Patients with IPSS low or intermediate-1 risk myelodysplastic syndrome (MDS) and thrombocytopenia MedDRA version: 20.0 Level: LLT Classification code 10068361 Term: MDS System Organ Class: 100000004864
Conditions
Interventions
Trade Name: Nplate
Product Name: Nplate
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: N-plate
CAS Number: 267639-76-9
Current Sponsor code: AMG531
Other descriptive name:
Sponsors
GMIHO Gesellschaft für Medizinische Innovation - Hämatologie und Onkologie mbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects must meet the following inclusion criteria tobe eligible for the study, 1. Must understand and voluntarily sign an informed consent form 2. Age > 18 years at the time of signing the informed consent form 3. Must be able to adhere to the study visit schedule and other protocol requirements 4. Diagnosis of MDS using the WHO classification for myeloid neoplasms (Vardiman et al, 2002) as assessed during the screening period 5. Per MDS IPSS, low or intermediate-1 risk MDS as assessed during the screening period 6. 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 • 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 randomization) 7. 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 8. Bone marrow aspirate (central diagnostics) with cytogenetics (local) within 8 weeks of starting first dose of investigational product 9. Female subjects of childbearing potential† must: o 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 Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 75 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 75
Exclusion criteria
Exclusion criteria: 1. Pregnant or lactating females 2. IPSS intermediate-2 or high-risk 3. = 5% blasts in the bone marrow as determined by central morphology during screening 4. Previous treatment with any thrombopoietic growth factor 5. Prior history of hematopoietic stem cell transplantation 6. Active or uncontrolled disease including infections or cancer 7. Unstable angina, congestive heart failure (NYHA > class II), uncontrolled hypertension 8. History of arterial thrombosis (eg, stroke or transient ischemic attack) within the past year 9. History of venous thrombosis that currently requires anti-coagulation therapy 10. Received IL-11 within 4 weeks of the first dose of investigational product 11. Receipt of G-CSF, peg-G-CSF, or GM-CSF within 4 weeks of the first dose of investigational product 12. Planned receipt of peg-G-CSF or GM-CSF after the first dose of investigational product 13. Subjects of reproductive potential who are not using adequate contraceptive precautions, in the judgment of the investigator. The sponsor recommends double barrier contraception is used for all applicable patients enrolled on this study. A double barrier method is defined as 2 methods of contraception, for example 2 actual barrier methods, or 1 actual barrier method and 1 hormonal method. 14. Known hypersensitivity to any recombinant E coli-derived product (eg, Infergen?, Neupogen?, Somatropin, and Actimmune) 15. Inability to comply with study procedures. 16. Subject currently is enrolled in or has not yet completed at least 30 days since ending other investigational device or drug study(s) 17. Any serious medical condition or psychiatric illness that will prevent the subject from signing the informed consent form or will place the subject at unacceptable risk if he/she participates in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to investigate prospectively whether the current TPO level based response model can predict response to romiplostim in thrombocytopenic patients with IPPS low/int-1 MDS;Secondary Objective: Secondary objectives are Safety, bleeding events, AML evolution, peripheral blasts during therapy, identification of molecular parameters associated with response and progression;Primary end point(s): • Hematologic improvement of platelets (HI-P) after 4 months on therapy;Timepoint(s) of evaluation of this end point: after 4 months on therapy | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Cumulative hematologic improvement of platelets (HI-P), erythrocytes (HI-E) and neutrophils (HI-N) • The incidence of disease progression to higher stage MDS or AML • Increase of peripheral blasts during therapy • Association of the presence of certain mutations with disease progression in a retrospective analysis • Incidence of bleeding events • Type, incidence and severity of all adverse events including clinically significant changes in laboratory values ;Timepoint(s) of evaluation of this end point: End of study for all patients | — |
Countries
France, Germany
Contacts
Public ContactSponsor Office
GMIHO Gesellschaft für Medizinische Innovation - Hämatologie und Onkologie mbH
Outcome results
None listed