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Sponsored by: |
National Heart, Lung, and Blood Institute (NHLBI) |
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Information provided by: | National Heart, Lung, and Blood Institute (NHLBI) |
ClinicalTrials.gov Identifier: | NCT00004563 |
To evaluate the efficacy and safety of cyclophosphamide versus placebo for the prevention and progression of symptomatic pulmonary disease in patients with systemic sclerosis.
Condition | Intervention | Phase |
---|---|---|
Lung Diseases Pulmonary Fibrosis Systemic Scleroderma Scleroderma, Systemic |
Drug: cyclophosphamide Drug: azathioprine |
Phase III |
Study Type: | Interventional |
Study Design: | Prevention, Randomized, Double-Blind, Placebo Control |
Study Start Date: | August 1999 |
Estimated Study Completion Date: | June 2005 |
BACKGROUND:
Systemic sclerosis is a connective tissue disease of unknown etiology characterized by microvascular injury and excessive fibrosis of the skin and viscera. In the United States, 5,000 to 10,000 new cases are diagnosed annually. Approximately 80 percent of these persons will eventually develop some degree of lung involvement, and restrictive lung disease (interstitial fibrosis) is now the leading cause of morbidity and mortality in systemic sclerosis. An inflammatory alveolitis is thought to be the precursor of interstitial pulmonary fibrosis in systemic sclerosis. An effective treatment for SSc interstitial lung disease has yet to be identified. Cyclophosphamide (CYC) is already being widely used by rheumatologists desperate to do something to halt rapidly declining lung function in SSC patients. Thus, the time is ripe to perform a placebo-controlled trial of CYC in this disease.
Pulmonary scleroderma strikes all races and is most prevalent among women during their child-bearing, child-rearing, and working years. A positive outcome from this trial, demonstrating that oral cyclophosphamide has a beneficial effect on pulmonary fibrosis, would be of great importance by offering a scientific basis for treatment. Similarly, a negative result, demonstrating no benefit from cyclophosphamide therapy, would also be important in avoiding hazardous and expensive therapy that is now being used widely.
DESIGN NARRATIVE:
Multicenter, placebo-controlled, randomized, double-blind. Subjects are recruited at 12 clinical centers and randomized to 2 mg/kg/day of cyclophosphamide or placebo. Follow-up visits for pulmonary assessments occur every three months for two years after treatment. If patients fail the cyclophosphamide treatment, they will be offered azathioprine for the remainder of the 24 month trial. The primary endpoint of the study is change in forced vital capacity at the end of 12 months of treatment. Secondary endpoints include quality of life, activity, and dyspnea indices, and carbon monoxide diffusing capacity. Recruitment ends in December, 2003.
Ages Eligible for Study: | 18 Years to 75 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
No eligibility criteria
Study ID Numbers: | 220 |
Study First Received: | February 9, 2000 |
Last Updated: | August 25, 2005 |
ClinicalTrials.gov Identifier: | NCT00004563 History of Changes |
Health Authority: | United States: Federal Government |
Antimetabolites Lung Diseases, Interstitial Immunologic Factors Skin Diseases Fibrosis Cyclophosphamide Immunosuppressive Agents Pulmonary Fibrosis Azathioprine Respiratory Tract Diseases |
Lung Diseases Connective Tissue Diseases Scleroderma, Diffuse Scleroderma Scleroderma, Systemic Antineoplastic Agents, Alkylating Antirheumatic Agents Alkylating Agents Scleroderma, Localized |
Antimetabolites Antimetabolites, Antineoplastic Molecular Mechanisms of Pharmacological Action Immunologic Factors Fibrosis Antineoplastic Agents Physiological Effects of Drugs Cyclophosphamide Pulmonary Fibrosis Azathioprine Pathologic Processes Respiratory Tract Diseases Therapeutic Uses |
Connective Tissue Diseases Scleroderma, Localized Alkylating Agents Lung Diseases, Interstitial Skin Diseases Immunosuppressive Agents Pharmacologic Actions Lung Diseases Myeloablative Agonists Scleroderma, Diffuse Scleroderma, Systemic Antineoplastic Agents, Alkylating Antirheumatic Agents |