A Study of Eltrombopag in Patients With CMML and Thrombocytopenia

NCT ID: NCT02323178

Last Updated: 2021-04-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-08-07

Study Completion Date

2021-04-30

Brief Summary

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

Detailed Description

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

Conditions

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CMML Thrombocytopenia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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eltrombopag

Group Type EXPERIMENTAL

eltrombopag

Intervention Type DRUG

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

Interventions

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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

Intervention Type DRUG

Other Intervention Names

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revolade

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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GlaxoSmithKline

INDUSTRY

Sponsor Role collaborator

Novartis

INDUSTRY

Sponsor Role collaborator

Groupe Francophone des Myelodysplasies

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Raphaël Itzykson, MD

Role: PRINCIPAL_INVESTIGATOR

Saint-Louis Hospital, Paris, France

Pierre Fenaux, MD, PHD

Role: STUDY_DIRECTOR

Saint-Louis Hospital, Paris, France

Locations

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CHU d'Amiens

Amiens, , France

Site Status

CHU d'Angers

Angers, , France

Site Status

CH Victor Dupouy

Argenteuil, , France

Site Status

Hôpital Avicenne

Bobigny, , France

Site Status

Hôpital privé Sévigné

Cesson-Sévigné, , France

Site Status

CHU Henri Mondor

Créteil, , France

Site Status

CHU de Grenoble

Grenoble, , France

Site Status

CH Le Mans

Le Mans, , France

Site Status

CHRU de Limoges

Limoges, , France

Site Status

Centre Hospitalier Lyon Sud

Lyon, , France

Site Status

Institut Paoli Calmettes

Marseille, , France

Site Status

Centre Hospitalier de Meaux

Meaux, , France

Site Status

CHU de Nantes

Nantes, , France

Site Status

Centre Catherine de Sienne

Nantes, , France

Site Status

Hôpital Archet 1

Nice, , France

Site Status

Hôpital Saint Louis - Service d'hématologie AJA

Paris, , France

Site Status

Hôpital Saint Louis - Service d'hématologie séniors

Paris, , France

Site Status

CHU de Haut-Lévèque

Pessac, , France

Site Status

CHU de Poitiers

Poitiers, , France

Site Status

Centre Hospitalier de la région d'Annecy

Pringy, , France

Site Status

Hôpital Pontchaillou

Rennes, , France

Site Status

Centre Henri Becquerel

Rouen, , France

Site Status

IUCT Oncopole - Médecine interne

Toulouse, , France

Site Status

IUCT Oncopole - Service d'Hématologie Clinique

Toulouse, , France

Site Status

CHU Brabois

Vandœuvre-lès-Nancy, , France

Site Status

Institut Gustave Roussy

Villejuif, , France

Site Status

Countries

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France

Other Identifiers

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2013-001779-19

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GFM-LMMC-Eltrombopag

Identifier Type: -

Identifier Source: org_study_id

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