NCT02335268

Brief Summary

There are currently no licensed drugs in the EU to treat thrombocytopenia in MDS patients classified as IPSS low/int-1. Prior studies with romiplostim (a TPO receptor agonist) in MDS found that baseline concentration of TPO as well as transfusion history were predictive of subsequent response in a retrospective model. The current prospective study has the aim to explore whether both pretreatment variables (endogenous TPO, TPO-level, platelet transfusion history) can predict the response to subsequent short-term treatment with romiplostim.

Trial Health

90
On Track

Trial Health Score

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

Enrollment
77

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started May 2015

Longer than P75 for phase_2

Geographic Reach
2 countries

36 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

January 7, 2015

Completed
2 days until next milestone

First Posted

Study publicly available on registry

January 9, 2015

Completed
4 months until next milestone

Study Start

First participant enrolled

May 21, 2015

Completed
6.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2021

Completed
2 years until next milestone

Results Posted

Study results publicly available

July 14, 2023

Completed
Last Updated

August 16, 2023

Status Verified

July 1, 2023

Enrollment Period

6.1 years

First QC Date

January 7, 2015

Results QC Date

August 15, 2022

Last Update Submit

July 26, 2023

Conditions

Myelodysplastic Syndromes

Keywords

MDS classified as IPSS low/int-1

Outcome Measures

Primary Outcomes (1)

  • Hematologic Improvement of Platelets (HI-P) After 4 Months on Therapy

    The primary efficacy endpoint was the rate of HI-P defined as an absolute increase of platelet count to ≥ 30/nL for patients starting at \> 20/nL or an increase of platelets from \< 20/nL to \> 20/nL and by at least 100%, according to IWG 2006 criteria lasting for ≥ 8 weeks after at least 16 Weeks of romiplostim treatment.

    after 4 months on therapy (week 16)

Secondary Outcomes (6)

  • Cumulative Hematologic Improvement

    week 16

  • The Incidence of Disease Progression to Higher Stage MDS or AML

    week 16

  • Increase of Peripheral Blasts During Therapy

    week 16

  • Association of the Presence of Certain Mutations With Disease Progression in a Retrospective Analysis

    week 16

  • Incidence of Bleeding Events

    up to 12 months

  • +1 more secondary outcomes

Study Arms (3)

Group 1

EXPERIMENTAL

Stratification into group 1 if Score (Baseline-TPO Level and previous thrombocyte transfusions) are +3 TPO based model to predict subsequent response to romiplostim in MDS patients with IPSS Low/Int-1 according to a model developed by Sekeres et. al./ BJH 2014

Drug: N-Plate / romiplostim

Group 2

EXPERIMENTAL

Stratification into group 2 if Score (Baseline-TPO Level and previous thrombocyte transfusions) are -1 or -2 TPO based model to predict subsequent response to romiplostim in MDS patients with IPSS Low/Int-1 according to a model developed by Sekeres et. al./ BJH 2014

Drug: N-Plate / romiplostim

Group 3

EXPERIMENTAL

Stratification into group 3 if Score (Baseline-TPO Level and previous thrombocyte transfusions) are -6 TPO based model to predict subsequent response to romiplostim in MDS patients with IPSS Low/Int-1 according to a model developed by Sekeres et. al./ BJH 2014

Drug: N-Plate / romiplostim

Interventions

N-Plate / romiplostimDRUG

medical intervention in 3 patient groups (MDS patients with IPSS Low/Int-1) that are stratified according to their baseline TPO-Level and previous transfusions

Group 1Group 2Group 3

Eligibility Criteria

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

You may not qualify if:

  • Must understand and voluntarily sign the informed consent form
  • Age older 18 years at the time of signing the informed consent form
  • Must be able to adhere to the study visit schedule and other protocol requirements
  • Diagnosis of MDS using the 2008 WHO classification for myeloid neoplasms as assessed during the screening period
  • Per MDS IPSS, low or intermediate-1 risk MDS as assessed during the screening period
  • The mean of the 2 platelet counts taken within 4 weeks prior to stratification must be:
  • ≤ 30 x 109/L (with no individual count \> 30 x 109/L during the screening period), with or without a history of bleeding associated with the diagnosis of MDS, OR
  • \< 50 x 109/L (with no individual count \>60 x 109/L during the screening period), with a history of bleeding associated with the diagnosis of MDS (A standard of care platelet count taken prior to Informed consent may be used as 1 of the 2 counts taken within 4 weeks prior to stratification)
  • Adequate liver function, as evidenced by ALT ≤ 3 times the laboratory normal range, AST ≤ 3 times the laboratory normal range and total bilirubin ≤ 2 times the laboratory normal range
  • Bone marrow aspirate (central diagnostics) with cytogenetics (local) within 8 weeks of starting first dose of investigational product
  • Female subjects of childbearing potential† must:
  • Agree to use, and be able to comply with, effective contraception without interruption, 4 weeks before starting study drug, throughout study drug therapy and for 4 weeks after the end of study drug therapy, even if she has amenorrhoea. This applies unless the subject commits to absolute and continued abstinence confirmed on a monthly basis. Male patients who wish to participate in the study and their partner may become pregnant must agree also to reliable contraception during the study and for three months thereafter.
  • The following are effective methods of contraception\*
  • Implant, - Levonorgestrel-releasing intrauterine system (IUS), Medroxyprogesterone acetate depot, Tubal sterilization, Sexual intercourse with a vasectomised male partner only; Ovulation inhibitory progesterone-only pills (i.e., desogestrel)
  • Agree to have a medically supervised pregnancy test with a minimum sensitivity of 25 mIU/ml not more than 3 days before the start of study medication once the subject has been on effective contraception for at least 4 weeks. This requirement also applies to women of childbearing potential who practice complete and continued abstinence.
  • +17 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (36)

CH

Annecy, France

Location

CH Victor Dupouy

Argenteuil, France

Location

CH Henri Duffaut

Avignon, France

Location

CHU de Haut Lévèque

Bordeaux, France

Location

CHRU Côte de Nacre

Caen, France

Location

CHU Estaing

Clermont-Ferrand, France

Location

CHU

Grenoble, France

Location

CH

Le Mans, France

Location

CHRU

Limoges, France

Location

CH Lyon sud

Lyon, France

Location

IPC

Marseille, France

Location

CH

Meaux, France

Location

Clinique Beausoleil

Montpellier, France

Location

CHU Brabois

Nancy, France

Location

Centre Catherine de Sienne

Nantes, France

Location

Polyclinique Le Languedoc

Narbonne, France

Location

CHR

Orléans, France

Location

Hôpital Cochin

Paris, France

Location

Hôpital Saint Louis

Paris, France

Location

CHU

Poitiers, France

Location

Centre Henri Becquerel

Rouen, France

Location

Hôpital Bretonneau

Tours, France

Location

MVZ Onkologischer Schwerpunkt am Oskar-Helene-Heim

Berlin, Germany

Location

Vivantes Klinikum am Urban / Hämatologie Onkologie

Berlin, Germany

Location

Klinikum Chemnitz gGmbH / Klinik für Innere Medizin III

Chemnitz, 09116, Germany

Location

Universitätsklinikum Dresden / Medizinische Klinik I

Dresden, 01307, Germany

Location

Marienhospital Düsseldorf GmbH / Innere Medizin u. Hämatologie

Düsseldorf, Germany

Location

Universitätsklinikum Halle (Saale) / Klinik für Innere Medizin IV

Halle, 06120, Germany

Location

Universitätsklinikum Hamburg-Eppendorf /Onkologisches Zentrum

Hamburg, 20246, Germany

Location

Medizinische Hochschule Hannover / Hämatologie u. Onkologie

Hanover, Germany

Location

Universitätsklinikum Mannheim / Klinik III / Hämatologie, Onkologie

Mannheim, Germany

Location

Klinikum Rechts der Isar, Tumortherapiezentrum

München, Germany

Location

MVZ für Blut u. Krebserkrankungen

Potsdam, Germany

Location

Universitätsklinikum Ulm / Klinik Innere Medizin III

Ulm, Germany

Location

Onkologische Gemeinschaftspraxis

Weilheim, Germany

Location

Rems-Murr-Kliniken Winnenden / Hämatologie, Onkologie

Winnenden, Germany

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Results Point of Contact

Title
Dr. Carsta Köhler
Organization
GMIHO Gesellschaft für Medizinische Innovation - Hämatologie und Onkologie mbH
info@gmiho.de
+49 351 25933

Study Officials

  • Uwe Platzbecker, Prof. Dr.

    University of Dresden

    STUDY DIRECTOR

  • Lionel Ades, Prof. Dr.

    Groupe Francophone des Myelodysplasies

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 7, 2015

First Posted

January 9, 2015

Study Start

May 21, 2015

Primary Completion

July 1, 2021

Study Completion

July 1, 2021

Last Updated

August 16, 2023

Results First Posted

July 14, 2023

Record last verified: 2023-07

Locations

FR(22)DE(14)