NCT02323178

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Aug 2014

Longer than P75 for phase_1

Geographic Reach
1 country

26 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 26, 2014

Completed
2 months until next milestone

Study Start

First participant enrolled

August 7, 2014

Completed
5 months until next milestone

First Posted

Study publicly available on registry

December 23, 2014

Completed
3.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2018

Completed
2.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2021

Completed
Last Updated

April 20, 2021

Status Verified

April 1, 2021

Enrollment Period

4.3 years

First QC Date

May 26, 2014

Last Update Submit

April 19, 2021

Conditions

CMMLThrombocytopenia

Keywords

Chronic myelomonocytic leukemiathrombocytopeniaeltrombopag

Outcome Measures

Primary Outcomes (1)

  • Platelet response

    Hematological improvement after twelve weeks of eltrombopag treatment

    12 weeks

Secondary Outcomes (1)

  • Duration of platelet response

    30 months

Other Outcomes (1)

  • safety of eltrombopag assessed by clinical and biological toxicity of eltrombopag evaluated using NCI CTCAE v4.0

    24 months

Study Arms (1)

eltrombopag

EXPERIMENTAL
Drug: eltrombopag

Interventions

eltrombopagDRUG

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

Also known as: revolade
eltrombopag

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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
  • 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)
  • +9 more criteria

You may not qualify if:

  • 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
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (26)

CHU d'Amiens

Amiens, 80054, France

Location

CHU d'Angers

Angers, 49 033, France

Location

CH Victor Dupouy

Argenteuil, 95107, France

Location

Hôpital Avicenne

Bobigny, 93009, France

Location

Hôpital privé Sévigné

Cesson-Sévigné, 35510, France

Location

CHU Henri Mondor

Créteil, 94010, France

Location

CHU de Grenoble

Grenoble, 38043, France

Location

CH Le Mans

Le Mans, 72037, France

Location

CHRU de Limoges

Limoges, 87046, France

Location

Centre Hospitalier Lyon Sud

Lyon, 69495, France

Location

Institut Paoli Calmettes

Marseille, 13009, France

Location

Centre Hospitalier de Meaux

Meaux, 77100, France

Location

CHU de Nantes

Nantes, 44093, France

Location

Centre Catherine de Sienne

Nantes, 44277, France

Location

Hôpital Archet 1

Nice, 06202, France

Location

Hôpital Saint Louis - Service d'hématologie AJA

Paris, 75010, France

Location

Hôpital Saint Louis - Service d'hématologie séniors

Paris, 75010, France

Location

CHU de Haut-Lévèque

Pessac, 33604, France

Location

CHU de Poitiers

Poitiers, 86021, France

Location

Centre Hospitalier de la région d'Annecy

Pringy, 74374, France

Location

Hôpital Pontchaillou

Rennes, 35033, France

Location

Centre Henri Becquerel

Rouen, 76038, France

Location

IUCT Oncopole - Médecine interne

Toulouse, 31059, France

Location

IUCT Oncopole - Service d'Hématologie Clinique

Toulouse, 31059, France

Location

CHU Brabois

Vandœuvre-lès-Nancy, 54511, France

Location

Institut Gustave Roussy

Villejuif, 94805, France

Location

MeSH Terms

Conditions

ThrombocytopeniaLeukemia, Myelomonocytic, Chronic

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Blood Platelet DisordersHematologic DiseasesHemic and Lymphatic DiseasesCytopeniaLeukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsMyelodysplastic-Myeloproliferative DiseasesBone Marrow DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Raphaël Itzykson, MD

    Saint-Louis Hospital, Paris, France

    PRINCIPAL INVESTIGATOR

  • Pierre Fenaux, MD, PHD

    Saint-Louis Hospital, Paris, France

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 26, 2014

First Posted

December 23, 2014

Study Start

August 7, 2014

Primary Completion

December 1, 2018

Study Completion

April 1, 2021

Last Updated

April 20, 2021

Record last verified: 2021-04

Locations

FR(26)