Trial Review

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03683186




Registration number
NCT03683186
Ethics application status
Date submitted
19/09/2018
Date registered
25/09/2018
Date last updated
23/05/2024

Titles & IDs
Public title
A Study Evaluating the Long-Term Efficacy and Safety of Ralinepag in Subjects With PAH Via an Open-Label Extension
Scientific title
A Phase 3 Open-label Extension (OLE) Study to Evaluate the Long-term Safety and Efficacy of Ralinepag in Subjects With World Health Organization (WHO) Group 1 Pulmonary Arterial Hypertension (PAH)
Secondary ID [1] 0 0
APD811-303
Secondary ID [2] 0 0
ROR-PH-303
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
PAH 0 0
Pulmonary Hypertension 0 0
Pulmonary Arterial Hypertension 0 0
Hypertension 0 0
Connective Tissue Diseases 0 0
Familial Primary Pulmonary Hypertension 0 0
Vascular Diseases 0 0
Cardiovascular Diseases 0 0
Hypertension, Pulmonary 0 0
Lung Diseases 0 0
Respiratory Tract Disease 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
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Skin 0 0 0 0
Other skin conditions

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

Experimental: Ralinepag - Ralinepag once daily extended-release tablets (oral) 50, 250, and 400 mcg titrated to the highest tolerated dose.


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

Outcomes
Primary outcome [1] 0 0
Number of subjects with treatment-emergent adverse events [Safety and Tolerability]
Timepoint [1] 0 0
Up to 6 years

Eligibility
Key inclusion criteria
1. Evidence of a personally signed and dated informed consent form indicating that the
subject has been informed of all pertinent aspects of the study prior to initiation of
any study-related procedures.

2. Subjects who are willing and able to comply with scheduled visits, treatment plan,
laboratory tests, and other study procedures.

3. Completed the protocol-defined Study Drug Termination Visit or End of Study Visit
procedures in the preceding ralinepag study.

4. Both male and female subjects agree to use a medically acceptable method of
contraception throughout the entire study period from informed consent through the 30
day Follow-up Visit, if the possibility of conception exists. Eligible male and female
subjects must also agree not to participate in a conception process (i.e., actively
attempt to become pregnant or to impregnate, sperm donation, in vitro fertilization)
during the study and for 30 days after the last dose of ralinepag.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Subjects who prematurely discontinued investigational medicinal product (IMP) due to a
drug-related AE/SAE or tolerability issue in the preceding ralinepag study in which
they were enrolled, or subjects who did not complete all protocol defined study
procedures at a Study Drug Termination Visit or End of Study Visit in the preceding
ralinepag study.

2. Subjects who withdrew consent during participation in another ralinepag study.

3. Female subjects who wish to become pregnant or who have a positive pregnancy test on
Day 1 (OLE Entry Visit), or are lactating or breastfeeding.

4. Subjects who have undergone lung or heart/lung transplant or the initiation of
long-term parenteral or inhaled therapy with a prostacyclin during the time since
participation in their original ralinepag study.

5. Subjects who had an emergency unblinding procedure in a prior Phase 2 or 3 study.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
23/09/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2024
Actual
Sample size
Target
1000
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,TAS,VIC,WA
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
St Vincent's Hospital Sydney - Darlinghurst
Recruitment hospital [3] 0 0
Macquarie University - Sydney
Recruitment hospital [4] 0 0
Westmead Hospital - Sydney
Recruitment hospital [5] 0 0
Nepean Hospital - Sydney
Recruitment hospital [6] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [7] 0 0
Princess Alexandra Hospital - Woolloongabba
Recruitment hospital [8] 0 0
Royal Hobart Hospital - Hobart
Recruitment hospital [9] 0 0
St Vincent's Hospital Melbourne - Fitzroy
Recruitment hospital [10] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [11] 0 0
Fiona Stanley Hospital - Perth
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [3] 0 0
2109 - Sydney
Recruitment postcode(s) [4] 0 0
2145 - Sydney
Recruitment postcode(s) [5] 0 0
2751 - Sydney
Recruitment postcode(s) [6] 0 0
4032 - Chermside
Recruitment postcode(s) [7] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [8] 0 0
7000 - Hobart
Recruitment postcode(s) [9] 0 0
3065 - Fitzroy
Recruitment postcode(s) [10] 0 0
3004 - Melbourne
Recruitment postcode(s) [11] 0 0
6150 - Perth
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
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Colorado
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Georgia
Country [7] 0 0
United States of America
State/province [7] 0 0
Illinois
Country [8] 0 0
United States of America
State/province [8] 0 0
Indiana
Country [9] 0 0
United States of America
State/province [9] 0 0
Iowa
Country [10] 0 0
United States of America
State/province [10] 0 0
Kentucky
Country [11] 0 0
United States of America
State/province [11] 0 0
Louisiana
Country [12] 0 0
United States of America
State/province [12] 0 0
Maine
Country [13] 0 0
United States of America
State/province [13] 0 0
Maryland
Country [14] 0 0
United States of America
State/province [14] 0 0
Massachusetts
Country [15] 0 0
United States of America
State/province [15] 0 0
Michigan
Country [16] 0 0
United States of America
State/province [16] 0 0
Missouri
Country [17] 0 0
United States of America
State/province [17] 0 0
Nebraska
Country [18] 0 0
United States of America
State/province [18] 0 0
New Mexico
Country [19] 0 0
United States of America
State/province [19] 0 0
New York
Country [20] 0 0
United States of America
State/province [20] 0 0
North Carolina
Country [21] 0 0
United States of America
State/province [21] 0 0
Ohio
Country [22] 0 0
United States of America
State/province [22] 0 0
Oklahoma
Country [23] 0 0
United States of America
State/province [23] 0 0
Oregon
Country [24] 0 0
United States of America
State/province [24] 0 0
Pennsylvania
Country [25] 0 0
United States of America
State/province [25] 0 0
Tennessee
Country [26] 0 0
United States of America
State/province [26] 0 0
Texas
Country [27] 0 0
United States of America
State/province [27] 0 0
Vermont
Country [28] 0 0
United States of America
State/province [28] 0 0
Virginia
Country [29] 0 0
United States of America
State/province [29] 0 0
Wisconsin
Country [30] 0 0
Argentina
State/province [30] 0 0
Buenos Aires
Country [31] 0 0
Argentina
State/province [31] 0 0
Santa Fe De La Vera Cruz
Country [32] 0 0
Argentina
State/province [32] 0 0
Santa Fe
Country [33] 0 0
Argentina
State/province [33] 0 0
Caba
Country [34] 0 0
Argentina
State/province [34] 0 0
Ciudad Autonoma Buenos Aires
Country [35] 0 0
Argentina
State/province [35] 0 0
Cordoba
Country [36] 0 0
Argentina
State/province [36] 0 0
Corrientes
Country [37] 0 0
Argentina
State/province [37] 0 0
Córdoba
Country [38] 0 0
Argentina
State/province [38] 0 0
Mar Del Plata
Country [39] 0 0
Austria
State/province [39] 0 0
Innsbruck
Country [40] 0 0
Austria
State/province [40] 0 0
Linz
Country [41] 0 0
Austria
State/province [41] 0 0
Wien
Country [42] 0 0
Belgium
State/province [42] 0 0
Bruxelles
Country [43] 0 0
Belgium
State/province [43] 0 0
Leuven
Country [44] 0 0
Brazil
State/province [44] 0 0
Goias
Country [45] 0 0
Brazil
State/province [45] 0 0
MG
Country [46] 0 0
Brazil
State/province [46] 0 0
R.S
Country [47] 0 0
Brazil
State/province [47] 0 0
RS
Country [48] 0 0
Brazil
State/province [48] 0 0
Sao Paulo
Country [49] 0 0
Brazil
State/province [49] 0 0
SP
Country [50] 0 0
Bulgaria
State/province [50] 0 0
Sofia
Country [51] 0 0
Canada
State/province [51] 0 0
Alberta
Country [52] 0 0
Canada
State/province [52] 0 0
Ontario
Country [53] 0 0
Canada
State/province [53] 0 0
Quebec
Country [54] 0 0
Chile
State/province [54] 0 0
Santiago De Chile
Country [55] 0 0
China
State/province [55] 0 0
Beijing
Country [56] 0 0
China
State/province [56] 0 0
Guangdong
Country [57] 0 0
China
State/province [57] 0 0
Hunan
Country [58] 0 0
China
State/province [58] 0 0
Shanghai
Country [59] 0 0
Croatia
State/province [59] 0 0
Zagreb
Country [60] 0 0
Czechia
State/province [60] 0 0
Praha 2
Country [61] 0 0
Denmark
State/province [61] 0 0
Copenhagen
Country [62] 0 0
Denmark
State/province [62] 0 0
Århus
Country [63] 0 0
France
State/province [63] 0 0
Bas Rhin
Country [64] 0 0
France
State/province [64] 0 0
Isere
Country [65] 0 0
France
State/province [65] 0 0
Loire
Country [66] 0 0
France
State/province [66] 0 0
Meurthe Et Moselle
Country [67] 0 0
France
State/province [67] 0 0
Rhone
Country [68] 0 0
France
State/province [68] 0 0
Seine Maritime
Country [69] 0 0
France
State/province [69] 0 0
Val De Marne
Country [70] 0 0
France
State/province [70] 0 0
Besançon Cedex
Country [71] 0 0
France
State/province [71] 0 0
Brest cedex 2
Country [72] 0 0
France
State/province [72] 0 0
Lille Cedex
Country [73] 0 0
France
State/province [73] 0 0
Marseille
Country [74] 0 0
Germany
State/province [74] 0 0
Baden-Württemberg
Country [75] 0 0
Germany
State/province [75] 0 0
Mecklenburg-Vorpommern
Country [76] 0 0
Germany
State/province [76] 0 0
Rheinland Pfalz
Country [77] 0 0
Germany
State/province [77] 0 0
Saarland
Country [78] 0 0
Germany
State/province [78] 0 0
Sachsen
Country [79] 0 0
Germany
State/province [79] 0 0
Donaueschingen
Country [80] 0 0
Germany
State/province [80] 0 0
Dresden
Country [81] 0 0
Germany
State/province [81] 0 0
Hamburg
Country [82] 0 0
Greece
State/province [82] 0 0
Alexandroupolis
Country [83] 0 0
Greece
State/province [83] 0 0
Athens
Country [84] 0 0
Greece
State/province [84] 0 0
Chaidari
Country [85] 0 0
Greece
State/province [85] 0 0
Thessaloniki
Country [86] 0 0
Hungary
State/province [86] 0 0
Budapest
Country [87] 0 0
Hungary
State/province [87] 0 0
Pecs
Country [88] 0 0
Israel
State/province [88] 0 0
Haifa
Country [89] 0 0
Israel
State/province [89] 0 0
Jerusalem
Country [90] 0 0
Israel
State/province [90] 0 0
Kfar- Sava
Country [91] 0 0
Israel
State/province [91] 0 0
Petach Tikva
Country [92] 0 0
Israel
State/province [92] 0 0
Tel Aviv
Country [93] 0 0
Italy
State/province [93] 0 0
PA
Country [94] 0 0
Italy
State/province [94] 0 0
Foggia
Country [95] 0 0
Italy
State/province [95] 0 0
Genova
Country [96] 0 0
Italy
State/province [96] 0 0
Milano
Country [97] 0 0
Italy
State/province [97] 0 0
Milan
Country [98] 0 0
Italy
State/province [98] 0 0
Monza
Country [99] 0 0
Italy
State/province [99] 0 0
Pavia
Country [100] 0 0
Italy
State/province [100] 0 0
Roma
Country [101] 0 0
Korea, Republic of
State/province [101] 0 0
Gangnam-gu
Country [102] 0 0
Korea, Republic of
State/province [102] 0 0
Jongno-gu
Country [103] 0 0
Korea, Republic of
State/province [103] 0 0
Namdong-gu
Country [104] 0 0
Mexico
State/province [104] 0 0
Distrito Federal
Country [105] 0 0
Mexico
State/province [105] 0 0
Jalisco
Country [106] 0 0
Mexico
State/province [106] 0 0
N.l.
Country [107] 0 0
Netherlands
State/province [107] 0 0
Amsterdam
Country [108] 0 0
Poland
State/province [108] 0 0
Bialystok
Country [109] 0 0
Poland
State/province [109] 0 0
Krakow
Country [110] 0 0
Poland
State/province [110] 0 0
Otwock
Country [111] 0 0
Poland
State/province [111] 0 0
Poznan
Country [112] 0 0
Poland
State/province [112] 0 0
Wroclaw
Country [113] 0 0
Portugal
State/province [113] 0 0
Almada
Country [114] 0 0
Portugal
State/province [114] 0 0
Coimbra
Country [115] 0 0
Portugal
State/province [115] 0 0
Lisboa
Country [116] 0 0
Portugal
State/province [116] 0 0
Porto
Country [117] 0 0
Romania
State/province [117] 0 0
Bucharest
Country [118] 0 0
Romania
State/province [118] 0 0
Bucuresti
Country [119] 0 0
Romania
State/province [119] 0 0
Cluj-Napoca
Country [120] 0 0
Romania
State/province [120] 0 0
Timisoara
Country [121] 0 0
Serbia
State/province [121] 0 0
Belgrade
Country [122] 0 0
Serbia
State/province [122] 0 0
Sremska Kamenica
Country [123] 0 0
Singapore
State/province [123] 0 0
Singapore
Country [124] 0 0
Spain
State/province [124] 0 0
Barcelona
Country [125] 0 0
Spain
State/province [125] 0 0
Madrid
Country [126] 0 0
Spain
State/province [126] 0 0
Valencia
Country [127] 0 0
Sweden
State/province [127] 0 0
Göteborg
Country [128] 0 0
Sweden
State/province [128] 0 0
Linköping
Country [129] 0 0
Sweden
State/province [129] 0 0
Umeå
Country [130] 0 0
Taiwan
State/province [130] 0 0
Tainan
Country [131] 0 0
Taiwan
State/province [131] 0 0
Taipei
Country [132] 0 0
Turkey
State/province [132] 0 0
Odumpazari
Country [133] 0 0
Turkey
State/province [133] 0 0
Saricam
Country [134] 0 0
Turkey
State/province [134] 0 0
Yenisehir
Country [135] 0 0
Turkey
State/province [135] 0 0
Ankara
Country [136] 0 0
Turkey
State/province [136] 0 0
Bursa
Country [137] 0 0
Turkey
State/province [137] 0 0
Istanbul
Country [138] 0 0
Turkey
State/province [138] 0 0
Izmir
Country [139] 0 0
Ukraine
State/province [139] 0 0
Dnipro
Country [140] 0 0
Ukraine
State/province [140] 0 0
Kyiv
Country [141] 0 0
Ukraine
State/province [141] 0 0
Lviv
Country [142] 0 0
United Kingdom
State/province [142] 0 0
Greater London
Country [143] 0 0
United Kingdom
State/province [143] 0 0
Tyne & Wear
Country [144] 0 0
United Kingdom
State/province [144] 0 0
West Dunbartonshire
Country [145] 0 0
United Kingdom
State/province [145] 0 0
London

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

Ethics approval
Ethics application status

Summary
Brief summary
Study ROR-PH-303, ADVANCE EXTENSION, is an open-label extension (OLE) study for participants
with WHO Group 1 PAH who have participated in another Phase 2 or Phase 3 study of ralinepag.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03683186
Trial related presentations / publications
Public notes

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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03683186