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Withdrawal of Prostacyclin Pathway Therapy in Patients With Pulmonary Arterial Hypertension Receiving Sotatercept (WATERLOO) (WATERLOO)

13. juli 2026 oppdatert av: University of Alberta

Withdrawal of Background Prostacyclin Pathway Therapy in Patients With Pulmonary Arterial Hypertension Receiving Sotatercept: an Open Label Non-inferiority Trial

Pulmonary arterial hypertension (PAH) is a rare lung disease that leads to elevated blood pressure in the lungs and strain on the right side of the heart. For many years, treatments for PAH have included drugs that target the prostacyclin pathway using intravenous, subcutaneous, oral, and inhaled drugs. These drugs help widen the blood vessels in the lungs so the heart does not have to work as hard. However, these medicines can cause side effects such as jaw pain, flushing, diarrhea, and nausea, and the pump therapy can be very hard to manage day-to-day.

A newer medicine called sotatercept works in a different way. It helps fix some of the root causes of PAH. Early reports suggest that some people do very well on sotatercept and may not need to keep taking their prostacyclin therapy. However, investigators do not yet know if it is safe to stop prostacyclin therapies or how to do so. This study, called WATERLOO, is designed to find out whether slowly stopping prostacyclin therapy while the participant is doing well on sotatercept is safe. Investigators will compare people who stop their prostacyclin therapy to people who keep taking it. This study is being done at PAH expert centres in Canada and Europe.

Studieoversikt

Status

Har ikke rekruttert ennå

Forhold

Intervensjon / Behandling

Studietype

Intervensjonell

Registrering (Antatt)

78

Fase

  • Ikke aktuelt

Kontakter og plasseringer

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

Studiekontakt

  • Navn: Canadian VIGOUR Centre Clinical Trial Project Lead
  • Telefonnummer: 1-800-707-9098
  • E-post: waterloo@ualberta.ca

Studiesteder

  • Canada
    • Alberta
      • Edmonton, Alberta, Canada
        • University of Alberta Hospital
        • Ta kontakt med:

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: In order to be eligible for this study, a participant must meet all of the following criteria:

  1. Adults ≥ 18 years old diagnosed with PAH.
  2. Treatment with sotatercept for ≥6 months.
  3. Background therapy with ≥2 PAH vasodilator therapies, one of which is a parenteral prostacyclin or selexipag.
  4. At low or intermediate-low risk, defined using the 2022 ESC/ERS guidelines 4-strata risk assessment tool (Table 1).
  5. RHC at screening or historical within 8 weeks of screening, and after at least 6 months of sotatercept treatment, demonstrating a mPAP ≤40 mmHg and PVR ≤5 WU
  6. The ability to adhere to the study visit schedule and to comprehend and comply with all protocol requirements.
  7. Ability to provide informed consent.

Exclusion Criteria: A potential participant who meets any of the following criteria will be excluded from participation in this study:

  1. Known intolerance to sotatercept
  2. A RHC at screening or historical within 8 weeks of screening and after ≥6 months of sotatercept treatment demonstrating mPAP > 40 mmHg or PVR > 5 WU.
  3. Hospitalization for worsening PAH or right heart failure within the 3 months prior to screening.
  4. Active listing for lung or heart/lung transplantation.
  5. Metastatic cancer or any other condition with a life expectancy < 6 months,
  6. Female patients who are pregnant or who are of childbearing age and are unwilling to use contraception during the study.
  7. History of ≥ 3 interruptions or missed doses of sotatercept for any reason within the previous 6 months prior to screening.
  8. Patients who received any investigational medication within 1 month prior to screening (unless known to be placebo) or who are scheduled to receive another investigational drug during the course of this study.

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: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Ingen inngripen: Control group / no change to therapy
The control group will continue prostacyclin pathway therapies as per their authorized indications, with no change in dosage.
Eksperimentell: Prostacyclin pathway therapy withdrawal
Dose de-escalation and discontinuation of parenteral prostacyclins analogues or selexipag
Fremgangsmåte: Discontinuation of parenteral prostacyclins analogues or selexipag
Discontinuation of parenteral prostacyclins analogues or selexipag

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Change in Mean Pulmonary Arterial Pressure (mPAP) From Baseline to 24 Weeks
Tidsramme: Baseline to 24 weeks
Difference between the prostacyclin pathway therapy withdrawal group and the continuation group in the change in mean pulmonary arterial pressure (mPAP), measured in mmHg, from baseline to Week 24 by right heart catheterization.
Baseline to 24 weeks

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Time to First Occurrence of All-Cause Death or Clinical Worsening
Tidsramme: Baseline to 24 weeks
Comparison between the prostacyclin pathway therapy withdrawal group and the continuation group in the time to first occurrence of the composite endpoint of all-cause death or clinical worsening. Clinical worsening is defined as any of the following: hospitalization for worsening PAH, decline in 6-minute walk distance (6MWD) ≥10% from baseline on two consecutive tests at least 4 hours apart accompanied by worsening WHO functional class, or worsening ESC/ERS 4-strata risk status.
Baseline to 24 weeks
Change in EmPHasis-10 Score From Baseline to Week 24
Tidsramme: Baseline to 24 weeks

Difference between the prostacyclin pathway therapy withdrawal group and the continuation group in the change from baseline to Week 24

Unit of Measure: Score Range: 0-50 Higher scores indicate worse pulmonary hypertension-related quality of life.
Baseline to 24 weeks
Change in EQ-5D-5L Index Score From Baseline to Week 24
Tidsramme: Baseline to Week 24

Difference between the prostacyclin pathway therapy withdrawal group and the continuation group in the change from baseline to Week 24 in the EQ-5D-5L Index Score.

Unit of Measure: Index score Country-specific value set; Higher scores indicate better health status.
Baseline to Week 24
Change in EQ Visual Analogue Scale (EQ VAS) Score From Baseline to Week 24
Tidsramme: Baseline to Week 24

Difference between the prostacyclin pathway therapy withdrawal group and the continuation group in the change from baseline to Week 24 in the EQ-5D-5L Index Score.

Unit of Measure: score Range: 0-100 Higher scores indicate better perceived health.
Baseline to Week 24
Change in Living with Medicines Questionnaire Version 3 (LMQ-3) Score From Baseline to Week 24
Tidsramme: Baseline to Week 24

Difference between the prostacyclin pathway therapy withdrawal group and the continuation group in the change from baseline to Week 24 in the EQ-5D-5L Index Score.

Unit of Measure: Score Range: 41-205 Higher scores indicate a greater medication burden.
Baseline to Week 24
Number of Participants Reporting Prostanoid-Associated Side Effects
Tidsramme: Baseline to 24 weeks
Difference between the prostacyclin pathway therapy withdrawal group and the continuation group in the number of participants reporting prostanoid-associated side effects, including jaw pain, flushing, nausea, diarrhea, and myalgia.
Baseline to 24 weeks

Andre resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Number of Participants Experiencing Adverse Events (AEs)
Tidsramme: Baseline to 24, 52 and 104 (End of Study) weeks
Difference between the prostacyclin pathway therapy withdrawal group and the continuation group in the number of participants experiencing one or more adverse events.
Baseline to 24, 52 and 104 (End of Study) weeks
Number of Participants Experiencing Serious Adverse Events (SAEs)
Tidsramme: Baseline to 24, 52 and 104 (End of Study) weeks
Difference between the prostacyclin pathway therapy withdrawal group and the continuation group in the number of participants experiencing one or more serious adverse events.
Baseline to 24, 52 and 104 (End of Study) weeks
Participant Enrollment Rate
Tidsramme: Baseline to 24 weeks
Number of participants enrolled per site per month during the 24-week recruitment period.
Baseline to 24 weeks
Protocol Adherence Rate
Tidsramme: Baseline to Week 24
Proportion of participants who complete study procedures according to protocol through Week 24 without major protocol deviations.
Baseline to Week 24
Completeness of Secondary and Exploratory Outcome Data
Tidsramme: Baseline to Week 24
Proportion of expected secondary and exploratory outcome data points successfully collected through Week 24.
Baseline to Week 24

Samarbeidspartnere og etterforskere

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

Sponsor

University of Alberta

Etterforskere

  • Hovedetterforsker: Dr. Jason Weatherald, MD, MSc, FRCPC, University of Alberta

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

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)

30. september 2026

Primær fullføring (Antatt)

30. mars 2028

Studiet fullført (Antatt)

30. mars 2030

Datoer for studieregistrering

Først innsendt

15. juni 2026

Først innsendt som oppfylte QC-kriteriene

8. juli 2026

Først lagt ut (Faktiske)

14. juli 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

15. juli 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

13. juli 2026

Sist bekreftet

1. juli 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • WATERLOO

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Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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