Myelodysplastic syndrome (MDS) MedDRA version: 18.0 Level: HLT Classification code 10028536 Term: Myelodysplastic syndromes System Organ Class: 100000004851
Conditions
Interventions
Product Name: Imetelstat sodium
Product Code: JNJ-63935937
Pharmaceutical Form: Lyophilisate for solution for infusion
INN or Proposed INN: Imetelstat sodium
CAS Number: 1007380-31-5
Current Sponsor
Sponsors
Janssen-Cilag International NV
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Man or woman greater than or equal to (>=) 18 years of age - Diagnosis of myelodysplastic syndrome (MDS) according to WHO criteria or French-American-British (FAB) classification confirmed by bone marrow aspirate and biopsy within 12 weeks prior to Study Entry. A local laboratory report from this diagnostic bone marrow aspirate and biopsy must be reviewed and approved by the sponsor - International Prognostic Scoring System (IPSS) low Risk or intermediate-1 risk MDS - Red blood cell (RBC) transfusion dependent, defined as requiring 4 units RBC over 8 weeks during the 12 weeks prior to Study Entry; pre-transfusion hemoglobin (Hb) should be less than equal to 9.0 gram per deciliter (g/dL) to count towards the 4 units total - Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2 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 60 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 140
Exclusion criteria
Exclusion criteria: - Participant has known allergies, hypersensitivity, or intolerance to imetelstat or its excipients - Participant has received an investigational drug or used an invasive investigational medicaldevice within 30 days prior to Study Entry or is currently enrolled in an investigational study - Prior treatment with imetelstat - Have received any chemotherapy, immunomodulatory or immunosuppressive therapy, corticosteroids greater than 30 milligram per day prednisone or equivalent, or growth factor treatment within 28 days prior to study entry - Have received other treatments for MDS within 4 weeks prior to Study Entry
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Part 1: To evaluate the efficacy and safety of imetelstat in transfusion dependent subjects with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment. Part 2: To compare the efficacy, in terms of RBC TI, of imetelstat to placebo in transfusion dependent subjects with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment.;Secondary Objective: - To assess the safety of imetelstat in subjects with MDS - To assess the time to RBC TI and duration of RBC TI - To assess the rate of hematologic improvement - To assess the rates of CR or PR - To assess OS - To assess time to progression to AML - To assess the rate and amount of supportive care, including transfusions and myeloid growth factors - To evaluate the pharmacokinetics and immunogenicity of imetelstat in subjects with MDS - To assess the effect of imetelstat treatment on patient reported outcomes (PROs) - To assess the effect of treatment on medical resource utilization;Primary end point(s): Percentage of participants without any red blood cell (RBC) transfusion during any consecutive 8 week period.;Timepoint(s) of evaluation of this end point: 8 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1/ Number of Participants with Adverse Events (AEs) 2/ Percentage of participants without any red blood cell (RBC) transfusion during any consecutive 24 week period 3/ Time to the 8-week RBC transfusion independence (TI) 4/ Duration of RBC TI 5/ Percentage of Participants with hematologic improvement 6/ Percentage of Participants with Complete remission (CR) or Partial remission (PR) as Per International Working Group (IWG) Response CriteriaI 2006 7/ Overall survival 8/ Time to Progression to Acute Myeloid Leukemia 9/ Percentage of Participants with Transfusion 10/ Amount of Transfusions 11/ Percentage of Participants receiving any myeloid growth factors 12/ Change from baseline in Functional Assessment of Cancer Therapy -Anemia-Related Effects (FACT-An) Score and EuroQol-EQ-5D-5L ESA (EQ-5D-5L) Score 13/ Maximum Observed Plasma Concentration (Cmax) 14/ Area under the drug concentrationplasma time curve from time zero to last measurable concentration (AUC0-t) 15/ Percentage of Participants with antibodies to imetelstat 16/ Medical resource utilization data;Timepoint(s) of evaluation of this end point: 1/ up to follow-up (30 days posttreatment [approximately 2 years]) 2-12/ up to 2 years after enrollment of the last participant 13-14/ During treatment (approximately 2 years) 15-16/ up to 2 years after enrollment of the last participant | — |
Countries
Belgium, Czech Republic, France, Germany, Italy, Korea, Republic of, Mexico, Netherlands, Poland, Russian Federation, Spain, Switzerland, United Kingdom, United States
Contacts
Public ContactClinical Registry Group
Janssen-Cilag International NV
Outcome results
None listed