A Study in Subjects With Sarcoidosis-associated Pulmonary Hypertension (SAPH) to Assess the Efficacy and Safety of Oral Selexipag

Study Purpose

Oral selexipag is commercially available in several countries for the treatment of a particular group of pulmonary hypertension (PH) called pulmonary arterial hypertension (PAH). The aim of the present study is to investigate whether selexipag could be helpful to treat patients with another form of PH called sarcoidosis-associated pulmonary hypertension (SAPH).

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 18 Years - 75 Years
Gender All
More Inclusion & Exclusion Criteria

Main

Inclusion Criteria:

  • - Confirmed diagnosis of sarcoidosis as per ATS criteria - Sarcoidosis-associated precapillary PH, confirmed by RHC (at rest) within 90 days prior to randomization.
  • - PH severity according to modified WHO FC II-IV at Screening and randomization; participants of WHO FC IV must be in a stable condition and able to perform a 6MWT.
  • - No treatment or stable treatment of PH (ie, riociguat, PDE5i, ERA) during at least 90 days prior to randomization and the RHC qualifying for enrollment, (ie, no introduction of new therapies or dose change).
  • - Stable sarcoidosis treatment regimen, ie, no new specific anti-inflammatory treatment for sarcoidosis for at least 90 days, and stable dose(s) for at least 30 days prior to randomization and the RHC qualifying for enrollment.
  • - 6MWD between 50 and 450 m both at Screening and at time of randomization.
  • - Forced vital capacity (FVC) >50% and FEV1 >30% of predicted at Screening.
  • - FEV1/FVC ≥60%, or if FEV1/FVC <60% then FEV1 must be ≥65% of predicted at Screening.
  • - Women of childbearing potential must have a negative pregnancy test at screening and randomization, must agree to undertake monthly urine pregnancy tests, and to practice an acceptable method of contraception and agree to remain on an acceptable method while receiving study intervention and until 30 days after last dose of study intervention.
  • - A woman using oral contraceptives must have been using this method for at least 1 month prior to randomization.
Main

Exclusion Criteria:

  • - PH due to left heart disease (PAWP >15 mmHg).
  • - History of left heart failure (LHF) as assessed by the investigator including cardiomyopathies, cardiac sarcoidosis, with a left ventricular ejection fraction (LVEF) <40%.
.
  • - Treatment with prostacyclin, prostacyclin analogues or IP receptor agonists (ie, selexipag) during 90 days prior to randomization and/or prior to the RHC qualifying for enrollment, except those given at vasodilator testing during RHC.
  • - SBP <90 mmHg at Screening or at randomization.
  • - Included on a lung transplant list or planned to be included until Visit 5 / Week 26.
  • - Change in dose or initiation of new diuretics and/or calcium channel blockers within 1 week prior to RHC qualifying for enrollment.
  • - Received an investigational intervention or used an invasive investigational medical device within 90 days prior to randomization.
  • - Any condition for which, in the opinion of the investigator, participation would not be in the best interests of the participant (eg, compromise well-being), or that could prevent, limit, or confound the protocol-specified assessments.
  • - Any acute or chronic impairment that may influence the ability to comply with study requirements such as to perform RHC, a reliable and reproducible 6MWT (eg, use of walking aids (cane, walker, etc.), or lung function tests.
- Any other criteria as per selexipag Summary of Product Characteristics (SmPC)

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT03942211
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2/Phase 3
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Actelion
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Ralph Preiss
Principal Investigator Affiliation Actelion
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Not yet recruiting
Countries
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Sarcoidosis-associated Pulmonary Hypertension
Additional Details

Pulmonary hypertension (PH) is a pathophysiological disorder that may involve multiple clinical conditions and can complicate several cardiovascular and respiratory diseases. Sarcoidosis is a multisystemic disorder that is characterized by non-caseating granulomas which are present in multiple tissues, particularly in the lung and lymphatic system. Severe untreated pulmonary arterial hypertension (PAH) carries a poor prognosis and is associated with higher mortality in patients with interstitial lung diseases and sarcoidosis. While there is no approved treatment for SAPH, PH-specific treatments are frequently used. Selexipag is a selective, orally available and long-acting non-prostanoid agonist of the prostacyclin receptor (prostacyclin [IP] receptor) for the treatment of patients with PAH. The rationale for this study is based on the unmet medical need for new therapeutic options for patients with SAPH and is supported by the established efficacy and safety of selexipag in the PAH indication, the shared pathomechanism between SAPH and PAH, and the available data on the efficacy and safety of PH-specific therapies in SAPH. This study consists of screening period, 2 intervention periods and safety follow-up period. The duration of individual participation will be approximately 3 years. The efficacy assessments include right heart catheterization (RHC), assessment of exercise capacity, dyspnea, pulmonary function tests, etc. Safety and tolerability will be evaluated throughout the study and includes review of concomitant medications and adverse events (AEs), clinical laboratory tests, 12-lead electrocardiogram (ECG), vital signs, physical examination, and pregnancy testing.

Contact Information

This trial has no sites locations listed at this time. If you are interested in learning more, you can contact the trial's primary contact:

For additional contact information, you can also visit the trial on clinicaltrials.gov.

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