A clinical study comparing Epoetin Alfa with Placebo in Anaemic patients with MDS

• Register: EUCTR
• Last refreshed on: 11 April 2016
• Main ID: EUCTR2010-022884-36-IT
• Date of registration: 23/01/2012
• Prospective Registration: No
• Primary sponsor: JANSSEN-CILAG INTERNATIONAL N.V.
• Public title: A clinical study comparing Epoetin Alfa with Placebo in Anaemic patients with MDS
• Scientific title: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study Evaluating Epoetin Alfa Versus Placebo in Anemic Patients With IPSS Low- or Intermediate 1 Risk Myelodysplastic Syndromes
• Date of first enrolment: 03/08/2011
• Target sample size: 159
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-022884-36
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2

Countries of recruitment

Bulgaria, Greece, Italy, Russian Federation

Contacts

• Name: CGO
• Affiliation: JANSSEN CILAG SPA

• Name: CGO
• Affiliation: JANSSEN CILAG SPA

Key inclusion & exclusion criteria

Inclusion criteria:
• Male or female subjects at least18 years of age; • diagnosis of MDS at screening, according to World Health Organization (WHO) or French-American-British (FAB) Cooperative Group pathologic classification and confirmed via bone marrow aspirate; • documentation of an IPSS score indicating Low- or Intermediate 1 risk disease; • hemoglobin concentration at screening and baseline (before the first dose of study drug) of 10.0 g/dL or less, based on untransfused hemoglobin (ie, the subject did not receive an RBC transfusion within the previous week); • screening serum erythropoietin concentration of less than 500 mU/mL; • RBC transfusion requirement of less than 4 RBC units over the last 8 weeks before randomization; • screening Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2; • adequate iron stores, defined as serum ferritin greater than 100 ng/mL, transferrin saturation greater than 20%, or both, measured within the 2-week screening period, or adequate iron stores as demonstrated by recent (within 12 weeks) bone marrow examination with iron stain; • documentation of adequate vitamin B12 and folate levels (above the lower limit of the reference range for the laboratory performing the assay), measured at screening and baseline.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 48
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 111

Exclusion criteria:
• Anemia attributed to factors other than MDS; • secondary MDS (ie, MDS arising after chemotherapy, immunotherapy, or radiation therapy/exposure; • active malignancy within the past year, except basal cell or carcinoma in situ of the cervix or breast; • prior therapy with epoetin alfa or any other approved or experimental ESA for more than 8 weeks before randomization; • prior use of approved or experimental agents for the treatment of MDS; • history (within 6 months) of deep vein thrombosis (including proximal and distal), pulmonary embolism, or other venous thrombosis.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Anemia due to IPSS Low- or Intermediate-1-Risk Myelodysplastic Syndromes
MedDRA version: 14.1 Level: LLT Classification code 10002272 Term: Anemia System Organ Class: 10005329 - Blood and lymphatic system disorders

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Intervention(s)

Trade Name: EPREX*1SIR 40000UI/ML 1ML
Pharmaceutical Form: Solution for injection
INN or Proposed INN: EPOETIN ALFA
CAS Number: 113427-24-0
Concentration unit: IU/ml international unit(s)/millilitre
Concentration type: equal
Concentration number: 40000-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: 24 weeks

Main Objective: The primary objective is: ? To demonstrate that epoetin alfa treatment is better at improving anemia outcome (as evaluated by Erythroid response – International Working Group [IWG] 2006) in subjects with International Prognostic Scoring Systems (IPSS) Low- or Intermediate-1-risk myelodysplastic syndromes (MDS), compared with placebo, through Week 24.

Primary end point(s): The primary efficacy endpoint is the proportion of subjects at Week 24 with improved anemia outcome, defined as Erythroid response according to the IWG 2006 criteria.

Secondary Objective: The secondary objectives include: • For responders at Week 24, observe the duration of the response through Week 48. • To assess the proportion of responders at Week 24 maintaining response through Week 48 (as measured by Erythroid response – IWG 2006). • To compare time to first RBC transfusion, transfusion-free intervals, and number of RBC units transfused. • To measure and compare changes in patient-reported outcome (PRO)/quality of life from baseline via the Functional Assessment of Cancer Therapy ? Anemia/Fatigue (FACT-An) and EuroQol 5 dimension (EQ-5D) questionnaires. • To collect medical resource utilization data that may be used in future economic modeling (the construction and reporting of the economic model will be conducted separately from this study). Overall safety will also be assessed.

Secondary Outcome(s)

Secondary end point(s): • For responders at Week 24, the duration of the response through Week 48 • The proportion of responders at Week 24 maintaining response through Week 48 (as measured by Erythroid response – IWG 2006). • Time to first RBC transfusion, transfusion-free intervals, and number of RBC units transfused. • Change from baseline in PRO/quality of life (as assessed with the FACT-An and EQ-5D).

Timepoint(s) of evaluation of this end point: 48 weeks

Secondary ID(s)

EPOANE3021

2010-022884-36-GR

Source(s) of Monetary Support

JANSSEN CILAG SPA