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Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis

NCT01670565 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2022-05-24

Study results available
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Summary

This is a 48 week, phase IIa, single center, randomized, double-blind, placebo-controlled, proof-of-concept pilot study. All participants will first be treated with mycophenolate mofetil (MMF, Cellcept) and titrated up to a dose of 2 grams/day. Following this period, half will be given either a belimumab (Benlysta®) or placebo intravenous infusion to treat early diffuse cutaneous systemic sclerosis. Belimumab/MMF is expected to improve disease activity measured by an improvement in skin thickening and stability of pulmonary function test measurements when compared to patients treated with placebo/MMF.

Conditions

Interventions

DRUG

Belimumab

Belimumab (Benlysta®) decreases B-Cell survival and has been FDA approved for the treatment of systemic lupus erythematosus, another rheumatic autoimmune disease. Belimumab is a recombinant, fully human monoclonal antibody; it binds to the soluble human B lymphocyte stimulator (BLyS) with high affinity and inhibits its biologic activity. Prior research provides a robust rationale for the investigation of belimumab in combination with MMF (Cellcept ®) for the treatment of early diffuse cutaneous systemic sclerosis.

DRUG

Mycophenolate Mofetil

Patients received background MMF therapy, some who were naive to MMF were titrated up to 1,000 mg twice daily and others had been receiving MMF at \<2,000 mg/day for \<3 months. MMF was chosen so that background therapy would be uniform and not a further source of variability in the small study.

OTHER

Placebo Infusion

Infusion of normal saline

Sponsors & Collaborators

  • Human Genome Sciences Inc.

    collaborator INDUSTRY

  • Hospital for Special Surgery, New York

    lead OTHER

Principal Investigators

  • Robert Spiera, MD · Hospital for Special Surgery, New York

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-08-31
Primary Completion
2015-11-30
Completion
2016-02-29

Countries

  • United States

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01670565 on ClinicalTrials.gov