A Double Blind, Randomized, Placebo-Controlled Trial Evaluating Efficacy and Safety of Oral Nintedanib Treatment for at Least 52 Weeks in Patients with “Systemic Sclerosis associated Interstitial Lung Disease” (SSc-ILD)

October 06, 2016

Institutional Review Board, Hospital for Special Surgery

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Principal Investigator

Robert F. Spiera, MD

Co-Investigators

Jessica K. Gordon, MD
Lindsay S. Lally, MD
Horatio Wildman, MD
Robert Kaner, MD
Annel Fernandez
Emily Bakaj
Alexandra Morquette

Summary

In this study, patients diagnosed with Systemic Sclerosis (SSc) associated Interstitial Lung Disease based upon classification according to the most recent ACR/EULAR criteria and HRCT (within previous 12 months) will be treated in a randomized, double-blinded, 1:1 fashion with Nintedanib 150 mg BID or with placebo. The purpose of this study is to investigate how well the treatment with the study drug nintedanib can demonstrate a slowing of the rate of lung scarring, how well the study treatment has an effect on the skin thickening, and evaluate how patients feel about their condition while on placebo or nintedanib. The duration of study treatment is 52 weeks with the option of continuation of blinded treatment to a maximum of 100 weeks. The primary endpoint of the study, measured at week 52 is the rate of decline in FVC in mL over 52 weeks. After the screening visit, the study requires 9 to 12 follow up visits. Visits 2 to 5 are 2 weeks apart, weeks 6 to 9 are 6 weeks apart, and visits 9 to 12 are 16 weeks apart.  An estimated 520 subjects are expected to participate worldwide. It is expected that 3 patients will be enrolled at this site.

Inclusion/Exclusion Criteria

Inclusion

• Aged ≥ 18 yrs
• Patient fulfills 2013 ACR/EULAR criteria for SSc.
• SSc disease onset (first non-Raynaud Symptom) must be within 5 years of Visit 1
• SSc related Interstitial Lung Disease pattern confirmed by HRCT performed within 12 months of Visit 1.
  • Extent of disease in lung: ≥10% on HRCT (accessed by central review)
• FVC≥40% of predicted normal at Visit 2
• DLCO: 30% to 89% of predicted at Visit 2

Exclusion

• Previous treatment with nintedanib or pirfenidone
• Treatment with
  • Prednisone > 10 mg/day within 2 weeks prior to Vis. 2
  • Azathiprine, hydroxzychloroquine, colchicine, D-penicillamine, sulfasalazine within 8 weeks of Vis. 2
  • Cyclophosphamide, rituximab, tocilizumab, abatacept, leflunomide, tacrolimus, newer anti-arthritic treatments like tofacitinib/ciclosproine within 6mo of Vis. 2
• Unstable background therapy with either mycophenolate mofetil/sodium or methotrexate. Patients have to be either
  • Not on immunosuppressive therapy, or
  • On stable therapy with mycophenolate mofetil/sodium or methotrexate for 6 mo prior to Vis. 2 and should stay on this background therapy for 6 mo after randomization
• AST, ALT > 1.5 x ULN

Bilirubin > 1.5 x ULN   Creatine clearance <30 mL/min

Contact Information

Annel Fernandez
Research Coordinator
212.774.2123
Fernandeza@hss.edu