Study Summary
This trial is designed to see if adding ralinepag to standard PAH care or PAH-specific background therapy can improve outcomes for people with WHO Group 1 PAH.
Eligible Conditions
- Pulmonary Arterial Hypertension (PAH)
- Severe Pulmonary Arterial Hypertension
- Pulmonary Arterial Hypertension
- Systemic Sclerosis-Associated Pulmonary Arterial Hypertension
- Pulmonary Hypertension
- Familial Primary Pulmonary Hypertension
- Vascular Disease
- Pulmonary Arterial Hypertension Exacerbation
- Connective Tissue Disease
- Lung Disease
- Pulmonary Arterial Hypertension of Congenital Heart Disease
- Connective Tissue Disease-Associated Pulmonary Arterial Hypertension
- Pulmonary Arterial Hypertension Gene Expression
- Cardiovascular Disease
- High Blood Pressure
- Respiratory Diseases
Treatment Effectiveness
Phase-Based Effectiveness
Phase 3
Study Objectives
1 Primary · 11 Secondary · Reporting Duration: The study duration was event-based. This parameter was assessed from randomization until the conclusion of the study, up to 3 years
Baseline to Week 28
Change from Baseline in 6-minute walk distance (6MWD)
Change from Baseline in N-terminal pro-brain natriuretic peptide (NT-proBNP)
Change from Baseline in WHO/New York Heart Association (NYHA) Functional Class
Change from Baseline in health-related quality of life as measured by patient-reported outcomes.
Change from Baseline in heart rate recovery (HRR) following completion of the 6MWT
Clinical improvement as defined by the absence of clinical worsening and fulfillment of at least 2 of the 3 of the following: increase in 6MWD ≥10% or ≥30 m, improvement to or maintenance of WHO FC I or II, and decrease in NT-proBNP by at least 30%.
Registry to Evaluate Early and Long-term PAH Disease Management (REVEAL) risk score
Safety and tolerability of ralinepag in subjects with PAH
Shift and proportion of subjects who attain all 3 of the following: NT-proBNP level <300 pg/mL, 6MWD >440 meters, and WHO/NYHA Functional Class I or II
The study duration was event-based. This parameter was assessed from randomization until the conclusion of the study (when the target number of adjudicated events was achieved, as defined in the study protocol).
Time to all-cause mortality
Time to first all-cause nonelective hospitalization
Year 3
Time from randomization to the first adjudicated protocol-defined clinical worsening event
Trial Safety
Phase-Based Safety
This is further along than 85% of similar trials
Side Effects for
Oral Ralinepag
64%Headache
38%Diarrhoea
33%Pain in jaw
31%Nausea
27%Myalgia
27%Flushing
18%Anaemia
18%Dizziness
16%Arthralgia
16%Pain in extremity
16%Fatigue
13%Right ventricular failure
13%N-terminal prohormone brain natriuretic peptide increased
13%Hypotension
11%Vomiting
11%Upper respiratory tract infection
11%Iron deficiency
11%Palpitations
11%Muscle spasms
9%Cardiac failure
9%Oedema peripheral
9%Pneumonia
9%Lower respiratory tract infection
9%Urinary tract infection
9%Dyspnoea exertional
9%Dyspnoea
9%Pruritus
7%Non-cardiac chest pain
7%Bronchitis
7%Syncope
7%Anxiety
7%Pulmonary arterial hypertension
7%Hyperkalaemia
7%Abdominal pain
7%Influenza
7%Back pain
7%Hypokalaemia
7%Presyncope
7%Respiratory tract infection
4%Cardiac arrest
4%Haematemesis
2%Pleural effusion
2%Pulmonary infarction
2%Acute kidney injury
2%Arrhythmia supraventricular
2%Asthenia
2%Atrial fibrillation
2%Brain abscess
2%Cardiopulmonary failure
2%Chest pain
2%Drug withdrawal syndrome
2%Epilepsy
2%Foot fracture
2%Haemoptysis
2%Head injury
2%Myositis
2%Oesophageal varices haemorrhage
2%Varices oesophageal
2%Atrial flutter
2%Deep vein thrombosis
2%Hyponatraemia
2%Benign breast neoplasm
2%COVID-19 pneumonia
2%Gastroenteritis viral
2%Multiple organ dysfunction syndrome
2%Abdominal distension
2%COVID-19
2%Breast cancer in situ
2%Acute respiratory failure
2%Cardiac failure congestive
2%Clostridium difficile infection
2%Device related sepsis
2%Pneumonia aspiration
Awards & Highlights
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Trial Design
2 Treatment Groups
Ralinepag
1 of 2
Placebo
1 of 2
Experimental Treatment
Non-Treatment Group
1000 Total Participants · 2 Treatment Groups
Primary Treatment: Ralinepag · Has Placebo Group · Phase 3
Ralinepag
Drug
Experimental Group · 1 Intervention: Ralinepag · Intervention Types: DrugPlacebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: DrugTreatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ralinepag
Not yet FDA approved
Trial Logistics
Trial Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: the study duration was event-based. this parameter was assessed from randomization until the conclusion of the study, up to 3 years
Who is running the clinical trial?
United TherapeuticsLead Sponsor
106 Previous Clinical Trials
11,264 Total Patients Enrolled
60 Trials studying Pulmonary Arterial Hypertension
7,259 Patients Enrolled for Pulmonary Arterial Hypertension
Eligibility Criteria
Age 18 - 75 · All Participants · 1 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:You are able to walk at least 150 meters (approximately 500 feet) in six minutes or less.
Who else is applying?
What state do they live in?
| Texas | 57.1% |
| Florida | 42.9% |
How old are they?
| 18 - 65 | 100.0% |
What site did they apply to?
| University of Miami | 50.0% |
| Memorial Hermann Hospital | 50.0% |
What portion of applicants met pre-screening criteria?
| Did not meet criteria | 100.0% |
How many prior treatments have patients received?
| 3+ | 100.0% |
Frequently Asked Questions
What are some other studies that have been done using Ralinepag?
"3 trials for Ralinepag are ongoing, with the majority being Phase 3 studies. The town of Leuven in Belgium has the most clinical studies for this treatment, but 425 locations worldwide are running trials." - Anonymous Online Contributor
Unverified Answer
Are recruitment efforts for this trial focused on individuals above the age of 20?
"This study is looking for individuals who are over the age of 18 and under the age of 75." - Anonymous Online Contributor
Unverified Answer
Has Ralinepag undergone the FDA's regulatory process?
"There is data from previous phases of clinical trials and multiple rounds of safety testing that suggest Ralinepag is a safe medication, so it received a score of 3." - Anonymous Online Contributor
Unverified Answer
Might I be a compatible candidate for this research?
"The study's aim is to treat respiratory tract diseases in patients between the ages of 18 and 75. A total of 700 individuals are needed for the trial." - Anonymous Online Contributor
Unverified Answer