Completed

Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis: A Phase 2a, Single-centered, Randomized, Placebo-controlled, Double-blind, Proof-of-concept Pilot Study.

What is being tested

Belimumab

+ Mycophenolate Mofetil

DrugOther

Who is being recruted

Connective Tissue Diseases+2

+ Skin Diseases

+ Skin and Connective Tissue Diseases

Over 18 Years

+24 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Placebo-ControlledPhase 2

Interventional

Study Start: August 2012

See protocol details

Summary

Principal SponsorHospital for Special Surgery, New York

Last updated: May 24, 2022

Sourced from a government-validated database.Claim as a partner

Study start date: August 1, 2012

Actual date on which the first participant was enrolled.

The specific objectives of this study are to: Determine whether belimumab used in combination with MMF is safe and tolerable in the treatment of patients with early diffuse cutaneous systemic sclerosis (Disease duration < 3 years). Determine whether belimumab used in combination with MMF is more effective in the treatment of diffuse cutaneous systemic sclerosis than MMF alone, as measured by change in modified Rodnan Skin Score (mRSS), forced vital capacity (FVC), hemoglobin corrected diffusion capacity (DLCO), Medsger Severity Scale (MSS), and by other physician and patient derived outcome measures. Determine the biological activity of Belimumab/MMF as assessed by effect on histology of skin, change in B-Cell profiles, effect on BLyS levels, and effect on serological and cutaneous biomarkers of disease activity.

NCT01670565

Principal SponsorHospital for Special Surgery, New York

Last updated: May 24, 2022

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

20 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Over 18 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Connective Tissue DiseasesSkin DiseasesSkin and Connective Tissue DiseasesScleroderma, SystemicScleroderma, Diffuse

Criteria

3 inclusion criteria required to participate

Age greater than or equal to eighteen years

Clinical diagnosis of diffuse systemic sclerosis by ACR criteria, with a stable modified Rodnan skin score in the one month preceding introduction of belimumab therapy

Disease duration of less than or equal to 3 years as defined by the date of onset of the first non-Raynaud's symptom

21 exclusion criteria prevent from participating

A positive pregnancy test at entry into this study

Any other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that may affect the interpretation of the results or render the patient at high risk from treatment complications

Breastfeeding. Breastfeeding is contraindicated with the use of MMF

Concurrent serious medical condition which in the opinion of the investigator makes the patient inappropriate for this study such as uncontrollable CHF, arrhythmia, severe pulmonary or systemic hypertension, severe GI involvement, hepatic impairment, serum creatinine of greater than 2.0, active infection, severe diabetes, unstable atherosclerotic cardiovascular disease, malignancy, HIV, or severe peripheral vascular disease

Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

2 intervention groups are designated in this study

50% chance of being blinded to the placebo group

Treatment Groups

Group I

Experimental

All patients who enroll in this trial will FIRST receive mycophenolate mofetil (MMF, Cellcept), which is a drug commonly given to patients with scleroderma in clinical practice. This drug will be given at no cost to the patient. After the patient has been titrated to 2 grams of MMF per day, the patient will receive EITHER a 10 mg/kg belimumab (Benlysta) intravenous infusion OR a placebo (saline) infusion. This medication and infusion will of course be covered by the study.

Group II

Placebo

In order to observe the difference between belimumab/MMF compared to MMF alone, half of the patients will receive a normal saline infusion that appears identical to the belimumab infusion.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

Hospital for Special Surgery

New York, United StatesOpen Hospital for Special Surgery in Google Maps

CompletedOne Study Center