Treprostinil sodium for Pulmonary arterial hypertension

Inclusion criteria

• {"criterion_text":"- 1. Signed informed consent by the parents or the legal representatives and written assent from appropriately aged participants\n- 2. Males or females from birth to under 18 years of age at the time informed consent was signed\n- 3. Confirmed diagnosis of severe PAH classified as PH Group 1 requiring a treatment with prostacyclin infusion\n- 4. Current diagnosis of PAH confirmed by right heart catheterisation (RHC) at screening or by historical RHC prior to screening with following haemodynamic findings: •\tMean pulmonary arterial pressure (mPAP) >20 mmHg •\tPulmonary vascular resistance Index (PVRI) >3 Wood Units (WU) m² If RHC is not possible due to medical reasons (e.g. neonates and infants), the confirmation by ECHO at the screening is sufficient.\n- 5. PAH-treatment naïve or pretreated patients\n- 6. A subject is eligible to participate in this study, as assessed by the investigator, if they are of: •\tNon-childbearing potential (i.e., physiologically incapable of becoming pregnant); or, •\tChild-bearing potential - has a negative pregnancy test and is not lactating and, if sexually active, agrees to continue to use 2 reliable methods of contraception until study completion and for at least 30 days following the last dose of study drug. Examples of reliable birth control methods include true abstinence as a lifestyle choice (periodic sexual abstinence method is not acceptable); the use of oral contraceptives; a reliable barrier method of birth control (diaphragms with contraceptive jelly; cervical caps with contraceptive jelly; condoms with contraceptive foam; intrauterine devices)"}

Exclusion criteria

• {"criterion_text":"- 1. Known intolerance to prostacyclin analogues\n- 13. Other concurrent severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study\n- 14. Participation in another clinical trial of an investigational drug or device (including placebo) within 5 half-lives (max. 30 days) prior to screening\n- 3. PH related to conditions other than specified above\n- 4. Unrepaired congenital heart disease if surgery is planned within next 5 months\n- 5. Subjects diagnosed with any lung disease\n- 2. Patient receiving any other prostacyclin analogue, except parenteral treprostinil, or any prostacyclin receptor agonist within 72 hours prior to enrolment\n- 6. Acutely decompensated heart failure within previous 30 days from screening\n- 7. Subjects who have had an atrial septostomy or potts shunt within the previous 6 months of screening\n- 8. Any clinically significant laboratory abnormality that precludes initiation or continuation of treprostinil therapy\n- 9. Moderate to severe hepatic dysfunction as defined by elevated liver function tests (aspartate aminotransferase or alanine aminotransferase) ≥3 times the upper limit of normal at Screening, or Child Pugh class B or C hepatic disease\n- 15. Patients not able to handle pumps and infusion site if there is no parent, family member, guardian present in their household taking over pump handling or if they are not treated in hospital set-up\n- 10. Subjects who are pregnant or breastfeeding\n- 11. Haematological abnormalities (e.g., severe anaemia, Hgb <10 g/dL, leukopenia, WBC <2500/µL)\n- 12. History of substance use disorder, unless a proof of abstinence ≥1 year is provided"}

Primary endpoints

• {"endpoint_text":"- The frequency and seriousness of adverse events and adverse drug reactions during the first 5 months (20 weeks ± 1 weeks) of treatment according to Common Terminology Criteria for Adverse Events (CTCAE, version 5.0).","definition_or_measurement_approach":"Assessed during the first 5 months (20 weeks ±1 week) and graded using Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 (frequency and seriousness of AEs and ADRs)."}

Secondary endpoints

• {"endpoint_text":"- Change from baseline in quality of Life (QoL) as assessed by the Pediatric Quality of Life Inventory (PedsQoL Version 4.0) questionnaire.","definition_or_measurement_approach":"Measured as change from baseline using the PedsQoL Version 4.0 questionnaire."}
• {"endpoint_text":"- Change from baseline in 6MWD (patients > 6 years)","definition_or_measurement_approach":"Measured as change from baseline in 6-minute walk distance for patients older than 6 years."}
• {"endpoint_text":"- Change from baseline in WHO FC","definition_or_measurement_approach":"Measured as change from baseline in World Health Organization Functional Class."}
• {"endpoint_text":"- Change from baseline in echocardiography (ECHO) parameters","definition_or_measurement_approach":"Measured as change from baseline in specified echocardiographic parameters (ECHO)."}
• {"endpoint_text":"- Change from baseline in plasma N-terminal pro-brain natriuretic peptide (NTproBNP)","definition_or_measurement_approach":"Measured as change from baseline in plasma NT-proBNP concentration."}
• {"endpoint_text":"- The frequency and seriousness of adverse events and adverse drug reactions according to Common Terminology Criteria for Adverse Events (CTCAE, version 5.0) during the observational period","definition_or_measurement_approach":"Assessed during the observational period and graded using CTCAE v5.0."}
• {"endpoint_text":"- Treprostinil plasma concentration","definition_or_measurement_approach":"Measurement of treprostinil concentration in plasma (pharmacokinetic sampling)."}

Methods

• Country-specific recruitment procedures documented (recruitment procedure documents: K1_AT_Recruitment Procedure, K1_FR_Recruitment Procedure_Bilingual, K1_ES_Recruitment Procedure, K1_SK_Recruitment Procedure, K1_DE_Recruitment Procedure, K1_HU_Recruitment Procedure) — specific channel details not present in the JSON.
• Use of Scout services referenced (Scout Study Brochure, Scout Email Comm, Scout-related subject materials) and mentions of Travel Concierge/Patient Reimbursement via Scout (central printing and travel concierge referenced in third-party duties).

Austria

Earliest CTIS Part Ii Submission Date

11-01-2024

Latest Decision Or Authorization Date

23-06-2025

Processing Time Days

529

Number Of Sites

1

Number Of Participants

5

France

Earliest CTIS Part Ii Submission Date

13-11-2023

Latest Decision Or Authorization Date

18-06-2025

Processing Time Days

583

Number Of Sites

1

Number Of Participants

10

Spain

Earliest CTIS Part Ii Submission Date

01-12-2023

Latest Decision Or Authorization Date

19-06-2025

Processing Time Days

566

Number Of Sites

2

Number Of Participants

10

Slovakia

Earliest CTIS Part Ii Submission Date

11-10-2024

Latest Decision Or Authorization Date

17-06-2025

Processing Time Days

249

Number Of Sites

1

Number Of Participants

5

Germany

Earliest CTIS Part Ii Submission Date

23-09-2025

Latest Decision Or Authorization Date

28-10-2025

Processing Time Days

35

Number Of Sites

1

Number Of Participants

2

Hungary

Earliest CTIS Part Ii Submission Date

29-11-2023

Latest Decision Or Authorization Date

20-02-2026

Processing Time Days

814

Number Of Sites

1

Number Of Participants

5

Primary sponsor

Full Name

Aop Orphan Pharmaceuticals GmbH

Organisation Type

Pharmaceutical company

Country Of Registered Address

Austria

Contract research organisations

Name

Icon Clinical Research Limited

Responsibilities

Clinical research organisation duties per sponsorDuties codes [1,12,15,2,5]; central printing and travel concierge/patient reimbursement via Scout referenced; contact CTIS-Biotech@iconplc.com

Name

Datamedrix GmbH

Responsibilities

Data management/related responsibilities per sponsorDuties codes [6,7]; contact a.redl@datamedrix.com

Third parties

• {"country":"Austria","full_name":"Pharm-Analyt Labor Ges.m.b.H.","duties_or_roles":"sponsorDuties codes: [4]; contact office@pharm-analyt.com","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"sponsorDuties codes: [1,12,15,2,5]; includes 'Central Printing by Imperial and Travel Concierge/Patient Reimbursement via Scout' (value provided in JSON); contact CTIS-Biotech@iconplc.com","organisation_type":"Pharmaceutical company"}
• {"country":"Austria","full_name":"Datamedrix GmbH","duties_or_roles":"sponsorDuties codes: [6,7]; contact a.redl@datamedrix.com","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Czechia","full_name":"Aixial s.r.o.","duties_or_roles":"sponsorDuties codes: [8]; contact AOP-vigilance@aixial.com","organisation_type":"Pharmaceutical company"}
• {"country":"Austria","full_name":"Gnn GmbH & Co. KG","duties_or_roles":"sponsorDuties codes: [15]; value: 'Clinical Trial Logistics'; contact office@gnn-group.com","organisation_type":"Non-Pharmaceutical company"}