Tiprelestat Versus Placebo When Added to Standard of Care for the Treatment of Pulmonary Arterial Hypertension (PAH) (ATHENA)
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)
Panoramica dello studio
Stato
Stato
Condizioni
Condizioni
Intervento / Trattamento
Intervento / Trattamento
Tipo di studio
Tipo di studio
Iscrizione (Stimato)
Iscrizione
Fase
Fase
- Fase 2
Contatti e Sedi
Luoghi di studio
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Arizona
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Tucson, Arizona, Stati Uniti, 85719
- The University of Arizona / Banner University Medical Center
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California
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San Francisco, California, Stati Uniti, 94143
- University of California, San Francisco
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Stanford, California, Stati Uniti, 94305
- Stanford University
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Colorado
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Aurora, Colorado, Stati Uniti, 80045
- University of Colorado (UCD) Anschutz
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Massachusetts
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Boston, Massachusetts, Stati Uniti, 02138
- Harvard University / Brigham and Women's Hospital
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North Carolina
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Durham, North Carolina, Stati Uniti, 27710
- Duke University
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Pennsylvania
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Philadelphia, Pennsylvania, Stati Uniti, 19104
- University of Pennsylvania
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Rhode Island
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Providence, Rhode Island, Stati Uniti, 02904
- Brown University/Rhode Island Hospital
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Texas
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Dallas, Texas, Stati Uniti, 75390
- University of Texas Southwestern
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Washington
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Seattle, Washington, Stati Uniti, 98195
- University of Washington
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Criteri di partecipazione
Criteri di ammissibilità
Criteri di ammissibilità
Età idonea allo studio
- Adulto
- Adulto più anziano
Accetta volontari sani
Descrizione
Inclusion Criteria:
- Adults age 18 to 75 years.
- 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.
- Diagnosis of WHO Group I PAH.
- 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).
- 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.
- 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.
- 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).
- 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.
- 6MWD ≥100 and ≤500 meters at screening.
- Willing to adhere to study restrictions and examination schedule.
Exclusion Criteria:
- Diagnosis of WHO Group 2 - 5 Pulmonary Hypertension.
- Participation in another clinical trial, or experimental use, involving a PAH investigational drug or device within the last 3 months.
- 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.
- FEV1 / FVC < 70% predicted and FEV1 < 60% predicted.
Significant left-sided heart disease (based on screening Echocardiogram):
- Moderate or severe aortic or mitral valve disease
- Diastolic dysfunction ≥ Grade II
- LV systolic function < 45%
- Pericardial constriction
- Restrictive cardiomyopathy
- Significant coronary disease with demonstrable ischemia
- Chronic renal insufficiency defined as an estimated creatinine clearance < 30 ml/min.
- Current atrial arrhythmias not under optimal control.
- Uncontrolled systemic hypertension: SBP > 160 mmHg or DBP > 100mmHg.
- Severe hypotension: SBP < 80 mmHg.
- Pregnant or breast-feeding.
- Psychiatric, addictive, or other disorders that compromise the patient's ability to provide informed consent, to follow study protocol, and adhere to treatment instructions.
- Known allergy or hypersensitivity to tiprelestat.
- Moderate to severe hepatic dysfunction with a Child Pugh score >10.
- Hyperkalemia defined as Potassium > 5.1 mEq/L at screening.
- 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.
- 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.
- 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).
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione parallela
- Mascheramento: Triplicare
Numero di armi
Armi e interventi
Gruppo di partecipanti / ArmGruppo di partecipanti / Arm |
Intervento / TrattamentoIntervento / Trattamento |
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Sperimentale: Tiprelestat (5 mg)
Participants receive tiprelestat injection daily for 168 days.
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5 mg of tiprelestat in 1 mL saline administered as a daily subcutaneous injection for 168 days.
Altri nomi:
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Sperimentale: Tiprelestat (10 mg)
Participants receive tiprelestat injection daily for 168 days.
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10 mg of tiprelestat in 1 mL saline administered as a daily subcutaneous injection for 168 days.
Altri nomi:
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Comparatore placebo: Placebo (1 mL 0.9% saline solution)
Participants receive placebo injection (matching tiprelestat) daily for 168 days.
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Matching 1 mL 0.9% saline solution administered as a daily subcutaneous injection for 168 days.
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Change in Pulmonary Vascular Resistance (PVR)
Lasso di tempo: Baseline to week 24
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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.
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Baseline to week 24
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Collaboratori e investigatori
Sponsor
Sponsor
Collaboratori
Collaboratori
Investigatori
Investigatori
- Investigatore principale: Roham T Zamanian, MD, Stanford University
Studiare le date dei record
Studia le date principali
Inizio studio (Stimato)
Inizio studio
Completamento primario (Stimato)
Completamento primario
Completamento dello studio (Stimato)
Completamento dello studio
Date di iscrizione allo studio
Primo inviato
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Primo Inserito
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento pubblicato
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
- Malattie delle vie respiratorie
- Malattie polmonari
- Ipertensione, polmonare
- Ipertensione arteriosa polmonare
- Peptidi
- Aminoacidi, peptidi e proteine
- Proteine
- Preparati farmaceutici
- Soluzioni cristalloidi
- Soluzioni isotoniche
- Soluzioni
- Proteine Inibitorie della Proteinasi, Secretorie
- Elafin
- Soluzione salina
Altri numeri di identificazione dello studio
Altri numeri di identificazione dello studio
- 84755
- UG3HL180990 (Sovvenzione/contratto NIH degli Stati Uniti)
- U24HL180994 (Sovvenzione/contratto NIH degli Stati Uniti)
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Informazioni su farmaci e dispositivi, documenti di studio
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