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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02310672




Registration number
NCT02310672
Ethics application status
Date submitted
4/12/2014
Date registered
8/12/2014
Date last updated
24/09/2020

Titles & IDs
Public title
REPAIR: Right vEntricular Remodeling in Pulmonary ArterIal hypeRtension
Scientific title
A Prospective, Multicenter, Single-arm, Open-label, Phase 4 Study to Evaluate the Effects of Macitentan on Right vEntricular Remodeling in Pulmonary ArterIal hypeRtension Assessed by Cardiac Magnetic Resonance Imaging
Secondary ID [1] 0 0
AC-055-403
Universal Trial Number (UTN)
Trial acronym
REPAIR
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
Cardiovascular 0 0 0 0
Other cardiovascular diseases

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

Experimental: Macitentan - All patients take open-label macitentan 10mg o.d.


Treatment: Drugs: Macitentan
All patients take open-label macitentan 10mg o.d.

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

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Right Ventricular Stroke Volume (RVSV) to Week 26
Timepoint [1] 0 0
Baseline and Week 26
Primary outcome [2] 0 0
Ratio of Week 26 to Baseline Pulmonary Vascular Resistance (PVR)
Timepoint [2] 0 0
Baseline and Week 26
Secondary outcome [1] 0 0
Change From Baseline in Right Ventricular End Diastolic Volume (RVEDV) to Week 26
Timepoint [1] 0 0
Baseline to Week 26
Secondary outcome [2] 0 0
Change From Baseline in Right Ventricular End Systolic Volume (RVESV) to Week 26
Timepoint [2] 0 0
Baseline to Week 26
Secondary outcome [3] 0 0
Change From Baseline in Right Ventricular Ejection Fraction (RVEF) to Week 26 (% Blood Volume)
Timepoint [3] 0 0
Baseline to Week 26
Secondary outcome [4] 0 0
Change From Baseline in Right Ventricle (RV) Mass to Week 26
Timepoint [4] 0 0
Baseline to Week 26
Secondary outcome [5] 0 0
Change From Baseline in Six-minutes Walk Distance (6MWD) to Week 26
Timepoint [5] 0 0
Baseline to Week 26
Secondary outcome [6] 0 0
Change From Baseline in World Health Organization Functional Class (WHO FC) to Week 26
Timepoint [6] 0 0
Baseline to Week 26

Eligibility
Key inclusion criteria
1. Signed informed consent prior to any study-mandated procedure
2. Symptomatic pulmonary arterial hypertension (PAH)
3. World Health Organization (WHO) Functional Class (FC) I to III
4. PAH etiology belonging to one of the following groups according to Nice classification:

* Idiopathic PAH
* Heritable PAH
* Drug- and toxin-induced PAH
* PAH associated with congenital heart diseases: only simple (atrial septal defect, ventricular septal defect, patent ductus arteriosus) congenital systemic to pulmonary shunts at least 2 year post surgical repair
5. Hemodynamic diagnosis of PAH confirmed by right heart catheterization (RHC) during screening showing:

• mean pulmonary arterial pressure (mPAP) = 25 mmHg and
* PCWP (pulmonary capillary wedge pressure) or left ventricular end diastolic pressure (LVEDP) = 12 mmHg and pulmonary vascular resistance (PVR) = 4 Wood Units (WU) (320 dyn.sec.cm-5) or
* 12 mmHg = PCWP or LVEDP = 15 mmHg and PVR = 6WU (480 dyn.sec.cm-5)
6. 6-minute walk distance (6MWD) = 150 m during screening
7. For patients treated with oral diuretics, treatment dose must have been stable at least 1 month prior to RHC during the screening period
8. For patients treated with phosphodiesterase type-5 (PDE-5) inhibitors, treatment dose must have been stable at least 3 months prior to RHC during the screening period
9. For patients treated with beta blockers, treatment dose must have been stable at least 1 month prior to the RHC during the screening period
10. Men or women =18 and < 65 years
11. Women of childbearing potential (defined in protocol) must:

* Have a negative serum pregnancy test during screening and a negative urine pregnancy test on Day 1, and
* Agree to use reliable methods of contraception (defined in protocol) from screening up to 30 days after study treatment discontinuation, and
* Agree to perform monthly pregnancy tests up to 30 days after study treatment discontinuation
Minimum age
18 Years
Maximum age
64 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Body weight < 40 kg
2. Body mass index (BMI) > 35kg/m2. For patients with 30kg/m2 < BMI < 35kg/m2, an eligibility form will be submitted to a Steering Committee member who will reserve the right to exclude the patient.
3. Pregnancy, breastfeeding or intention to become pregnant during the study
4. Recently started (< 8 weeks prior to informed consent signature) or planned cardio-pulmonary rehabilitation program
5. Known concomitant life-threatening disease with a life expectancy < 12 months
6. Any condition likely to affect protocol or treatment compliance
7. Hospitalization for PAH within 3 months prior to informed consent signature
8. Left atrial volume indexed for body surface area = 43mL/m2 by echocardiography or cardiac MRI
9. Valvular disease grade 2 or higher
10. History of pulmonary embolism or deep vein thrombosis
11. Documented moderate to severe chronic obstructive pulmonary disease
12. Documented moderate to severe restrictive lung disease
13. Historical evidence of significant coronary artery disease established by:

* History of myocardial infarction or
* More than 50% stenosis in a coronary artery (by percutaneous coronary intervention or angiography) or
* Elevation of the ST segment on electrocardiogram or
* History of coronary artery bypass grafting or
* Stable angina
14. Diabetes mellitus
15. Moderate to severe renal insufficiency (calculated creatinine clearance < 60 mL/min/1.73 m2)
16. Cancer
17. Systolic blood pressure < 90 mmHg
18. Severe hepatic impairment (with or without cirrhosis) according to National Cancer Institute organ dysfunction working group criteria, defined as total bilirubin > 3 × upper limit of the normal range (ULN) accompanied by an aspartate aminotransferase (AST) elevation > ULN at Screening.
19. Hemoglobin < 100g/L
20. AST and/or alanine aminotransferase (ALT) > 3× ULN
21. Need for dialysis
22. Responders to acute vasoreactivity test based on medical history
23. Prior use of endothelin receptor antagonists (ERAs), stimulators of soluble guanylate cyclase or prostacyclin or prostacyclin analogues
24. Treatment with strong inducers of cytochrome P450 isozyme 3A4 (CYP3A4) within 4 weeks prior to study treatment initiation (e.g., carbamazepine, rifampicin, rifabutin, phenytoin and St. John's Wort)
25. Treatment with strong inhibitors of CYP3A4 within 4 weeks prior to study treatment initiation (e.g., ketoconazole, itraconazole, voriconazole, clarithromycin, telithromycin, nefazodone, ritonavir, and saquinavir)
26. Treatment with another investigational drug (planned, or taken within the 3 months prior to study treatment initiation).
27. Hypersensitivity to any ERA or any excipients of the formulation of macitentan (lactose, magnesium stearate, microcrystalline cellulose, povidone, sodium starch glycolate, polyvinyl alcohol, polysorbate, titanium dioxide, talc, xanthan gum, and lecithin soya)
28. Claustrophobia
29. Permanent cardiac pacemaker, automatic internal cardioverter
30. Metallic implant (e.g., defibrillator, neurostimulator, hearing aid, permanent use of infusion device)
31. Atrial fibrillation, multiple premature ventricular or atrial contractions, or any other condition that would interfere with proper cardiac gating during MRI.
32. For patients enrolling in the metabolism sub-study only: glucose intolerance
33. For patients enrolling in the biopsy sub-study only: PAH etiology belonging to Nice classification 1.4.4: PAH associated with congenital heart diseases

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
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 4
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
The Prince Charles Hospital - Chermside
Recruitment postcode(s) [1] 0 0
4032 - Chermside
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Massachusetts
Country [2] 0 0
United States of America
State/province [2] 0 0
Minnesota
Country [3] 0 0
United States of America
State/province [3] 0 0
Missouri
Country [4] 0 0
United States of America
State/province [4] 0 0
New Jersey
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Wisconsin
Country [9] 0 0
France
State/province [9] 0 0
Clermont-Ferrand
Country [10] 0 0
France
State/province [10] 0 0
La Tronche
Country [11] 0 0
France
State/province [11] 0 0
Lille Cedex
Country [12] 0 0
France
State/province [12] 0 0
Nancy
Country [13] 0 0
France
State/province [13] 0 0
Nantes Cedex 01
Country [14] 0 0
France
State/province [14] 0 0
Nice
Country [15] 0 0
France
State/province [15] 0 0
Paris
Country [16] 0 0
Germany
State/province [16] 0 0
Bonn
Country [17] 0 0
Germany
State/province [17] 0 0
Heidelberg
Country [18] 0 0
Germany
State/province [18] 0 0
Köln
Country [19] 0 0
Germany
State/province [19] 0 0
Mainz
Country [20] 0 0
Hong Kong
State/province [20] 0 0
Hong Kong
Country [21] 0 0
Israel
State/province [21] 0 0
Beer-Sheva
Country [22] 0 0
Israel
State/province [22] 0 0
Jerusalem
Country [23] 0 0
Italy
State/province [23] 0 0
Bologna
Country [24] 0 0
Italy
State/province [24] 0 0
Pavia
Country [25] 0 0
Malaysia
State/province [25] 0 0
George Town
Country [26] 0 0
Malaysia
State/province [26] 0 0
Kuala Lumpur
Country [27] 0 0
Netherlands
State/province [27] 0 0
Amsterdam
Country [28] 0 0
Netherlands
State/province [28] 0 0
Maastricht
Country [29] 0 0
Netherlands
State/province [29] 0 0
Nieuwegein
Country [30] 0 0
Netherlands
State/province [30] 0 0
Nijmegen
Country [31] 0 0
Netherlands
State/province [31] 0 0
Rotterdam
Country [32] 0 0
Russian Federation
State/province [32] 0 0
Moscow
Country [33] 0 0
Russian Federation
State/province [33] 0 0
Saint Petersburg
Country [34] 0 0
Singapore
State/province [34] 0 0
Singapore
Country [35] 0 0
United Kingdom
State/province [35] 0 0
Glasgow
Country [36] 0 0
United Kingdom
State/province [36] 0 0
London
Country [37] 0 0
United Kingdom
State/province [37] 0 0
Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Actelion
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Loïc Perchenet
Address 0 0
Actelion
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

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.