Back to resultsA Study Comparing Siltuximab Plus Best Supportive Care to Placebo Plus Best Supportive Care in Anemic Patients With International Prognostic Scoring System Low- or Intermediate-1-Risk Myelodysplastic Syndrome
Primary Purpose
Myelodysplastic Syndrome
Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Siltuximab
Placebo
Best supportive care (BSC)
Sponsored by
About this trial
This is an interventional treatment trial for Myelodysplastic Syndrome focused on measuring Myelodysplastic Syndrome, MDS, Blood and lymphatic diseases, Siltuximab, Anemic
Eligibility Criteria
Inclusion Criteria:
- Confirmed diagnosis of myelodysplastic syndrome (MDS), according to World Heath Organization or the French-American-British Cooperative Group pathologic classification, with an International Prognostic Scoring System score 0, 0.5, or 1.0, indicating Low- or INT-1-risk disease.
- Documented RBC transfusion of at least 2 units of RBC for the treatment of the anemia of MDS in the 8 weeks preceding the start of the Screening Period.
- Adequate iron stores, demonstrated by either the presence of stainable iron in the bone marrow or a serum ferritin of > 100 ng/mL.
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 2.
- Symptomatic anemia (defined by a score > 0 on the Non-Chemotherapy Anemia Symptom Scale [NCA-SS]).
Exclusion Criteria:
- Had treatment with drugs or other agents targeting IL-6 or its receptor within 4 weeks of randomization.
- Any condition that, in the opinion of the investigator, would make participation not in the best interest (eg, compromise the well-being) of the patient or that could prevent, limit, or confound the protocol-specified assessments.
- Patients with Chronic Myelomonocytic Leukemia (CMML).
- Causes other than MDS contributing to anemia, such as Vitamin B12 or folate deficiency, bleeding, hemolysis, hemoglobinopathy, or chronic renal failure.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Siltuximab
Placebo
Arm Description
15 mg/kg of siltuximab administered as a 1-hour infusion every 4 weeks + best supportive care (BSC)
Placebo administered as a 1-hour infusion every 4 weeks + BSC
Outcomes
Primary Outcome Measures
Percentage of Participants Who Achieved a Reduction in Red Blood Cell (RBC) Transfusions to Treat Anemia of Myelodysplastic Syndrome (MDS)
Reduction in RBC transfusions to treat the anemia of MDS is defined as a ≥50 percentage relative decrease and a ≥2 unit absolute decrease in RBC transfusions in the 8 weeks before the unblinding (scheduled to occur after 12 weeks of treatment) compared with RBC transfusions in the 8 weeks before the date the informed consent form was signed.
Secondary Outcome Measures
Change From Baseline in the Mean Hemoglobin Concentrations at Week 13
Percentage of Participants Achieving Hemoglobin Improvement (≥1.5 g/dL Increase From Baseline) Unrelated to Red Blood Cell (RBC) Transfusion at Week 13
Percentage of Participants Who Did Not Require a Red Blood Cell (RBC) Transfusions to Treat Anemia of Myelodysplastic Syndrome (MDS) in the 8 Weeks of Treatment Before Unblinding at Week 13
Mean Changes From Baseline in Percentages of Bone Marrow Blast Cells at Week 13
Median Number of Red Blood Cell (RBC) Transfusions to Treat Anemia of Myelodysplastic Syndrome (MDS) During the 8 Weeks of Treatment Before Unblinding at Week 13
Full Information
NCT ID
NCT01513317
First Posted
October 21, 2011
Last Updated
September 24, 2014
Sponsor
Janssen Research & Development, LLC
1. Study Identification
Unique Protocol Identification Number
NCT01513317
Brief Title
A Study Comparing Siltuximab Plus Best Supportive Care to Placebo Plus Best Supportive Care in Anemic Patients With International Prognostic Scoring System Low- or Intermediate-1-Risk Myelodysplastic Syndrome
Official Title
A Phase 2, Randomized, Double-blind, Placebo-controlled, Multicenter Study Comparing Siltuximab Plus Best Supportive Care to Placebo Plus Best Supportive Care in Anemic Subjects With International Prognostic Scoring System Low- or Intermediate-1-Risk Myelodysplastic Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
September 2014
Overall Recruitment Status
Terminated
Why Stopped
The study was stopped after the interim analysis based on lack of sufficient efficacy. There were no safety concerns.
Study Start Date
November 2011 (undefined)
Primary Completion Date
September 2012 (Actual)
Study Completion Date
September 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Janssen Research & Development, LLC
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to evaluate the efficacy of siltuximab, demonstrated by a reduction in red blood cell (RBC), transfusions to treat the anemia of Myelodysplastic Syndrome (MDS).
Detailed Description
The study treatments will be administered double-blind for 12 weeks, meaning that the patient and study personnel will not know the identity of the treatment. Approximately 75 patients will be randomized (patients are assigned to a treatment by a chance) in a 2:1 ratio to receive siltuximab plus best supportive care (BSC) (Group A) or placebo plus BSC (Group B). BSC includes RBC transfusion, antimicrobials, white blood cell (WBC) growth factors, and platelet transfusions. Patients who complete 12 weeks of treatment may qualify to receive siltuximab as open-label (identity of treatment will be known) treatment. Treatment may continue until death, unacceptable toxicity, withdrawal of consent, or the clinical cutoff (defined as 24 weeks after the last patient is randomized), whichever occurs first. The study will end approximately 36 weeks after the last patient is randomized. Patient safety will be monitored. Siltuximab and matching placebo will be supplied as a sterile, lyophilized formulation for reconstitution and intravenous (IV) infusion. Group A: siltuximab (15 mg/kg) administered as a 1-hour infusion every 4 weeks + BSC, or Group B: placebo administered as a 1-hour infusion every 4 weeks + BSC.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndrome
Keywords
Myelodysplastic Syndrome, MDS, Blood and lymphatic diseases, Siltuximab, Anemic
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
76 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Siltuximab
Arm Type
Experimental
Arm Description
15 mg/kg of siltuximab administered as a 1-hour infusion every 4 weeks + best supportive care (BSC)
Arm Title
Placebo
Arm Type
Experimental
Arm Description
Placebo administered as a 1-hour infusion every 4 weeks + BSC
Intervention Type
Drug
Intervention Name(s)
Siltuximab
Intervention Description
15 mg/kg administered as a 1-hour intravenous infusion every 4 weeks
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Administered as a 1-hour intravenous infusion every 4 weeks
Intervention Type
Drug
Intervention Name(s)
Best supportive care (BSC)
Intervention Description
Best supportive care according to local standards and guidelines
Primary Outcome Measure Information:
Title
Percentage of Participants Who Achieved a Reduction in Red Blood Cell (RBC) Transfusions to Treat Anemia of Myelodysplastic Syndrome (MDS)
Description
Reduction in RBC transfusions to treat the anemia of MDS is defined as a ≥50 percentage relative decrease and a ≥2 unit absolute decrease in RBC transfusions in the 8 weeks before the unblinding (scheduled to occur after 12 weeks of treatment) compared with RBC transfusions in the 8 weeks before the date the informed consent form was signed.
Time Frame
Up to Week 13
Secondary Outcome Measure Information:
Title
Change From Baseline in the Mean Hemoglobin Concentrations at Week 13
Time Frame
Baseline and Week 13
Title
Percentage of Participants Achieving Hemoglobin Improvement (≥1.5 g/dL Increase From Baseline) Unrelated to Red Blood Cell (RBC) Transfusion at Week 13
Time Frame
Week 13
Title
Percentage of Participants Who Did Not Require a Red Blood Cell (RBC) Transfusions to Treat Anemia of Myelodysplastic Syndrome (MDS) in the 8 Weeks of Treatment Before Unblinding at Week 13
Time Frame
8 weeks
Title
Mean Changes From Baseline in Percentages of Bone Marrow Blast Cells at Week 13
Time Frame
Baseline and Week 13
Title
Median Number of Red Blood Cell (RBC) Transfusions to Treat Anemia of Myelodysplastic Syndrome (MDS) During the 8 Weeks of Treatment Before Unblinding at Week 13
Time Frame
8 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Confirmed diagnosis of myelodysplastic syndrome (MDS), according to World Heath Organization or the French-American-British Cooperative Group pathologic classification, with an International Prognostic Scoring System score 0, 0.5, or 1.0, indicating Low- or INT-1-risk disease.
Documented RBC transfusion of at least 2 units of RBC for the treatment of the anemia of MDS in the 8 weeks preceding the start of the Screening Period.
Adequate iron stores, demonstrated by either the presence of stainable iron in the bone marrow or a serum ferritin of > 100 ng/mL.
Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 2.
Symptomatic anemia (defined by a score > 0 on the Non-Chemotherapy Anemia Symptom Scale [NCA-SS]).
Exclusion Criteria:
Had treatment with drugs or other agents targeting IL-6 or its receptor within 4 weeks of randomization.
Any condition that, in the opinion of the investigator, would make participation not in the best interest (eg, compromise the well-being) of the patient or that could prevent, limit, or confound the protocol-specified assessments.
Patients with Chronic Myelomonocytic Leukemia (CMML).
Causes other than MDS contributing to anemia, such as Vitamin B12 or folate deficiency, bleeding, hemolysis, hemoglobinopathy, or chronic renal failure.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Janssen Research & Development, LLC Clinical Trial
Organizational Affiliation
Janssen Research & Development, LLC
Official's Role
Study Director
Facility Information:
City
Tampa
State/Province
Florida
Country
United States
City
Boston
State/Province
Massachusetts
Country
United States
City
New York
State/Province
New York
Country
United States
City
Winston-Salem
State/Province
North Carolina
Country
United States
City
Houston
State/Province
Texas
Country
United States
City
Box Hill
Country
Australia
City
Camperdown
Country
Australia
City
St Leonards
Country
Australia
City
Antwerpen
Country
Belgium
City
Brugge
Country
Belgium
City
Gent
Country
Belgium
City
Yvoir
Country
Belgium
City
Den Haag
Country
Netherlands
City
Dordrecht
Country
Netherlands
City
Krasnodar
Country
Russian Federation
City
Moscow N/A
Country
Russian Federation
City
Nizhny Novgorod
Country
Russian Federation
City
Barcelona
Country
Spain
City
Madrid
Country
Spain
City
Oviedo (Asturias)
Country
Spain
City
Salamanca
Country
Spain
City
Valencia
Country
Spain
City
Stockholm
Country
Sweden
12. IPD Sharing Statement
Learn more about this trial
A Study Comparing Siltuximab Plus Best Supportive Care to Placebo Plus Best Supportive Care in Anemic Patients With International Prognostic Scoring System Low- or Intermediate-1-Risk Myelodysplastic Syndrome
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