Efficacy and Safety of Riociguat in Patients With Systemic Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02283762
• Date of registration: 03/11/2014
• Prospective Registration: Yes
• Primary sponsor: Bayer
• Public title: Efficacy and Safety of Riociguat in Patients With Systemic Sclerosis
• Scientific title: A Randomized, Double-Blind, Placebo-Controlled Phase II Study to Investigate the Efficacy and Safety of Riociguat in Patients With Diffuse Cutaneous Systemic Sclerosis (dcSSc)
• Date of first enrolment: January 15, 2015
• Target sample size: 121
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02283762
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2

Countries of recruitment

Australia, Belgium, Canada, Czech Republic, Czechia, France, Germany, Hungary, Italy, Japan, Netherlands, New Zealand, Spain, Switzerland, Turkey, United Kingdom, United States

Contacts

• Name: Bayer Study Director
• Affiliation: Bayer

Key inclusion & exclusion criteria

Inclusion Criteria:

- Men or women aged 18 years and older

- Systemic sclerosis, as defined by ACR/EULAR (American College of Rheumatology/European
League Against Rheumatism) 2013 criteria

- dcSSc (diffuse cutaneous systemic sclerosis) according to the LeRoy criteria, ie, skin
fibrosis proximal to the elbows and knees in addition to acral fibrosis

- Disease duration of = 18 months (defined as time from the first non-Raynaud's
phenomenon manifestation)

- = 10 and = 22 mRSS (modified Rodnan skin score) units at the screening visit

- FVC (forced vital capacity) = 45% of predicted at screening

- DLCO (diffusion capacity of the lung for carbon monoxide) = 40% of predicted
(hemoglobin-corrected) at screening

- Negative serum pregnancy test in a woman of childbearing potential at the screening
visit

- Women of childbearing potential must agree to use adequate contraception when sexually
active. "Adequate contraception" is defined as any combination of at least 2 effective
methods of birth control, of which at least 1 is a physical barrier (e.g. condom with
hormonal contraception like implants or combined oral contraceptives, condom with
intrauterine devices). This applies since signing of the informed consent form until
30 (+5) days after the last study drug administration.

Exclusion Criteria:

- Limited cutaneous SSc (systemic sclerosis) at screening

- Major surgery (including joint surgery) within 8 weeks prior to screening

- Hepatic insufficiency classified as Child-Pugh C

- Patients with isolated AST or ALT >3xULN or bilirubin >2xULN can be included in the
trial under the condition of additional monitoring during the trial

- Estimated glomerular filtration rate (eGFR) < 15 mL/min/1.73 m^2 (Modification of Diet
in Renal Disease formula) or on dialysis at the screening visit. Patients entering the
trial with eGFR 15-29 mL/min/1.73 m^2 will be undergo additional monitoring of renal
function

- Any prior history of renal crisis

- Sitting SBP (systolic blood pressure) < 95 mmHg at the screening visit

- Sitting heart rate < 50 beats per minute (BPM) at the screening visit

- Left ventricular ejection fraction < 40% prior to screening

- Any form of pulmonary hypertension as determined by right heart catheterization

- Pulmonary disease with FVC < 45% of predicted or DLCO (hemoglobin-corrected) < 40% of
predicted at screening

- Active state of hemoptysis or pulmonary hemorrhage, including those events managed by
bronchial artery embolization

- Not permitted prior and concomitant medication

- Pregnant or breast feeding women

- Women of childbearing potential not willing to use adequate contraception and not
willing to agree to 4-weekly pregnancy testing from Visit 1 (first administration of
study drug) onwards until 30 (+5) days after last study drug intake.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Scleroderma, Systemic

Intervention(s)

Drug: Riociguat (Adempas, BAY63-2521)

Drug: Placebo

Primary Outcome(s)

Change From Baseline in Modified Rodnan Skin Score (mRSS) to Week 52 [Time Frame: Baseline to week 52]

Secondary Outcome(s)

Change From Baseline in Patient's Global Assessment Score to Week 52 [Time Frame: Baseline to week 52]

Change From Baseline in Physician's Global Assessment Score to Week 52 [Time Frame: Baseline to week 52]

Change From Baseline in Forced Vital Capacity (FVC) Percent Predicted to Week 52 [Time Frame: Baseline to week 52]

Change From Baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) Score to Week 52 [Time Frame: Baseline to week 52]

CRISS (American College of Rheumatology Composite Response Index for Clinical Trials) at Week 52 Reported as Number of Participants With a CRISS Probability >=0.60 or <0.60 From Baseline to Week 52 [Time Frame: Week 52]

Secondary ID(s)

2014-001353-16

16277

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 25/01/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02283762