Comparison of Therapeutic Regimens for Scleroderma Interstitial Lung Disease (The Scleroderma Lung Study II)

Summary

Scleroderma is a rare, long-term autoimmune disease in which normal tissue is replaced with dense, thick fibrous tissue. Normally, the immune system helps defend the body against disease and infection. In people with scleroderma, the immune system triggers fibroblast cells to produce too much of the protein collagen. The extra collagen becomes deposited in the skin and organs, causing hardening and thickening that is similar to the scarring process. Although scleroderma most often affects the skin, it also can affect other parts of the body, including the lungs, and in its most severe forms scleroderma can be life-threatening. Scleroderma-related interstitial lung disease is one example of a life-threatening scleroderma condition. In people with symptomatic scleroderma-related interstitial lung disease, scarring occurs in the delicate lung tissue, compromising lung function. The purpose of this study is to determine whether people with symptomatic scleroderma-related interstitial lung disease experience more respiratory benefits from treatment with a 2-year course of mycophenolate mofetil or treatment with a 1-year course of oral cyclophosphamide.

Conditions

• Scleroderma
• Interstitial Lung Disease

Interventions

DRUG

Mycophenolate mofetil

24 months of oral mycophenolate mofetil, up to a maximal dose of 1.5 grams twice daily as tolerated

DRUG

Cyclophosphamide

12 months of oral cyclophosphamide, up to a maximal dose of 2 mg/kg daily as tolerated

DRUG

Placebo

12 months of placebo will be delivered to participants in the Cyclophosphamide arm during the second year in order to maintain the blind with the Mycophenolate arm, which receives drug for the entire 2 years.

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI)

  collaborator NIH

• Hoffmann-La Roche

  collaborator INDUSTRY

• Michael Roth

  lead OTHER

Principal Investigators

• Donald P. Tashkin, MD · University of California, Los Angeles

• Robert M. Elashoff, PhD · UCLA School of Public Health

• Michael D. Roth, MD · University of California, Los Angeles

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2009-09-30

Primary Completion

2015-01-31

Completion

2015-11-30

Countries

• United States

Study Locations