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A Study of CC-90002 in Subjects With Acute Myeloid Leukemia (AML) and High-risk Myelodysplastic Syndrome (MDS)

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ClinicalTrials.gov Identifier: NCT02641002
Recruitment Status : Terminated (Preliminary monotherapy data in relapsed/refractory AML and high-risk MDS did not offer a sufficiently encouraging profile for further dose escalation/expansion)
First Posted : December 29, 2015
Last Update Posted : October 18, 2018
Information provided by (Responsible Party):

Brief Summary:

Study CC-90002-AML-001 is an open-label, Phase 1 dose escalation (Part A) and expansion (Part B), clinical study of CC-90002, administered by intravenous (IV) infusion, in subjects with relapsed and/or primary refractory AML and high-risk MDS. The study will explore escalating doses of CC-90002 using a 3 + 3 dose escalation design in Part A, followed by dose expansion in Part B.

The primary objective is to determine the safety and tolerability of CC-90002 and also to define the non-tolerated dose (NTD), the maximum tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D) of CC-90002.

Condition or disease Intervention/treatment Phase
Leukemia, Myeloid, Acute Myelodysplastic Syndromes Drug: CC-90002 Phase 1

Detailed Description:
In both Part A and Part B, treatments will be administered in two phases starting with an induction phase followed by a maintenance phase. During the induction phase, treatments will be administered in 42-day cycles in Cycles 1 through 4. Following completion of Cycle 4 in the induction phase, subjects with non-progressive disease will enter the maintenance phase. During the maintenance phase, treatments will be administered in 28 day cycles. Subjects may continue CC-90002 for up to a maximum of 2 years (eg, induction phase Cycles 1 through 4 and maintenance phase Cycles 5 through 24) or until clinically significant disease progression, the occurrence of intolerable toxicity, or physician/subject decision to discontinue CC-90002, whichever comes first.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 28 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1, Open-label, Dose Finding Study of CC-90002, a Monoclonal Antibody Directed Against CD47, in Subjects With Acute Myeloid Leukemia and High-Risk Myelodsplastic Syndrome
Actual Study Start Date : March 1, 2016
Actual Primary Completion Date : July 18, 2018
Actual Study Completion Date : July 18, 2018

Arm Intervention/treatment
Experimental: Dose escalation of CC-90002
CC-90002 by intravenous (IV) infusion on a 28 day cycle
Drug: CC-90002
Monoclonal Ab to CD47

Primary Outcome Measures :
  1. Dose-limiting Toxicity (DLT) [ Time Frame: Up to 26 months ]
    Number of participants with a DLT

  2. Non-tolerated Dose (NTD) [ Time Frame: Up to 26 months ]
    The NTD is defined as the dose at which 2 or more of up to 6 evaluable subjects in a cohort experience a DLT in Cycle 1

  3. Maximum tolerated dose (MTD) [ Time Frame: Up to 26 months ]
    The MTD is defined as the last dose level(s) below the NTD with 0 or 1 out of 6 evaluable subjects experiencing a DLT during Cycle 1.

Secondary Outcome Measures :
  1. Preliminary Efficacy of CC-90002 [ Time Frame: Up to 35 months ]
    Determined by response rates of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) by disease-appropriate response criteria.

  2. Pharmacokinetics-Cmax [ Time Frame: Up to 35 months ]
    Maximum observed concentration in serum

  3. Pharmacokinetics-AUC [ Time Frame: Up to 35 months ]
    Area under the serum concentration - time curve

  4. Pharmacokinetics-Tmax [ Time Frame: Up to 35 months ]
    Time to peak (maximum) serum concentration

  5. Pharmacokinetics-T 1/2 [ Time Frame: Up to 35 months ]
    Terminal half-life (T 1/2)

  6. Pharmacokinetics- CL [ Time Frame: Up to 35 months ]
    Total body clearance of the drug from the serum

  7. Pharmacokinetics- Vss [ Time Frame: Up to 35 months ]
    Volume of distribution at steady-state

  8. Anti-Drug Antibodies (ADAs) [ Time Frame: Up to 35 months ]
    Determine the presence and frequency of anti-drug antibodies

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

Inclusion Criteria:

  1. Men and women ≥ 18 years of age, at the time of signing the informed consent form (ICF).
  2. Relapsed and/or primary refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) with subtype refractory anemia with excess blasts (RAEB)-2 defined as high or very high-risk that is recurrent or refractory, or the patient is intolerant to established therapy.
  3. Subject consents to hospitalization for first (Cycle 1 Day 1) dose of CC-90002 and for 72 hours after.
  4. Subject consents to serial bone marrow aspiration and biopsies as specified.
  5. Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2.
  6. Eligible study subjects must exhibit acceptable liver, renal, and coagulation function as assessed by laboratory tests.
  7. Females and males must practice true abstinence or agree to contraceptive methods throughout the study, and for up to 8 weeks following the last dose of CC 90002.

Exclusion Criteria:

  1. Active central nervous system (CNS) leukemia or known CNS leukemia.
  2. Immediately life-threatening, severe complications of leukemia.
  3. Impaired cardiac function or clinically significant cardiac diseases.
  4. Glucose-6-phosphate dehydrogenase (G6PD) deficiency.
  5. Prior autologous hematopoietic stem cell transplant ≤ 3 months.
  6. Prior allogeneic hematopoietic stem cell transplant (HSCT) with either standard or reduced intensity conditioning ≤ 6 months.
  7. Systemic immunosuppressive therapy post HSCT or with clinically significant graft-versus-host disease (GVHD).
  8. Prior systemic cancer-directed treatments or investigational modalities ≤ 5 half lives or 4 weeks whichever is shorter.
  9. Major surgery ≤ 2 weeks and recovered from any clinically significant effects of recent surgery.
  10. Pregnant or nursing females.
  11. Known HIV infection.
  12. Known chronic hepatitis B or C (HBV/HCV) infection.
  13. Ongoing treatment with chronic, therapeutic dosing of anti-coagulants.
  14. History of autoimmune hemolytic anemia or autoimmune thrombocytopenia.
  15. History of concurrent second cancers requiring active, ongoing systemic treatment.
  16. Subjects for whom potentially curative anticancer therapy is available.

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): NCT02641002

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United States, Arizona
Mayo Clinic Phoenix
Phoenix, Arizona, United States, 85054
United States, California
UCLA Division of Hematology Oncology
Los Angeles, California, United States, 90095-1752
United States, Connecticut
Yale Cancer Center
New Haven, Connecticut, United States, 06510
United States, Illinois
University of Chicago
Chicago, Illinois, United States, 60637
United States, Massachusetts
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
United States, New York
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10021
Sponsors and Collaborators
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Study Director: Michael Burgess, MD, PhD Celgene Corporation
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Celgene
ClinicalTrials.gov Identifier: NCT02641002    
Other Study ID Numbers: CC-90002-AML-001
First Posted: December 29, 2015    Key Record Dates
Last Update Posted: October 18, 2018
Last Verified: October 2018
Keywords provided by Celgene:
Hematologic Cancers
Acute Myeloid Leukemia
Myelodysplastic syndrome
Blood disorder
Additional relevant MeSH terms:
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Leukemia, Myeloid
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Pathologic Processes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions