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A Study of Eltrombopag in Patients With CMML and Thrombocytopenia

Sponsor
Groupe Francophone des Myelodysplasies (Other)
Overall Status
Completed
CT.gov ID
NCT02323178
Collaborator
GlaxoSmithKline (Industry), Novartis (Industry)
30
Enrollment
26
Locations
1
Arm
79.8
Actual Duration (Months)
1.2
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Treatment of patients with chronic myelomonocytic leukemia (CMML) and thrombocytopenia.

Condition or Disease Intervention/Treatment Phase
Phase 1/Phase 2

Detailed Description

All eligible patients will be treated with eltrombopag for a minimum of twelve weeks and a maximum of 24 months.

Study Design

Study Type:
Interventional
Actual Enrollment :
30 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase I/II Study of Eltrombopag in Patients With Chronic Myelomonocytic Leukemia and Thrombocytopenia
Actual Study Start Date :
Aug 7, 2014
Actual Primary Completion Date :
Dec 1, 2018
Actual Study Completion Date :
Apr 1, 2021

Arms and Interventions

Arm Intervention/Treatment
Experimental: eltrombopag

Drug: eltrombopag
initial dose of 50 mg once daily, then the dose can be sequentially increased every 2 weeks up to a maximum dose of 300mg/day
Other Names:
  • revolade

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Platelet response [12 weeks]

      Hematological improvement after twelve weeks of eltrombopag treatment

    Secondary Outcome Measures

    1. Duration of platelet response [30 months]

      Duration of platelet response at end of follow-up

    Other Outcome Measures

    1. safety of eltrombopag assessed by clinical and biological toxicity of eltrombopag evaluated using NCI CTCAE v4.0 [24 months]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Age 18 years or older

    • Chronic myelomonocytic leukemia (CMML) according to WHO criteria:

    • Stable excess in blood monocytes > 1 G/L

    • Lack of bcr-abl rearrangement (or Philadelphia chromosome)

    • Bone marrow blast cells < 20%

    • Dysplasia of at least one lineage or clonality marker or blood monocytosis during more than 3 months w/o other explanation

    • Platelet counts < 50 G/L on two successive blood counts in the 2 weeks preceding inclusion

    • Either of D1 or D2 criteria:

    • Lack of features of advanced disease If white blood cell count (WBC) < 13 G/L: International Prognostic Scoring System (IPSS) low or intermediate-1

    If WBC ≥ 13 G/L: no more than one of the following criteria:

    • Clonal cytogenetic abnormality other than t(5;12) (q33; p13)

    • Absolute neutrophil count (ANC) > 16 G/L

    • Anemia (Hb < 100 g/L)

    • Extramedullary localization (documented cutaneous, pleural or pericardial effusion, etc…) OR D2- Features of advanced disease If WBC < 13 G/L: IPSS intermediate-2 or high

    If WBC ≥ 13 G/L: two or more of the following criteria:

    • Clonal cytogenetic abnormality other than t(5;12) (q33; p13)

    • ANC > 16 G/L

    • Anemia (Hb < 100 g/L)

    • Extramedullary localization (documented cutaneous, pleural or pericardial effusion, etc…) And having resisted (progression or stable disease without hematological improvement according to International Working Group (IWG) 2006 criteria) or relapsed after a treatment with a hypomethylating agent (azacitidine or decitabine for a minimum of 6 cycles)

    • Blast cells ≤ 5% in the bone marrow

    • Performance status 0-2 on the Eastern Cooperative Oncology Group (ECOG) Scale

    • Serum Creatinin < 2 times the upper limit of normal (ULN)

    • Alanine transaminase (ALT) and aspartate transaminase (AST) < 3 ULN, total bilirubin < 1.5 ULN (except Gilbert Syndrome)

    • Adequate contraception if relevant

    • Signed informed consent

    Exclusion Criteria:

    • CMML with t(5 ;12) or Platelet-derived growth factor beta receptor (PDGFbetaR) rearrangement

    • Acute blastic transformation of CMML with bone marrow blast cells > 20%

    • Bone marrow blast cells > 5%

    • Patients eligible for allogeneic bone marrow transplantation with an identified donor

    • Intensive chemotherapy given less than 3 months before inclusion

    • Pregnant or breastfeeding

    • Hepatitis C infection

    • Splenomegaly > 16 cm by ultrasound or CT scan (Not Applicable in patients without palpable splenomegaly)

    • Significant (grade II-IV) myelofibrosis (bone marrow trephine if bone marrow aspirate with poor cellularity, or features of myelofibrosis on the peripheral blood smear (teardrop erythrocytes)

    • Clinically relevant thromboembolic risk factor which, in the investigator's opinion, is such that the benefit/risk ratio becomes unfavourable if platelet counts increase

    • Liver cirrhosis (Child-Pugh score ≥ 5)

    • Prior Cancer (except in situ cervix carcinoma, limited basal cell carcinoma, or other tumors if not active during the last 3 years)

    • Serious concomitant systemic disorder, including active bacterial, fungal or viral infection that, in the opinion of the investigator, would compromise the safety of the patient and/or his/her ability to complete the study.

    • Hypersensitivity to Eltrombopag

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 CHU d'Amiens Amiens France 80054
    2 CHU d'Angers Angers France 49 033
    3 CH Victor Dupouy Argenteuil France 95107
    4 Hôpital Avicenne Bobigny France 93009
    5 Hôpital privé Sévigné Cesson-Sévigné France 35510
    6 CHU Henri Mondor Créteil France 94010
    7 CHU de Grenoble Grenoble France 38043
    8 CH Le Mans Le Mans France 72037
    9 CHRU de Limoges Limoges France 87046
    10 Centre Hospitalier Lyon Sud Lyon France 69495
    11 Institut Paoli Calmettes Marseille France 13009
    12 Centre Hospitalier de Meaux Meaux France 77100
    13 CHU de Nantes Nantes France 44093
    14 Centre Catherine de Sienne Nantes France 44277
    15 Hôpital Archet 1 Nice France 06202
    16 Hôpital Saint Louis - Service d'hématologie AJA Paris France 75010
    17 Hôpital Saint Louis - Service d'hématologie séniors Paris France 75010
    18 CHU de Haut-Lévèque Pessac France 33604
    19 CHU de Poitiers Poitiers France 86021
    20 Centre Hospitalier de la région d'Annecy Pringy cedex France 74374
    21 Hôpital Pontchaillou Rennes France 35033
    22 Centre Henri Becquerel Rouen France 76038
    23 IUCT Oncopole - Médecine interne Toulouse France 31059
    24 IUCT Oncopole - Service d'Hématologie Clinique Toulouse France 31059
    25 CHU Brabois Vandoeuvre Les Nancy France 54511
    26 Institut Gustave Roussy Villejuif France 94805

    Sponsors and Collaborators

    • Groupe Francophone des Myelodysplasies
    • GlaxoSmithKline
    • Novartis

    Investigators

    • Principal Investigator: Raphaël Itzykson, MD, Saint-Louis Hospital, Paris, France
    • Study Director: Pierre Fenaux, MD, PHD, Saint-Louis Hospital, Paris, France

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Groupe Francophone des Myelodysplasies
    ClinicalTrials.gov Identifier:
    NCT02323178
    Other Study ID Numbers:
    • GFM-LMMC-Eltrombopag
    • 2013-001779-19
    First Posted:
    Dec 23, 2014
    Last Update Posted:
    Apr 20, 2021
    Last Verified:
    Apr 1, 2021
    Keywords provided by Groupe Francophone des Myelodysplasies
    Chronic myelomonocytic leukemia
    thrombocytopenia
    eltrombopag
    Additional relevant MeSH terms:
    Thrombocytopenia

    Study Results

    No Results Posted as of Apr 20, 2021