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

NCT01513317 | Terminated Phase 2 Results DMC

• 3 other identifiers: CR100752CNTO328MDS20012011-000261-12
• Study Type: interventional
• Target: 76
• Locations: 7 countries
• Sites: 23

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).

Conditions

Myelodysplastic Syndrome

Keywords

Myelodysplastic Syndrome; MDS; Blood and lymphatic diseases; Siltuximab; Anemic

Outcome Measures

Primary Outcomes (1)

• 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.

  Up to Week 13

Secondary Outcomes (5)

• Change From Baseline in the Mean Hemoglobin Concentrations at Week 13

  Baseline and 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

  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

  8 weeks

• Mean Changes From Baseline in Percentages of Bone Marrow Blast Cells at Week 13

  Baseline and 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

  8 weeks

Study Arms (2)

Siltuximab

EXPERIMENTAL

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

Drug: Siltuximab; Drug: Best supportive care (BSC)

Placebo

EXPERIMENTAL

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

Drug: Placebo; Drug: Best supportive care (BSC)

Interventions

Siltuximab DRUG

15 mg/kg administered as a 1-hour intravenous infusion every 4 weeks

Siltuximab

Placebo DRUG

Administered as a 1-hour intravenous infusion every 4 weeks

Placebo

Best supportive care (BSC) DRUG

Best supportive care according to local standards and guidelines

Placebo; Siltuximab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 \[NCA-SS\]).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (23)

Unknown Facility

Tampa, Florida, United States

Location

Unknown Facility

Boston, Massachusetts, United States

Location

Unknown Facility

New York, New York, United States

Location

Unknown Facility

Winston-Salem, North Carolina, United States

Location

Unknown Facility

Houston, Texas, United States

Location

Unknown Facility

Box Hill, Australia

Location

Unknown Facility

Camperdown, Australia

Location

Unknown Facility

St Leonards, Australia

Location

Unknown Facility

Antwerp, Belgium

Location

Unknown Facility

Bruges, Belgium

Location

Unknown Facility

Ghent, Belgium

Location

Unknown Facility

Yvoir, Belgium

Location

Unknown Facility

Dordrecht, Netherlands

Location

Unknown Facility

The Hague, Netherlands

Location

Unknown Facility

Krasnodar, Russia

Location

Unknown Facility

Moscow, Russia

Location

Unknown Facility

Nizhny Novgorod, Russia

Location

Unknown Facility

Barcelona, Spain

Location

Unknown Facility

Madrid, Spain

Location

Unknown Facility

Oviedo (Asturias), Spain

Location

Unknown Facility

Salamanca, Spain

Location

Unknown Facility

Valencia, Spain

Location

Unknown Facility

Stockholm, Sweden

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes; Lymphatic Diseases

Interventions

siltuximab

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: DIRECTOR CLINICAL RESEARCH
• Organization: Janssen Research & Development

ClinicalTrialDisclosure@its.jnj.com

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 21, 2011
• First Posted: January 20, 2012
• Study Start: November 1, 2011
• Primary Completion: September 1, 2012
• Study Completion: September 1, 2012
• Last Updated: September 29, 2014
• Results First Posted: September 29, 2014

Record last verified: 2014-09

Locations

NL (2); RU (3); SE (1); BE (4); AU (3); US (5); ES (5)