Pulmonary Arterial HypertensionPhase 1NCT05427162
A Study of Single and Multiple Doses of Different Formulations of a Prostacyclin Receptor Agonist
A Phase 1 study testing Prostacyclin Receptor Agonist in people with Pulmonary Arterial Hypertension, run by Actelion at 1 site in United States.
Trial summary
The purpose of the study is to assess safety and tolerability of prostacyclin receptor agonist formulation in treatment period 1 and with different formulation of prostacyclin receptor agonist in treatment period 2. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- Prostacyclin Receptor Agonist
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 88 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Jun 2022to 5 Dec 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Otherwise, healthy as deemed by the investigator on the basis of physical examination, medical history, vital signs, and 12-lead electrocardiograms (ECG) performed at Screening and Day -1 of oral treatment period
- Otherwise, healthy medically stable on the basis of clinical laboratory tests performed at screening. If the results of the serum chemistry panel, blood coagulation, hematology, or urinalysis are outside the normal reference ranges, the participant may be included only if the investigator judges the abnormalities or deviations from normal to be not clinically significant or to be appropriate and reasonable for the population under study. This determination must be recorded in the participant's source documents and initialed by the investigator
- Body mass index (BMI) within the range 18.0 to 32.0 kilograms per meter square (kg/m^2) (inclusive) and body weight not less than 50 kilograms (kg) at screening
- All female participants must have a negative highly sensitive serum (beta-human chorionic gonadotropin [beta-hCG]) at screening
- A male participant must agree not to donate sperm for the purpose of reproduction during the study and for a minimum of 90 days after receiving the last dose of study intervention
Who cannot join
- Known allergies, hypersensitivity, anaphylaxis, or intolerance to prostacyclin receptor agonist or drugs of the same class, or any excipient (including poloxamer and polysorbate) of the drug formulation(s)
- Clinically significant abnormal physical examination, vital signs, or 12-lead ECG (QTc greater than or equal to [>=]450 milliseconds [msec] for men and >=460 msec for women. QT corrected according to Bazett's formula [QTcB]) as assessed by the Investigator at Screening or Day -1 of oral treatment period
- History of malignancy within 3 years before screening (exceptions are squamous and basal cell carcinomas of the skin
- Positive serologic testing for human immunodeficiency virus (HIV), hepatitis B surface antigen (hBsAg), hepatitis C virus (HCV), active coronavirus disease 2019 (COVID-19) infection
- A history of repeated (more than once over the last 30 days) fainting due to cardiac cause, collapse, syncope, orthostatic hypotension, or vasovagal reactions
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Open-Label, Randomized Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of Different Formulations of an IP Receptor Agonist
The purpose of the study is to assess safety and tolerability of prostacyclin receptor agonist formulation in treatment period 1 and with different formulation of prostacyclin receptor agonist in treatment period 2.
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- Prostacyclin Receptor Agonist
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Prostacyclin Receptor AgonistDrug
Prostacyclin receptor agonist will be administered.
| Study group | Role | What they receive |
|---|---|---|
| Prostacyclin Receptor Agonist | Experimental | Participants in Cohort 1-7 will receive multiple doses of prostacyclin receptor agonist of formulation 1 in treatment period 1, followed by a single dose of various other formulations (formulation 2, 3, and 4) in treatment period 2. Cohort 7 will be optional. Doses in Cohorts 4, 5, 6 and cohort 7 will be based on PK, safety and tolerability data of previous 3 cohorts (preceding cohorts). Receives: Drug: Prostacyclin Receptor Agonist |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
An adverse event is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAEs are defined as AEs with onset or worsening on or after date of first dose of study treatment.
Measured: Up to 114 days
Number of Participants With Serious TEAEs
A SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Serious TEAEs are defined as serious events between administration of study drug and after the last dose that were absent before treatment or that worsen relative to pretreatment state.
Measured: Up to 114 days
Number of Participants with TEAEs by Severity
Number of participants with TEAEs by severity will be evaluated. An assessment of severity grade will be made using the following general categorical descriptors, such as Mild (Awareness of symptoms that are easily tolerated, causing minimal discomfort, and not interfering with everyday activities), Moderate (Sufficient discomfort is present to cause interference with normal activity), and Severe (Extreme distress, causing significant impairment of functioning or incapacitation, and prevents normal everyday activities).
Measured: Up to 114 days
Number of Participants with TEAEs Leading to Discontinuation
Number of participants with TEAEs leading to discontinuation will be reported.
Measured: Up to 114 days
Number of Participants With Change from Baseline in Clinical Laboratory Values
Number of participants with change from baseline in clinical laboratory values (chemistry, hematology, and urinalysis) will be evaluated.
Measured: Up to 114 days
Number of Participants With Injection Site Reactions
Number of participants with injection site reactions will be evaluated.
Measured: Up to 114 days
Secondary outcomes (13)
Treatment Period 1: Maximum Observed Plasma Concentration of Prostacyclin Receptor Agonist at Steady State (Cmax[ss])
Cmax(ss) is the maximum observed plasma concentration of prostacyclin receptor agonist at steady state, during treatment period 1.
Measured: Up to 114 days
Treatment Period 1: Time to Reach Maximum Observed Plasma Concentration of Prostacyclin Receptor Agonist at Steady State (Tmax[ss])
Tmax(ss) is the actual sampling time to reach maximum observed plasma concentration of prostacyclin receptor agonist at steady state, during treatment period 1.
Measured: Up to 114 days
Treatment Period 1: Area Under the Curve From Time Zero to tau of Prostacyclin Receptor Agonist at Steady State (AUC[0-tau{ss}])
AUC(0-tau[ss]) is defined as area under the curve from time 0 to tau hours post dose of prostacyclin receptor agonist at steady state, during treatment period 1.
Measured: Up to 114 days
Treatment Period 1: Plasma Concentration at the End of One Dose Interval of Prostacyclin Receptor Agonist at Steady State (Ctrough[ss])
Ctrough(ss) is the plasma concentration at the end of one dose interval of prostacyclin receptor agonist at steady state, during treatment period 1.
Measured: Up to 114 days
Treatment Period 2: Maximum Observed Plasma Concentration (Cmax) of Prostacyclin Receptor Agonist
Cmax is the maximum observed plasma concentration of prostacyclin receptor agonist, during treatment period 2.
Measured: Up to 114 days
Treatment Period 2: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Prostacyclin Receptor Agonist
Tmax is the actual sampling time to reach maximum observed plasma concentration of prostacyclin receptor agonist, during treatment period 2.
Measured: Up to 114 days
Treatment Period 2: Area Under the Plasma Concentration-time Curve from time Zero to time t (AUC[0-t]) of Prostacyclin Receptor Agonist
Area under the plasma concentration-time curve from time zero to time t of the last measured concentration above the limit of concentration of prostacyclin receptor agonist, during treatment period 2.
Measured: Up to 114 days
Treatment Period 2: Area Under the Plasma Concentration Time Curve From Time Zero to Infinite Time (AUC [0-infinity]) of Prostacyclin Receptor Agonist
AUC (0-infinity) is the area under the plasma concentration-time curve from time zero to infinite time calculated as the sum of AUC (0-last) and C (0-last)/ lambda(z); wherein AUC (0-last) is area under the plasma concentration time curve from time zero to last quantifiable time, C(0-last) is the last observed quantifiable concentration of prostacyclin receptor agonist, and lambda (z) is elimination rate constant, during treatment period 2.
Measured: Up to 114 days
Treatment Period 2: Area Under the Plasma Concentration-time Curve From Zero to Time 24 Hours (AUC [0-24h]) of Prostacyclin Receptor Agonist
AUC (0-24h) is the area under the plasma concentration-time curve from zero to time 24 hours, during treatment period 2.
Measured: Predose up to 24 hours post dose
Treatment Period 2: Area Under the Plasma Concentration-time Curve From Zero to Day 14 (AUC [0-Day 14]) of Prostacyclin Receptor Agonist
AUC (0-Day 14) is the area under the plasma concentration-time curve from zero to Day 14, during treatment period 2.
Measured: Up to Day 14
Treatment Period 2: Area Under the Plasma Concentration-time Curve From Zero to Day 28 (AUC [0-Day 28]) of Prostacyclin Receptor Agonist
AUC (0-Day 28) is the area under the plasma concentration-time curve from zero to Day 28, during treatment period 2.
Measured: Up to Day 28
Treatment Period 2: Plasma Concentration of Prostacyclin Receptor Agonist at Day 14 (C[Day 14])
C(Day 14) is the plasma concentration of prostacyclin receptor agonist at Day 14, during treatment period 2
Measured: Up to Day 14
Treatment Period 2: Plasma Concentration of Prostacyclin Receptor Agonist at Day 28 (C[Day 28])
C(Day 28) is the plasma concentration of prostacyclin receptor agonist at Day 28, during treatment period 2.
Measured: Up to Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2022
- Study start21 Jun 2022
- Primary completion5 Dec 2023
- Study completion5 Dec 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 88 participants (actual)
Sponsor and investigators
- Lead sponsor
- Actelion
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Actelion Pharmaceuticals Ltd. Clinical Trials, Study director, Actelion
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05427162
- Sponsor's protocol ID
- CR109201
- Status last verified
- Mar 2025
- First submitted
- 16 Jun 2022
- Last updated
- 30 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson \& Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-Label, Randomized Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of Different Formulations of an IP Receptor Agonist
- ClinicalTrials.gov ID
- NCT05427162
- Acronym / sponsor ID
- CR109201
- Conditions
- Pulmonary Arterial Hypertension
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 88 participants (actual)
- Lead sponsor
- ActelionIndustry (pharma, biotech, devices)
- Study start
- 21 Jun 2022 (actual)
- Primary completion
- 5 Dec 2023 (actual)
- Study completion
- 5 Dec 2023 (actual)
- First posted
- 22 Jun 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to assess safety and tolerability of prostacyclin receptor agonist formulation in treatment period 1 and with different formulation of prostacyclin receptor agonist in treatment period 2.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Prostacyclin Receptor Agonist | Experimental | Drug: Prostacyclin Receptor AgonistParticipants in Cohort 1-7 will receive multiple doses of prostacyclin receptor agonist of formulation 1 in treatment period 1, followed by a single dose of various other formulations (formulation 2, 3, and 4) in treatment period 2. Cohort 7 will be optional. Doses in Cohorts 4, 5, 6 and cohort 7 will be based on PK, safety and tolerability data of previous 3 cohorts (preceding cohorts). |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Otherwise, healthy as deemed by the investigator on the basis of physical examination, medical history, vital signs, and 12-lead electrocardiograms (ECG) performed at Screening and Day -1 of oral treatment period
- Otherwise, healthy medically stable on the basis of clinical laboratory tests performed at screening. If the results of the serum chemistry panel, blood coagulation, hematology, or urinalysis are outside the normal reference ranges, the participant may be included only if the investigator judges the abnormalities or deviations from normal to be not clinically significant or to be appropriate and reasonable for the population under study. This determination must be recorded in the participant's source documents and initialed by the investigator
- Body mass index (BMI) within the range 18.0 to 32.0 kilograms per meter square (kg/m^2) (inclusive) and body weight not less than 50 kilograms (kg) at screening
- All female participants must have a negative highly sensitive serum (beta-human chorionic gonadotropin [beta-hCG]) at screening
- A male participant must agree not to donate sperm for the purpose of reproduction during the study and for a minimum of 90 days after receiving the last dose of study intervention
Key exclusion criteria (5 listed)
- Known allergies, hypersensitivity, anaphylaxis, or intolerance to prostacyclin receptor agonist or drugs of the same class, or any excipient (including poloxamer and polysorbate) of the drug formulation(s)
- Clinically significant abnormal physical examination, vital signs, or 12-lead ECG (QTc greater than or equal to [>=]450 milliseconds [msec] for men and >=460 msec for women. QT corrected according to Bazett's formula [QTcB]) as assessed by the Investigator at Screening or Day -1 of oral treatment period
- History of malignancy within 3 years before screening (exceptions are squamous and basal cell carcinomas of the skin
- Positive serologic testing for human immunodeficiency virus (HIV), hepatitis B surface antigen (hBsAg), hepatitis C virus (HCV), active coronavirus disease 2019 (COVID-19) infection
- A history of repeated (more than once over the last 30 days) fainting due to cardiac cause, collapse, syncope, orthostatic hypotension, or vasovagal reactions
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Prostacyclin Receptor AgonistProstacyclin receptor agonist will be administered. | Drug | Prostacyclin Receptor Agonist |
Primary outcome
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Time frame: Up to 114 days
An adverse event is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAEs are defined as AEs with onset or worsening on or after date of first dose of study treatment.
Number of Participants With Serious TEAEs
Time frame: Up to 114 days
A SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Serious TEAEs are defined as serious events between administration of study drug and after the last dose that were absent before treatment or that worsen relative to pretreatment state.
Number of Participants with TEAEs by Severity
Time frame: Up to 114 days
Number of participants with TEAEs by severity will be evaluated. An assessment of severity grade will be made using the following general categorical descriptors, such as Mild (Awareness of symptoms that are easily tolerated, causing minimal discomfort, and not interfering with everyday activities), Moderate (Sufficient discomfort is present to cause interference with normal activity), and Severe (Extreme distress, causing significant impairment of functioning or incapacitation, and prevents normal everyday activities).
Number of Participants with TEAEs Leading to Discontinuation
Time frame: Up to 114 days
Number of participants with TEAEs leading to discontinuation will be reported.
Number of Participants With Change from Baseline in Clinical Laboratory Values
Time frame: Up to 114 days
Number of participants with change from baseline in clinical laboratory values (chemistry, hematology, and urinalysis) will be evaluated.
Number of Participants With Injection Site Reactions
Time frame: Up to 114 days
Number of participants with injection site reactions will be evaluated.
13 secondary outcomes
Treatment Period 1: Maximum Observed Plasma Concentration of Prostacyclin Receptor Agonist at Steady State (Cmax[ss])
Time frame: Up to 114 days
Treatment Period 1: Time to Reach Maximum Observed Plasma Concentration of Prostacyclin Receptor Agonist at Steady State (Tmax[ss])
Time frame: Up to 114 days
Treatment Period 1: Area Under the Curve From Time Zero to tau of Prostacyclin Receptor Agonist at Steady State (AUC[0-tau{ss}])
Time frame: Up to 114 days
Treatment Period 1: Plasma Concentration at the End of One Dose Interval of Prostacyclin Receptor Agonist at Steady State (Ctrough[ss])
Time frame: Up to 114 days
Treatment Period 2: Maximum Observed Plasma Concentration (Cmax) of Prostacyclin Receptor Agonist
Time frame: Up to 114 days
Treatment Period 2: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Prostacyclin Receptor Agonist
Time frame: Up to 114 days
Treatment Period 2: Area Under the Plasma Concentration-time Curve from time Zero to time t (AUC[0-t]) of Prostacyclin Receptor Agonist
Time frame: Up to 114 days
Treatment Period 2: Area Under the Plasma Concentration Time Curve From Time Zero to Infinite Time (AUC [0-infinity]) of Prostacyclin Receptor Agonist
Time frame: Up to 114 days
Treatment Period 2: Area Under the Plasma Concentration-time Curve From Zero to Time 24 Hours (AUC [0-24h]) of Prostacyclin Receptor Agonist
Time frame: Predose up to 24 hours post dose
Treatment Period 2: Area Under the Plasma Concentration-time Curve From Zero to Day 14 (AUC [0-Day 14]) of Prostacyclin Receptor Agonist
Time frame: Up to Day 14
Treatment Period 2: Area Under the Plasma Concentration-time Curve From Zero to Day 28 (AUC [0-Day 28]) of Prostacyclin Receptor Agonist
Time frame: Up to Day 28
Treatment Period 2: Plasma Concentration of Prostacyclin Receptor Agonist at Day 14 (C[Day 14])
Time frame: Up to Day 14
Treatment Period 2: Plasma Concentration of Prostacyclin Receptor Agonist at Day 28 (C[Day 28])
Time frame: Up to Day 28
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2025.
- Individual participant dataSharing planned: Yes. The data sharing policy of the Janssen Pharmaceutical Companies of Johnson \& Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Actelion Pharmaceuticals Ltd. Clinical Trials study director
Actelion
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Prostacyclin Receptor Agonist in people with Pulmonary Arterial Hypertension, run by Actelion at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Treatment-emergent Adverse Events (TEAEs) (time frame: Up to 114 days). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2025.