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Phase 3 N=158 Randomized Double-blind Prevention

Scleroderma Lung Disease

Lung Diseases · Pulmonary Fibrosis · Systemic Scleroderma · Scleroderma, Systemic

Enrolled (actual)
158
Serious AEs
6.3%
Results posted
Mar 2015
Primary outcome: Primary: Forced Vital Capacity — 66.6; 65.6 % of predicted

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Cyclophosphamide (Drug); Placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
The University of Texas Health Science Center, Houston
Primary completion
May 2013

Outcome Measures

OutcomeResultp-value
PRIMARY
Forced Vital Capacity
66.6; 65.6
SECONDARY
Total Lung Capacity
70.5; 64.7
SECONDARY
DLCO
42.8; 44.3

Summary

To evaluate the efficacy and safety of cyclophosphamide versus placebo for the prevention and progression of symptomatic pulmonary disease in patients with systemic sclerosis.

Eligibility Criteria

Inclusion Criteria

  • Patients with limited or diffuse systemic scleroderma if they had evidence of active alveolitis on examination of bronchoalveolar-lavage (BAL) fluid (defined as neutrophilia of ≥3 percent, eosinophilia of ≥2 percent, or both)on thoracic high-resolution computed tomography (CT), any ground-glass opacity,
  • Onset of the first symptom of scleroderma other than Raynaud's phenomenon within the previous seven years,
  • An FVC between 45 and 85 percent of the predicted value
  • Grade 2 exertional dyspnea according to the baseline instrument of the Mahler Dyspnea Index (as measured with the use of the magnitude-of-task component).

Exclusion Criteria

  • A single-breath carbon monoxide diffusing capacity (DlCO) that was less than 30 percent of the predicted value,
  • A history of smoking within the preceding six months, other clinically significant pulmonary abnormalities,
  • Clinically significant pulmonary hypertension requiring drug therapy.
  • Patients taking prednisone at a dose of more than 10 mg per day, those who had previously been treated for more than four weeks with oral cyclophosphamide or had received two or more intravenous doses,
  • Patients who recently received other potentially disease-modifying medications.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00004563). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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