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

15. mai 2026 oppdatert av: 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).

Studieoversikt

Studietype

Intervensjonell

Registrering (Antatt)

90

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Beskrivelse

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).

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Trippel

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: 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.
Andre navn:
  • Elafin
Eksperimentell: 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.
Andre navn:
  • Elafin
Placebo komparator: 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.

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Change in Pulmonary Vascular Resistance (PVR)
Tidsramme: 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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Etterforskere

  • Hovedetterforsker: Roham T Zamanian, MD, Stanford University

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Antatt)

1. juli 2026

Primær fullføring (Antatt)

1. februar 2030

Studiet fullført (Antatt)

1. mars 2030

Datoer for studieregistrering

Først innsendt

15. mai 2026

Først innsendt som oppfylte QC-kriteriene

15. mai 2026

Først lagt ut (Faktiske)

22. mai 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

22. mai 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

15. mai 2026

Sist bekreftet

1. mai 2026

Mer informasjon

Begreper knyttet til denne studien

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

NEI

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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