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

8. juli 2026 opdateret af: University of Alberta

Pulmonary arterial hypertension (PAH) is a rare lung disease which 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, we 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 patient is doing well on sotatercept is safe. We 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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

78

Fase

  • Ikke anvendelig

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

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

Studiesteder

  • Canada
    • Alberta
      • Edmonton, Alberta, Canada
        • University of Alberta Hospital
        • Kontakt:

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

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

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

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

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
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
Foranstaltningsbeskrivelse
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
Foranstaltningsbeskrivelse
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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

University of Alberta

Efterforskere

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

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Generelle publikationer

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

30. september 2026

Primær færdiggørelse (Anslået)

30. marts 2028

Studieafslutning (Anslået)

30. marts 2030

Datoer for studieregistrering

Først indsendt

15. juni 2026

Først indsendt, der opfyldte QC-kriterier

8. juli 2026

Først opslået (Faktiske)

14. juli 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

14. juli 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

8. juli 2026

Sidst verificeret

1. juli 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • WATERLOO

Plan for individuelle deltagerdata (IPD)

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