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Tiprelestat Versus Placebo When Added to Standard of Care for the Treatment of Pulmonary Arterial Hypertension (PAH) (ATHENA)

15 mei 2026 bijgewerkt door: Roham T. Zamanian, Stanford University

A Phase II, Randomized, Double-Blind, Safety and Efficacy Study of Tiprelestat Versus Placebo When Added to Standard of Care for the Treatment of Pulmonary Arterial Hypertension (PAH)

The primary objective of this study is to compare the efficacy, safety, and tolerability of tiprelestat plus Standard of Care (SOC) compared with placebo plus SOC in patients with World Health Organization (WHO) functional class II-IV pulmonary arterial hypertension (PAH).

Studie Overzicht

Studietype

Ingrijpend

Inschrijving (Geschat)

90

Fase

  • Fase 2

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

    • Arizona
      • Tucson, Arizona, Verenigde Staten, 85719
        • The University of Arizona / Banner University Medical Center
    • California
      • San Francisco, California, Verenigde Staten, 94143
        • University of California, San Francisco
      • Stanford, California, Verenigde Staten, 94305
        • Stanford University
    • Colorado
      • Aurora, Colorado, Verenigde Staten, 80045
        • University of Colorado (UCD) Anschutz
    • Massachusetts
      • Boston, Massachusetts, Verenigde Staten, 02138
        • Harvard University / Brigham and Women's Hospital
    • North Carolina
      • Durham, North Carolina, Verenigde Staten, 27710
        • Duke University
    • Pennsylvania
      • Philadelphia, Pennsylvania, Verenigde Staten, 19104
        • University of Pennsylvania
    • Rhode Island
      • Providence, Rhode Island, Verenigde Staten, 02904
        • Brown University/Rhode Island Hospital
    • Texas
      • Dallas, Texas, Verenigde Staten, 75390
        • University of Texas Southwestern
    • Washington
      • Seattle, Washington, Verenigde Staten, 98195
        • University of Washington

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

  • Volwassen
  • Oudere volwassene

Accepteert gezonde vrijwilligers

Nee

Beschrijving

Inclusion Criteria:

  1. Adults age 18 to 75 years.
  2. Willingness to give written informed consent prior to any study-related procedures being performed and to be able to adhere to the study restrictions and examination schedule.
  3. Diagnosis of WHO Group I PAH.
  4. WHO functional class II - IV despite optimized treatment SOC, with 1 or more modalities including phosphodiesterase 5 (PDE5) inhibitor, soluble guanylate cyclase stimulator (sGCS), endothelin receptor antagonist (ERA), and/or a prostacyclin analogue or receptor agonist (SC/inhaled/PO) (see #5), as well as Sotatercept (see #6).
  5. On stable doses of PDE5 inhibitor, ERA, sGCS, or prostacyclin analogue/receptor agonist for at least 90 days prior to screening; for infusion prostacyclins, dose adjustment within 10% of baseline dose during the duration of the study is allowed per medical practice.
  6. On stable doses of Sotatercept therapy for at least 6 months prior to screening, and intended to be continued during the duration of the study.
  7. Screening right heart catheterization mean pulmonary arterial pressure (mPAP) ≥ 25 mmHg at rest; pulmonary wedge pressure (PAWP) ≤ 15 mmHg or left ventricular end diastolic pressure (LVEDP) ≤ 15 mmHg; AND pulmonary vascular resistance (PVR) ≥ 400 dynes•sec/cm5 (5 Wood Units).
  8. If participant is of childbearing potential, willing to use adequate methods of contraception throughout the course of the study. If participant is of childbearing potential (a participant < 55 years of age who has not been postmenopausal for ≥ 5 years or who has not had a bilateral salpingectomy, hysterectomy and/or oophorectomy), need to employ two reliable means of contraception which may include surgical sterilization, barrier methods, spermicidals, intrauterine devices, and/or hormonal contraception, unless the participant chooses abstinence (to avoid heterosexual intercourse completely). If a participant chooses abstinence, then a second reliable means of contraception is not needed.
  9. 6MWD ≥100 and ≤500 meters at screening.
  10. Willing to adhere to study restrictions and examination schedule.

Exclusion Criteria:

  1. Diagnosis of WHO Group 2 - 5 Pulmonary Hypertension.
  2. Participation in another clinical trial, or experimental use, involving a PAH investigational drug or device within the last 3 months.
  3. Total lung capacity (TLC) < 60% predicted; if TLC is ≥ 60% and < 70% predicted, high resolution computed tomography (HRCT) must be available to exclude significant interstitial lung disease.
  4. FEV1 / FVC < 70% predicted and FEV1 < 60% predicted.
  5. Significant left-sided heart disease (based on screening Echocardiogram):

    1. Moderate or severe aortic or mitral valve disease
    2. Diastolic dysfunction ≥ Grade II
    3. LV systolic function < 45%
    4. Pericardial constriction
    5. Restrictive cardiomyopathy
    6. Significant coronary disease with demonstrable ischemia
  6. Chronic renal insufficiency defined as an estimated creatinine clearance < 30 ml/min.
  7. Current atrial arrhythmias not under optimal control.
  8. Uncontrolled systemic hypertension: SBP > 160 mmHg or DBP > 100mmHg.
  9. Severe hypotension: SBP < 80 mmHg.
  10. Pregnant or breast-feeding.
  11. Psychiatric, addictive, or other disorders that compromise the patient's ability to provide informed consent, to follow study protocol, and adhere to treatment instructions.
  12. Known allergy or hypersensitivity to tiprelestat.
  13. Moderate to severe hepatic dysfunction with a Child Pugh score >10.
  14. Hyperkalemia defined as Potassium > 5.1 mEq/L at screening.
  15. Initiation of an exercise program for cardiopulmonary rehabilitation within 90 days prior to screening or planned during the study. Subjects who are already stable in the maintenance phase of an exercise program which will continue for the duration of the study are eligible.
  16. Known active infection requiring antibiotic, antifungal, or antiviral therapies. Patients may be rescreened at physician discretion after the resolution of infection and discontinuation of antibiotic, antifungal, or antiviral therapies.
  17. Co-morbid conditions that would impair a patient's exercise performance and ability to assess WHO functional class, including but not limited to chronic low-back pain or peripheral musculoskeletal problems, other comorbidities expected to alter the patient's clinical course (i.e. active cancer; >3 comorbidities e.g., obesity, systemic HTN, diabetes).

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Verdrievoudigen

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: Tiprelestat (5 mg)
Participants receive tiprelestat injection daily for 168 days.
5 mg of tiprelestat in 1 mL saline administered as a daily subcutaneous injection for 168 days.
Andere namen:
  • Elafin
Experimenteel: Tiprelestat (10 mg)
Participants receive tiprelestat injection daily for 168 days.
10 mg of tiprelestat in 1 mL saline administered as a daily subcutaneous injection for 168 days.
Andere namen:
  • Elafin
Placebo-vergelijker: Placebo (1 mL 0.9% saline solution)
Participants receive placebo injection (matching tiprelestat) daily for 168 days.
Matching 1 mL 0.9% saline solution administered as a daily subcutaneous injection for 168 days.

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Change in Pulmonary Vascular Resistance (PVR)
Tijdsspanne: Baseline to week 24
PVR is calculated based on direct measurements during the right heart catheterization (RHC) procedure. PVR = (mPAP - PAWP) / CO, where mPAP is the mean pulmonary artery pressure, PAWP is the pulmonary wedge arterial pressure, and CO is the cardiac output. These cardiac measures are also obtained from right heart catheterization. PVR is measured in Wood units (WU) or dynes (dynes*sec/cm5), and higher values are associated with more severe disease. Normal range for PVR is 1-3 WU (80-240 dynes*sec/cm5) and can be as high as 30 WU (2,400 80-240 dynes*sec/cm5) in disease.
Baseline to week 24

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Onderzoekers

  • Hoofdonderzoeker: Roham T Zamanian, MD, Stanford University

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Geschat)

1 juli 2026

Primaire voltooiing (Geschat)

1 februari 2030

Studie voltooiing (Geschat)

1 maart 2030

Studieregistratiedata

Eerst ingediend

15 mei 2026

Eerst ingediend dat voldeed aan de QC-criteria

15 mei 2026

Eerst geplaatst (Werkelijk)

22 mei 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

22 mei 2026

Laatste update ingediend die voldeed aan QC-criteria

15 mei 2026

Laatst geverifieerd

1 mei 2026

Meer informatie

Termen gerelateerd aan deze studie

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

NEE

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

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