet the following disease activity criteria:

    • ECOG performance status of 0 to 2.
Exclusion Criteria
  • Participants with documented AML with acute promyelocytic leukemia and considered eligible for induction therapy.

  • Participant with documented AML having prior diagnosis of:

    -- known active central nervous system involvement with AML.

  • Participants with documented MDS having prior diagnosis of:

    • MDS evolving from a pre-existing myeloproliferative neoplasm (MPN);
    • MDS/MPN including chronic myelomonocytic leukemia, atypical chronic myeloid leukemia, juvenile myelomonocytic leukemia and unclassifiable MDS/MPN.
  • History of allogeneic HSCT or solid organ transplantation.

  • Previous exposure to anti-CD47 therapies.

  • History of an active malignancy within the past 2 years prior to Screening, with the exception of:

    -- Adequately treated carcinoma in situ of the cervix uteri or carcinoma in situ of the breast;

    • Adequately treated basal cell carcinoma or localized squamous cell carcinoma of the skin;
    • Asymptomatic prostate cancer without known metastatic disease and with no requirement for therapy;
    • Previous malignancy confined and surgically resected (or treated with other modalities) with curative intent.
  • Conditions that could interfere with drug absorption including but not limited to short bowel syndrome.

Japan Safety Lead-In Phase:

  • Documented AML have Acute Promyelocytic Leukemia.

  • Participant with documented AML having prior diagnosis of:

    -- Chronic myeloid leukemia with or without BCR-ABL1 translocation and AML with BCR-ABL1 translocation.

  • Participants with documented MDS having prior diagnosis of:

    • Therapy-related MDS.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Lemzoparlimab + Azacitidine + Venetoclax in AML (Expansion)LemzoparlimabLemzoparlimab (TJ011133) co-administered with azacitidine and venetoclax in expansion cohort in participants with treatment-naïve acute myeloid leukemia (AML) who are ineligible for standard induction therapy.
Lemzoparlimab + Azacitidine + Venetoclax in MDS (Expansion)LemzoparlimabLemzoparlimab (TJ011133) co-administered with azacitidine and venetoclax in expansion cohort in participants with treatment-naïve higher-risk myelodysplastic syndrome (MDS).
Lemzoparlimab + Azacitidine + Venetoclax in AML (Escalation)VenetoclaxLemzoparlimab (TJ011133) co-administered with azacitidine and venetoclax in escalated doses in participants with treatment-naïve acute myeloid leukemia (AML) who are ineligible for standard induction therapy.
Lemzoparlimab + Azacitidine + Venetoclax in AML (Escalation)AzacitidineLemzoparlimab (TJ011133) co-administered with azacitidine and venetoclax in escalated doses in participants with treatment-naïve acute myeloid leukemia (AML) who are ineligible for standard induction therapy.
Lemzoparlimab + Azacitidine in MDS (Escalation)LemzoparlimabLemzoparlimab (TJ011133) co-administered with azacitidine in escalated doses in participants with treatment-naïve higher-risk myelodysplastic syndrome (MDS).
Lemzoparlimab + Azacitidine + Venetoclax in MDS (Expansion)VenetoclaxLemzoparlimab (TJ011133) co-administered with azacitidine and venetoclax in expansion cohort in participants with treatment-naïve higher-risk myelodysplastic syndrome (MDS).
Lemzoparlimab + Azacitidine + Venetoclax in AML (Escalation)LemzoparlimabLemzoparlimab (TJ011133) co-administered with azacitidine and venetoclax in escalated doses in participants with treatment-naïve acute myeloid leukemia (AML) who are ineligible for standard induction therapy.
Lemzoparlimab + Azacitidine + Venetoclax in MDS (Escalation)LemzoparlimabLemzoparlimab (TJ011133) co-administered with azacitidine and venetoclax in escalated doses in participants with treatment-naïve higher-risk myelodysplastic syndrome (MDS).
Lemzoparlimab + Azacitidine + Venetoclax in MDS (Escalation)AzacitidineLemzoparlimab (TJ011133) co-administered with azacitidine and venetoclax in escalated doses in participants with treatment-naïve higher-risk myelodysplastic syndrome (MDS).
Lemzoparlimab + Azacitidine + Venetoclax in MDS (Escalation)VenetoclaxLemzoparlimab (TJ011133) co-administered with azacitidine and venetoclax in escalated doses in participants with treatment-naïve higher-risk myelodysplastic syndrome (MDS).
Lemzoparlimab + Azacitidine in MDS (Escalation)AzacitidineLemzoparlimab (TJ011133) co-administered with azacitidine in escalated doses in participants with treatment-naïve higher-risk myelodysplastic syndrome (MDS).
Lemzoparlimab + Azacitidine + Venetoclax in AML (Expansion)AzacitidineLemzoparlimab (TJ011133) co-administered with azacitidine and venetoclax in expansion cohort in participants with treatment-naïve acute myeloid leukemia (AML) who are ineligible for standard induction therapy.
Lemzoparlimab + Azacitidine + Venetoclax in AML (Expansion)VenetoclaxLemzoparlimab (TJ011133) co-administered with azacitidine and venetoclax in expansion cohort in participants with treatment-naïve acute myeloid leukemia (AML) who are ineligible for standard induction therapy.
Lemzoparlimab + Azacitidine + Venetoclax in MDS (Expansion)AzacitidineLemzoparlimab (TJ011133) co-administered with azacitidine and venetoclax in expansion cohort in participants with treatment-naïve higher-risk myelodysplastic syndrome (MDS).
Lemzoparlimab Monotherapy in AML (Japan Only Escalation)LemzoparlimabLemzoparlimab (TJ011133) administered in escalated doses in participants with treatment-naïve acute myeloid leukemia (AML) who are ineligible for standard induction therapy.
Lemzoparlimab Monotherapy in MDS (Japan Only Escalation)LemzoparlimabLemzoparlimab (TJ011133) administered in escalated doses in participants with treatment-naïve higher-risk myelodysplastic syndrome (MDS).
Primary Outcome Measures
NameTimeMethod
Dose Limiting Toxicities (DLTs) of Lemzoparlimab (TJ011133) When Co-administered With Venetoclax and Azacitidine in Participants With Treatment-Naïve Acute Myeloid Leukemia (AML) Ineligible for Standard Induction TherapyUp to 30 days after first dose of study drug

DLT events are defined as clinically significant adverse events or abnormal laboratory values assessed as unrelated to disease progression, underlying disease, intercurrent illness, or concomitant medications and occurring during the first 4 weeks after administration of the first dose and that meets additional criteria as described in the protocol.

Dose Limiting Toxicities (DLTs) of Lemzoparlimab (TJ011133) as a Monotherapy in Japanese Participants with Relapsed/Refractory (R/R) AMLUp to 30 days after first dose of study drug

DLT events are defined as clinically significant adverse events or abnormal laboratory values assessed as unrelated to disease progression, underlying disease, intercurrent illness, or concomitant medications and occurring during the first 4 weeks after administration of the first dose and that meets additional criteria as described in the protocol.

DLTs of Lemzoparlimab (TJ011133) When Co-administered With Azacitidine With or Without Venetoclax in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndrome (MDS)Up to 30 days after first dose of study drug

DLT events are defined as clinically significant adverse events or abnormal laboratory values assessed as unrelated to disease progression, underlying disease, intercurrent illness, or concomitant medications and occurring during the first 4 weeks after administration of the first dose and that meets additional criteria as described in the protocol.

DLTs of Lemzoparlimab (TJ011133) as a Monotherapy in Japanese Participants with R/R MDSUp to 30 days after first dose of study drug

DLT events are defined as clinically significant adverse events or abnormal laboratory values assessed as unrelated to disease progression, underlying disease, intercurrent illness, or concomitant medications and occurring during the first 4 weeks after administration of the first dose and that meets additional criteria as described in the protocol.

Secondary Outcome Measures
NameTimeMethod
Best Overall Response of Composite CR (CRc) for AMLUp to approximately 3 years

Best overall response of composite CR (CRc), \[CR or CR with incomplete blood count recovery (CRi)\] according to modified IWG 2003 criteria for AML.

Overall Survival (OS ) for AMLUp to approximately 3 years

Overall survival (OS), defined as the time from the date of the first dose of any study drug (lemzoparlimab or venetoclax or azacitidine) to death from any cause.

Best Overall Response of Marrow-Complete Remission (mCR), for MDSUp to approximately 3 years

Best overall response of marrow-complete remission (mCR), per the modified IWG 2006 criteria for MDS.

Platelet TI, for MDSUp to approximately 3 years

Platelet TI, defined as a participant who is transfusion dependent at baseline achieved TI post-baseline.

Progression Free Survival (PFS), for MDSUp to approximately 3 years

Progression Free Survival (PFS) defined as the time from the date of the first dose of any study drug to PD or death from any cause.

OS, for MDSUp to approximately 3 years

OS, defined as the time from the date of the first dose of any study drug to death from any cause.

Best Overall Response of CR or PR or mCR, for MDSUp to approximately 3 years

Best overall response of CR or PR or mCR, per the modified IWG 2006 criteria for MDS.

Best Overall Response of Complete Remission (CR) for AMLUp to approximately 3 years

Best overall response of complete remission (CR), defined as achieving CR according to modified international working group (IWG) 2003 criteria for AML.

Duration of Response (DOR) for AMLUp to approximately 3 years

Duration of response (DOR), defined for participants who achieve a best overall response, as the time from the first occurrence of response to disease progression/relapse from CR, CRi or CRh or death from disease progression, whichever occurs first.

Best Overall Response of CR, for MDSUp to approximately 3 years

Best overall response of CR per the modified IWG 2006 criteria for MDS.

Best Overall Response of CR or PR for MDSUp to approximately 3 years

Best overall response of CR or PR, per the modified IWG 2006 criteria for MDS.

Hematologic Improvement (HI), for MDSUp to approximately 3 years

Hematologic improvement (HI), defined as a participant achieving erythroid/platelet/neutrophil responses.

DOR, for MDSUp to approximately 3 years

DOR, defined for participants who achieve a best overall response, as the time from the first occurrence of response (CR or mCR or PR) to disease progression or death, whichever occurs first.

Best Overall Response of CR or Complete Remission With Partial Hematologic Recovery (CRh) for AMLUp to approximately 3 years

Best overall response of CR or CRh, defined according to modified IWG 2003 criteria for AML.

Event-Free Survival (EFS) for AMLUp to approximately 3 years

Event-free survival (EFS), defined as time from first dose of any study drug (lemzoparlimab or venetoclax or azacitidine) to the date of progressive disease (PD), relapse from CR or CRi, treatment failure defined as failure to achieve CR, CRi or MLFS after at least 6 cycles of study treatment, or death from any cause, whichever occurs first.

Red Blood Cell Transfusion Independence (TI), for MDSUp to approximately 3 years

Red blood cell transfusion independence (TI), defined as a participant who is transfusion dependent at baseline achieved TI post-baseline.

Trial Locations

Locations (29)

Hadassah Medical Center-Hebrew University /ID# 227275

🇮🇱

Jerusalem, Yerushalayim, Israel

Austin Health /ID# 227717

🇦🇺

Heidelberg, Victoria, Australia

Rabin Medical Center /ID# 227738

🇮🇱

Petakh Tikva, Israel

University of Fukui Hospital /ID# 232466

🇯🇵

Yoshida-gun, Fukui, Japan

Kyushu University Hospital /ID# 232564

🇯🇵

Fukuoka-shi, Fukuoka, Japan

The Chaim Sheba Medical Center /ID# 227389

🇮🇱

Ramat Gan, Tel-Aviv, Israel

ASST Grande Ospedale Metropolitano Niguarda /ID# 226952

🇮🇹

Milano, Italy

Norton Cancer Institute - St Matthews /ID# 228378

🇺🇸

Louisville, Kentucky, United States

University of Alabama at Birmingham - Main /ID# 227071

🇺🇸

Birmingham, Alabama, United States

University of Michigan /ID# 227030

🇺🇸

Ann Arbor, Michigan, United States

Massachusetts General Hospital /ID# 227273

🇺🇸

Boston, Massachusetts, United States

Beth Israel Deaconess Medical Center /ID# 231083

🇺🇸

Boston, Massachusetts, United States

UPMC Hillman Cancer Ctr /ID# 228048

🇺🇸

Pittsburgh, Pennsylvania, United States

MD Anderson Cancer Center at Texas Medical Center /ID# 227019

🇺🇸

Houston, Texas, United States

University of Pennsylvania /ID# 227024

🇺🇸

Philadelphia, Pennsylvania, United States

University of Virginia Health /ID# 227363

🇺🇸

Charlottesville, Virginia, United States

Liverpool Hospital /ID# 227723

🇦🇺

Liverpool, New South Wales, Australia

Universitaetsklinikum Hamburg-Eppendorf (UKE) /ID# 227748

🇩🇪

Hamburg, Germany

Universitaetsklinikum Carl Gustav Carus an der TU Dresden /ID# 227749

🇩🇪

Dresden, Germany

Universitaetsklinikum Leipzig /ID# 227750

🇩🇪

Leipzig, Sachsen, Germany

Marien Hospital Duesseldorf /ID# 227751

🇩🇪

Duesseldorf, Nordrhein-Westfalen, Germany

Tel Aviv Sourasky Medical Center /ID# 227387

🇮🇱

Tel Aviv-Yafo, Tel-Aviv, Israel

IRCCS Azienda Ospedaliero-Universitaria di Bologna /ID# 226950

🇮🇹

Bologna, Italy

Istituto Clinico Humanitas /ID# 226948

🇮🇹

Rozzano, Milano, Italy

National Cancer Center Hospital East /ID# 232498

🇯🇵

Kashiwa-shi, Chiba, Japan

Hospital Clinic de Barcelona /ID# 227772

🇪🇸

Barcelona, Spain

Hospital Universitario Fundacion Jimenez Diaz /ID# 227771

🇪🇸

Madrid, Spain

Yamagata University Hospital /ID# 232451

🇯🇵

Yamagata-shi, Yamagata, Japan

Hospital Universitario Virgen de la Victoria /ID# 227770

🇪🇸

Malaga, Spain

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