Riociguat for Sarcoidosis Associated Pulmonary Hypertension

• Register: ClinicalTrials.gov
• Last refreshed on: 14 December 2015
• Main ID: NCT02625558
• Date of registration: 26/05/2015
• Prospective Registration: No
• Primary sponsor: University of Cincinnati
• Public title: Riociguat for Sarcoidosis Associated Pulmonary Hypertension RioSAPH
• Scientific title: A Double Blind, Placebo Controlled Trial of Oral Riociguat for Sarcoidosis Associated Pulmonary Hypertension
• Date of first enrolment: April 2015
• Target sample size: 60
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT02625558
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 4

Countries of recruitment

United States

Contacts

• Name: Robert P Baughman, MD

• Name: Felicia Thompson

• Name: Robert P Baughman, MD
• Affiliation: University of Cincinnati

• Name: Steve Nathan, MD
• Affiliation: Inova Fairfax Hospital

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients with diagnosis of sarcoidosis

- Age = 18 years.

- Life expectancy of at least 2 years.

- Subjects must be able to understand and be willing to sign the written informed
consent form. A signed informed consent form must be appropriately obtained prior to
the conduct of any trial-specific procedure.

- Females of reproductive potential (FRP) must have a negative, pre-treatment pregnancy
test.

- FRP must obtain monthly pregnancy tests during treatment and one month after
treatment discontinuation. Post-menopausal women (defined as no menses for at least 1
year or post-surgical from bilateral oophorectomy) and surgically sterilized women
are not required to undergo a pregnancy test.

- Females of reproductive potential and all non-vasectomized male participants must
agree to use reliable contraception when sexually active.

- Subjects (males and females) of childbearing potential must agree to use adequate
contraception beginning at the signing of the informed consent form (ICF) until at
least 30 days after the last dose of study drug.

- Willing and able to comply with the protocol, including follow-up visits and
examinations

Exclusion Criteria:

- Patients with an FVC of less than 30% of predicted during screening visit.

- Patients with severe airway obstruction

- Patients unable to perform the 6 minute walk test

- Pregnant women (i.e. positive serum ß-human chorionic gonadotropin test or other
signs of pregnancy),

- Breast feeding women

- FRP not using reliable contraception as recommended in the Prescriber Guide for the
riociguat pregnancy monitoring program

- Subjects with a medical disorder, condition, or history of such that would impair the
subject's ability to participate or complete this study in the opinion of the
investigator

- Known significant left heart disease:

- Pulmonary venous hypertension indicated by baseline pulmonary capillary wedge
pressure > 15 mmHg

- Active state or history of hemoptysis or pulmonary hemorrhage

- Subjects requiring nitrates for any reason

- Subject using nitrates within one month of entering study

- Pulmonary veno-occlusive disease

- Subjects with underlying medical disorders with an anticipated life expectancy below
2 years (e.g. active cancer disease with localized and/or metastasized tumor).

- Subjects with hypersensitivity to the investigational drug or any of the excipients.

- Women who are pregnant or breast-feeding.

- Severe proven or suspected coronary artery disease

- Clinical relevant hepatic dysfunction indicated by: bilirubin >2 times upper limit
normal at Visit 0 and/or: alanine aminotransferase (ALT) or AST aspartate
aminotransferase (AST) >3 times upper limit normal at Visit 0 and/or: signs of severe
hepatic insufficiency (e.g. impaired albumin synthesis with an albumin <32 g/L,
hepatic encephalopathy > grade 1a) at Visit 0

West Haven Criteria of Altered Mental Status in Hepatic Encephalopathy

- Severe renal insufficiency indicated by a glomerular filtration rate <30 mL/min at
Visit 0, e.g. calculated based on the Cockcroft formula or the Modification of Diet
in Renal Disease Study Group (MDRD) formula

- Inability to comply with the protocol and/or not willing or not available for
follow-up assessments.

- Any condition which, in the investigator's opinion, makes the subject unsuitable for
trial participation.

Excluded therapies and medications, previous and concomitant

- Specific (e.g. sildenafil or tadalafil) or unspecific phosphodiesterase inhibitors
(e.g. dipyridamole, theophylline).

- NO donors (e.g. nitrates). Single applications of vasoactive drugs in connection with
diagnostic vasoreactive testing are allowed.

- Concurrent use of another investigational drug or device therapy (i.e., outside of
study treatment) during, or within 4 weeks of trial entry (signing of the informed
consent form).

- Major surgery within 30 days prior to start of study drug.



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

Health Condition(s) or Problem(s) studied

Sarcoidosis

Intervention(s)

Drug: Placebo

Drug: Riociguat

Primary Outcome(s)

Time until clinical worsening [Time Frame: 48 weeks]

Secondary Outcome(s)

Pulmonary function [Time Frame: 48 weeks]

Six minute walk [Time Frame: 48 weeks]

Safety: adverse events [Time Frame: 48 weeks]

Quality of life [Time Frame: 48 weeks]

Secondary ID(s)

2014-7130

Source(s) of Monetary Support

Please refer to primary and secondary sponsors