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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01513317




Registration number
NCT01513317
Ethics application status
Date submitted
21/10/2011
Date registered
20/01/2012
Date last updated
29/09/2014

Titles & IDs
Public 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
Scientific 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
Secondary ID [1] 0 0
CNTO328MDS2001
Secondary ID [2] 0 0
CR100752
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelodysplastic Syndrome 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Siltuximab
Treatment: Drugs - Placebo
Treatment: Drugs - Best supportive care (BSC)

Experimental: Siltuximab - 15 mg/kg of siltuximab administered as a 1-hour infusion every 4 weeks + best supportive care (BSC)

Experimental: Placebo - Placebo administered as a 1-hour infusion every 4 weeks + BSC


Treatment: Drugs: Siltuximab
15 mg/kg administered as a 1-hour intravenous infusion every 4 weeks

Treatment: Drugs: Placebo
Administered as a 1-hour intravenous infusion every 4 weeks

Treatment: Drugs: Best supportive care (BSC)
Best supportive care according to local standards and guidelines

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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.
Timepoint [1] 0 0
Up to Week 13
Secondary outcome [1] 0 0
Change From Baseline in the Mean Hemoglobin Concentrations at Week 13
Timepoint [1] 0 0
Baseline and Week 13
Secondary outcome [2] 0 0
Percentage of Participants Achieving Hemoglobin Improvement (=1.5 g/dL Increase From Baseline) Unrelated to Red Blood Cell (RBC) Transfusion at Week 13
Timepoint [2] 0 0
Week 13
Secondary outcome [3] 0 0
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
Timepoint [3] 0 0
8 weeks
Secondary outcome [4] 0 0
Mean Changes From Baseline in Percentages of Bone Marrow Blast Cells at Week 13
Timepoint [4] 0 0
Baseline and Week 13
Secondary outcome [5] 0 0
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
Timepoint [5] 0 0
8 weeks

Eligibility
Key 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]).
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Box Hill
Recruitment hospital [2] 0 0
- Camperdown
Recruitment hospital [3] 0 0
- St Leonards
Recruitment postcode(s) [1] 0 0
- Box Hill
Recruitment postcode(s) [2] 0 0
- Camperdown
Recruitment postcode(s) [3] 0 0
- St Leonards
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Massachusetts
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
North Carolina
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
Belgium
State/province [6] 0 0
Antwerpen
Country [7] 0 0
Belgium
State/province [7] 0 0
Brugge
Country [8] 0 0
Belgium
State/province [8] 0 0
Gent
Country [9] 0 0
Belgium
State/province [9] 0 0
Yvoir
Country [10] 0 0
Netherlands
State/province [10] 0 0
Den Haag
Country [11] 0 0
Netherlands
State/province [11] 0 0
Dordrecht
Country [12] 0 0
Russian Federation
State/province [12] 0 0
Krasnodar
Country [13] 0 0
Russian Federation
State/province [13] 0 0
Moscow N/A
Country [14] 0 0
Russian Federation
State/province [14] 0 0
Nizhny Novgorod
Country [15] 0 0
Spain
State/province [15] 0 0
Barcelona
Country [16] 0 0
Spain
State/province [16] 0 0
Madrid
Country [17] 0 0
Spain
State/province [17] 0 0
Oviedo (Asturias)
Country [18] 0 0
Spain
State/province [18] 0 0
Salamanca
Country [19] 0 0
Spain
State/province [19] 0 0
Valencia
Country [20] 0 0
Sweden
State/province [20] 0 0
Stockholm

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Janssen Research & Development, LLC
Address
Country

Ethics approval
Ethics application status

Summary
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).
Trial website
https://clinicaltrials.gov/show/NCT01513317
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Janssen Research & Development, LLC Clinical Trial
Address 0 0
Janssen Research & Development, LLC
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT01513317