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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02279745




Registration number
NCT02279745
Ethics application status
Date submitted
25/10/2014
Date registered
31/10/2014
Date last updated
22/12/2021

Titles & IDs
Public title
Long-term Safety and Efficacy of Ralinepag in Pulmonary Arterial Hypertension
Scientific title
An Open-label Extension Study of Ralinepag in Patients With Pulmonary Arterial Hypertension
Secondary ID [1] 0 0
APD811-007
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pulmonary Arterial Hypertension 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cardiovascular 0 0 0 0
Hypertension

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Ralinepag

Experimental: Oral Ralinepag - Ralinepag immediate-release (IR) capsules of 10, 20, 30, 40, and 100 mcg or extended-release (XR) tablets of 50, 250, and 400 mcg for oral administration.


Treatment: Drugs: Ralinepag
Active
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Pulmonary Vascular Resistance
Assessment method [1] 0 0
Pulmonary vascular resistance was collected by right heart catheterization (RHC).
Timepoint [1] 0 0
At 1 or 2 years after the subject enrolled into the study, pending their last RHC prior to Protocol Amendment 2.
Primary outcome [2] 0 0
Change From Baseline in Cardiac Output
Assessment method [2] 0 0
Cardiac output was collected by right heart catheterization (RHC).
Timepoint [2] 0 0
At 1 or 2 years after the subject enrolled into the study, pending their last RHC prior to Protocol Amendment 2.
Primary outcome [3] 0 0
Change From Baseline in Cardiac Index
Assessment method [3] 0 0
Cardiac index was collected by right heart catheterization (RHC).
Timepoint [3] 0 0
At 1 or 2 years after the subject enrolled into the study, pending their last RHC prior to Protocol Amendment 2.
Primary outcome [4] 0 0
Change From Baseline in Mean Pulmonary Arterial Pressure
Assessment method [4] 0 0
Mean pulmonary arterial pressure was collected by right heart catheterization (RHC).
Timepoint [4] 0 0
At 1 or 2 years after the subject enrolled into the study, pending their last RHC prior to Protocol Amendment 2.
Secondary outcome [1] 0 0
Time From Randomization to the First Protocol-defined Clinical Worsening Event
Assessment method [1] 0 0
Clinical worsening events were defined as death, or onset of a treatment-emergent adverse event (AE) with a fatal outcome occurring =14 days after treatment discontinuation; hospitalization for worsening PAH, heart-lung or lung transplant, or atrial septostomy; necessity of addition (or dose change) of any prostacyclin/prostacyclin analogue, phosphodiesterase type 5 inhibitor (PDE5-I) or soluble guanylate cyclase (sGC), or endothelin receptor antagonist (ERA); and the combined occurrence of a decrease in 6-Minute Walk Distance (6MWD) by at least 20% from Baseline, confirmed on two 6-Minute Walk Tests (6MWTs) on different days; worsening in WHO/New York Heart Association (NYHA) Functional Class (FC) from Baseline; and appearance of or worsening of signs/symptoms of right heart failure that did not respond to optimized oral diuretic therapy.
Timepoint [1] 0 0
From Baseline to 28 days following discontinuation of study drug, up to 235 weeks.
Secondary outcome [2] 0 0
Change From Baseline in 6MWD
Assessment method [2] 0 0
6MWD was measured at Baseline (prior to starting study drug) and every 3 months thereafter including the End of Study Visit.
Timepoint [2] 0 0
From Baseline to discontinuation of study drug, up to 235 weeks
Secondary outcome [3] 0 0
Change From Baseline in WHO/NYHA FC
Assessment method [3] 0 0
WHO/NYHA FC was measured at Baseline (prior to starting study drug) and every 3 months thereafter including at the End of Study and 28-Day Follow-up Visits. FC recorded as I, II, III, or IV based on the following: I: PH but without limitation of physical activity; physical activity without undue dyspnea or fatigue, chest pain or near syncope. II: PH with slight limitation of physical activity; physical activity causes undue dyspnea or fatigue, chest pain or near syncope. III: PH with marked limitation of physical activity; less than ordinary activity causes undue dyspnea or fatigue, chest pain or near syncope. IV: PH with inability to carry out any physical activity without symptoms. Signs of right heart failure. Dyspnea and/or fatigue at rest. Discomfort is increased by any physical activity.
Timepoint [3] 0 0
From Baseline to 28 days following discontinuation of study drug, up to 235 weeks

Eligibility
Key inclusion criteria
* Evidence of a personally signed and dated informed consent document.
* Was willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures and was deemed an appropriate candidate for participation in a long-term extension study.
* Female subjects were nonpregnant, nonlactating, surgically sterile or postmenopausal, or agreed to use an accepted method of birth control for at least 3 months prior to the first dose, during, and for at least 30 days after the last dose of study drug.
* Male subjects were either surgically sterile or agreed to use a condom with spermicide when sexually active with a female partner who was not using an acceptable method of birth control during the study and for 30 days after the last dose of study drug.
* Male and female subjects agreed not to participate in a conception process during the study and for 30 days after the last dose of study drug.
* Fulfilled all eligibility criteria for Study APD811-003 and completed the study as planned.

Subjects who were assigned to placebo in Study APD811-003 and experienced clinical worsening in that study could enroll in Study APD811-007 after completing all end of study procedures per protocol, including RHC, for Study APD811-003 and had their data locked.
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Subjects who enrolled in Study APD811-003 and were withdrawn from study drug treatment due to any adverse event (AE), serious adverse event (SAE), or subjects who did not complete Study APD811003, with the exception made for placebo-treated subjects who experienced a clinical worsening event.
* Female •subjects who wished to become pregnant.
* Systolic blood pressure <90 mmHg at Baseline.
* Other severe acute or chronic medical or laboratory abnormalities that could have increased the risk associated with study participation or investigational product administration or interfered with the interpretation of study results and, in the judgment of the investigator, would have made the subject inappropriate for entry into this study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/07/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
29/03/2021
Sample size
Target
Accrual to date
Final
45
Recruitment in Australia
Recruitment state(s)
NSW,QLD,TAS,VIC
Recruitment hospital [1] 0 0
St. Vincent's Hospital - Darlinghurst
Recruitment hospital [2] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [3] 0 0
Royal Hobart Hospital - Hobart
Recruitment hospital [4] 0 0
St Vincent's Hospital - Fitzroy
Recruitment hospital [5] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [2] 0 0
4032 - Chermside
Recruitment postcode(s) [3] 0 0
7001 - Hobart
Recruitment postcode(s) [4] 0 0
3065 - Fitzroy
Recruitment postcode(s) [5] 0 0
6150 - Murdoch
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Iowa
Country [6] 0 0
United States of America
State/province [6] 0 0
Maine
Country [7] 0 0
United States of America
State/province [7] 0 0
Maryland
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Michigan
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
Bulgaria
State/province [13] 0 0
Sofia
Country [14] 0 0
Czechia
State/province [14] 0 0
Olomouc
Country [15] 0 0
Czechia
State/province [15] 0 0
Prague
Country [16] 0 0
Hungary
State/province [16] 0 0
Budapest
Country [17] 0 0
Hungary
State/province [17] 0 0
Debrecen
Country [18] 0 0
Poland
State/province [18] 0 0
Krakow
Country [19] 0 0
Poland
State/province [19] 0 0
Kraków
Country [20] 0 0
Poland
State/province [20] 0 0
Lodz
Country [21] 0 0
Romania
State/province [21] 0 0
Bucharest
Country [22] 0 0
Romania
State/province [22] 0 0
Timisoara
Country [23] 0 0
Serbia
State/province [23] 0 0
Belgrade
Country [24] 0 0
Serbia
State/province [24] 0 0
Sremska Kamenica
Country [25] 0 0
Slovakia
State/province [25] 0 0
Bratislava
Country [26] 0 0
Slovakia
State/province [26] 0 0
Košice
Country [27] 0 0
Spain
State/province [27] 0 0
Barcelona
Country [28] 0 0
Spain
State/province [28] 0 0
Madrid

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
United Therapeutics
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02279745