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A Study of Eltrombopag in Patients With CMML and Thrombocytopenia

Phase 1
Completed
Conditions
CMML
Thrombocytopenia
Interventions
Registration Number
NCT02323178
Lead Sponsor
Groupe Francophone des Myelodysplasies
Brief Summary

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

Detailed Description

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

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
30
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
Read More
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
Read More

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
eltrombopageltrombopag-
Primary Outcome Measures
NameTimeMethod
Platelet response12 weeks

Hematological improvement after twelve weeks of eltrombopag treatment

Secondary Outcome Measures
NameTimeMethod
Duration of platelet response30 months

Duration of platelet response at end of follow-up

Trial Locations

Locations (26)

CHU d'Angers

🇫🇷

Angers, France

Hôpital privé Sévigné

🇫🇷

Cesson-Sévigné, France

Centre Hospitalier de Meaux

🇫🇷

Meaux, France

Centre Catherine de Sienne

🇫🇷

Nantes, France

CHU de Haut-Lévèque

🇫🇷

Pessac, France

Centre Henri Becquerel

🇫🇷

Rouen, France

CH Victor Dupouy

🇫🇷

Argenteuil, France

Hôpital Avicenne

🇫🇷

Bobigny, France

CHU Henri Mondor

🇫🇷

Créteil, France

CHRU de Limoges

🇫🇷

Limoges, France

CHU de Grenoble

🇫🇷

Grenoble, France

CH Le Mans

🇫🇷

Le Mans, France

Institut Paoli Calmettes

🇫🇷

Marseille, France

CHU de Nantes

🇫🇷

Nantes, France

Hôpital Archet 1

🇫🇷

Nice, France

Hôpital Saint Louis - Service d'hématologie AJA

🇫🇷

Paris, France

Hôpital Saint Louis - Service d'hématologie séniors

🇫🇷

Paris, France

CHU de Poitiers

🇫🇷

Poitiers, France

Centre Hospitalier de la région d'Annecy

🇫🇷

Pringy cedex, France

Hôpital Pontchaillou

🇫🇷

Rennes, France

IUCT Oncopole - Médecine interne

🇫🇷

Toulouse, France

IUCT Oncopole - Service d'Hématologie Clinique

🇫🇷

Toulouse, France

CHU Brabois

🇫🇷

Vandoeuvre Les Nancy, France

Institut Gustave Roussy

🇫🇷

Villejuif, France

Centre Hospitalier Lyon Sud

🇫🇷

Lyon, France

CHU d'Amiens

🇫🇷

Amiens, France

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