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A Dose-finding Study of CC-90009 in Subjects With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher-risk Myelodysplastic Syndromes

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02848001
Recruitment Status : Recruiting
First Posted : July 28, 2016
Last Update Posted : December 3, 2019
Information provided by (Responsible Party):

Brief Summary:
CC-90009-AML-001 is a phase 1, open-label, dose escalation and expansion, study in subjects with relapsed or refractory acute myeloid leukemia and relapsed or refractory high-risk myelodysplastic syndrome.

Condition or disease Intervention/treatment Phase
Leukemia, Myeloid, Acute Myelodysplastic Syndromes Drug: CC-90009 Phase 1

Detailed Description:

Study CC-90009-AML-001 is an open-label, Phase 1, dose escalation and expansion, first-in-human clinical study of CC-90009 in subjects with relapsed or refractory acute myeloid leukemia (AML) and relapsed or refractory high-risk myelodysplastic syndrome.

The dose escalation part (Part A) of the study will evaluate the safety and tolerability of escalating doses of CC-90009 in relapsed and refractory AML. The expansion part, (Part B), will further evaluate the safety and efficacy of CC-90009 administered at or below the maximum tolerated dose (MTD) in selected expansion cohorts of one or more dosing regimens in order to determine the recommended Phase 2 dose (RP2D) for subjects with relapsed or refractory AML and relapsed or refractory high-risk myelodysplastic syndrome.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1, Open-label, Dose Finding Study of CC-90009, a Novel Cereblon E3 Ligase Modulating Drug, in Subjects With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher-Risk Myelodysplastic Syndromes
Actual Study Start Date : November 14, 2016
Estimated Primary Completion Date : August 25, 2021
Estimated Study Completion Date : February 26, 2022

Arm Intervention/treatment
Experimental: CC-90009 - Part A
Will be administered intravenously per dosing schedule in a 28-day cycle.
Drug: CC-90009

Experimental: CC-90009 - Part B - AML and MDS patients
Relapsed or refractory AML and MDS subjects. IP will be administered intravenously per dosing schedule determined in Part A
Drug: CC-90009

Primary Outcome Measures :
  1. Dose- limiting toxicity (DLT) [ Time Frame: Up to 42 days ]
    Number of participants with a DLT

  2. Non-tolerated dose (NTD) [ Time Frame: Up to 42 days ]
    Dose level at which 2 or more of up to 6 evaluable subjects in any dose cohort experience a DLT in Cycle 1 during dose escalation.

  3. Maximum tolerated dose (MTD) [ Time Frame: Up to 42 days ]
    Last dose level(s) below the NTD with 0 or 1 out of 6 evaluable subjects experiencing a DLT in Cycle 1 during dose escalation

Secondary Outcome Measures :
  1. Pharmacokinetics-Cmax [ Time Frame: Up to Day 11 ]
    Maximum observed concentration in plasma

  2. Pharmacokinetics - AUC24 [ Time Frame: Up to Day 11 ]
    Area under the plasma concentration time-curve from time 0 to 24 hours

  3. Pharmacokinetics - tmax [ Time Frame: Up to Day 11 ]
    Time to peak (maximum) plasma concentration

  4. Pharmacokinetics - t 1/2 [ Time Frame: Up to Day 11 ]
    terminal half-life

  5. Pharmacokinetics - CL [ Time Frame: Up to Day 11 ]
    Total body clearance of the drug from plasma

  6. Pharmacokinetics - Vss [ Time Frame: Up to Day 11 ]
    Volume of distribution at steady-state

  7. Preliminary efficacy of CC-90009 [ Time Frame: Up to 2.5 years ]
    Determined by acute myeloid leukemia (AML) response rate based on the International Working Group (IWG) Response Criteria in AML (Cheson, 2003)

  8. Preliminary efficacy of CC-90009 - AML [ Time Frame: Up to 2.5 years ]
    Determined by acute myeloid leukemia (AML) response rate based on the International Working Group (IWG) Response Criteria in AML.

  9. Preliminary efficacy of CC-90009 - MDS [ Time Frame: Up to 2.5 years ]
    Determined by myelodysplastic syndrome (MDS) response rate based on the International Working Group (IWG) response criteria for Myelodysplasia.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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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 ICD (Informed Consent Document).
  2. Subject must understand and voluntarily sign an ICD prior to any study-related assessments/procedures being conducted.
  3. Relapsed or refractory AML (Acute Myeloid Leukemia) (Parts A and B) or relapsed or refractory high-risk MDS (Myelodysplastic Syndrome) (Part B only) as defined by World Health Organization criteria who are not suitable for other established therapies.
  4. Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2.
  5. At least 4 weeks (from first dose) has elapsed from donor lymphocyte infusion (DLI) without conditioning.
  6. Subjects must have the following screening laboratory values:

    • Total White Blood Cell count (WBC) < 25 x 109/L prior to first infusion. Prior or concurrent treatment with hydroxyurea to achieve this level is allowed.
    • Selected electrolytes within normal limits or correctable with supplements.
    • Serum bilirubin ≤ 1.5 x ULN (upper limit of normal).
    • Estimated serum creatinine clearance of ≥ 60 mL/min using the Cockcroft-Gault equation.
  7. Agree to follow the CC-90009 Pregnancy Prevention Plan (PPP)

Exclusion Criteria:

  1. Subjects with acute promyelocytic leukemia (APL)
  2. Subjects with clinical symptoms suggesting active central nervous system (CNS) leukemia or known CNS leukemia. Evaluation of cerebrospinal fluid is only required if there is clinical suspicion of CNS involvement by leukemia during screening.
  3. Patients with prior autologous hematopoietic stem cell transplant who, in the investigator's judgment, have not fully recovered from the effects of the last transplant (e.g., transplant related side effects).
  4. Prior allogeneic hematopoietic stem cell transplant (HSCT) with either standard or reduced intensity conditioning ≤ 6 months prior to starting CC-90009.
  5. Subjects on systemic immunosuppressive therapy post HSCT at the time of screening, or with clinically significant graft-versus-host disease (GVHD). The use of topical steroids for ongoing skin or ocular GVHD is permitted.
  6. Prior systemic cancer-directed treatments or investigational modalities ≤ 5 half lives or 4 weeks prior to starting CC-90009, whichever is shorter. Hydroxyurea is allowed to control peripheral leukemia blasts.
  7. Leukapheresis ≤ 2 weeks prior to starting CC-90009.

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 identifier (NCT number): NCT02848001

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Contact: Associate Director Clinical Trial Disclosure 1-888-260-1599

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United States, Connecticut
Yale Cancer Center Recruiting
New Haven, Connecticut, United States, 06510
United States, Illinois
Northwestern Memorial Recruiting
Chicago, Illinois, United States, 60611
United States, Massachusetts
Dana Farber Cancer Institute Recruiting
Boston, Massachusetts, United States, 02115
United States, Missouri
Washington University Siteman Cancer Center Recruiting
Saint Louis, Missouri, United States, 63110
United States, New Jersey
Hackensack University Medical Center Recruiting
Hackensack, New Jersey, United States, 07601
Canada, Ontario
Princess Margaret Hospital University Health Network Recruiting
Toronto, Ontario, Canada, M5G 2M9
Institut Paoli Calmettes Not yet recruiting
Marseille Cedex 9, France, 13273
Institut Gustave Roussy Faculte de Medecine Paris Sud Service de pneumologie Not yet recruiting
Villejuif, France, 94800
Spedali Civili Di Brescia Not yet recruiting
Brescia, Italy, 25123
A.O. Ospedale Ca Granda - Niguarda Not yet recruiting
Milano, Italy, 20162
Haukeland University Hospital Recruiting
Bergen, Norway, N-5053
Oslo University Hospital, Rikshospitalet HF Recruiting
Oslo, Norway, N-0027
H Clinic I Provincial Recruiting
Barcelona, Spain, 08036
Hospital Clinico Universitario de Salamanca Recruiting
Salamanca, Spain, 37007
Hosptial La Fe Recruiting
Valencia, Spain, 46009
United Kingdom
The Churchill Hospital Recruiting
Oxford, United Kingdom, 0X3 7LE
Sponsors and Collaborators
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Study Director: Michael Pourdehnad, MD Celgene Corporation
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Responsible Party: Celgene Identifier: NCT02848001    
Other Study ID Numbers: CC-90009-AML-001
2017-001535-39 ( EudraCT Number )
First Posted: July 28, 2016    Key Record Dates
Last Update Posted: December 3, 2019
Last Verified: December 2019
Keywords provided by Celgene:
Hematologic Cancers
Acute Myeloid Leukemia
Myelodysplastic Syndrome
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