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Dosvarierande studie av oral PHA-022121 för profylax mot angioödem attacker hos patienter med ärftligt angioödem typ I eller typ II (HAE CHAPTER-1)

12 augusti 2026 uppdaterad av: Pharvaris Netherlands B.V.

En fas II, dubbelblind, placebokontrollerad, randomiserad, dosvarierande, parallell gruppstudie för att utvärdera säkerheten och effekten av PHA-022121 administrerat oralt för profylax mot angioödem attacker hos patienter med ärftligt angioödem på grund av brist på C1-hämmare ( Typ I eller Typ II)

Denna studie utvärderar säkerheten och effekten av PHA-022121 administrerat oralt för profylax mot angioödem attacker hos patienter med ärftligt angioödem (HAE). Studien består av 2 delar, där patienter slutför deltagande i del 1 innan behandlingen påbörjas i del 2. Del 1 av studien har 3 parallella armar och cirka 30 patienter kommer att randomiseras lika till en av två dosregimer av PHA-022121 eller matchande placebo. Patienterna kommer att fortsätta till den enda öppna armen i del 2 av studien efter att del 1 har slutförts. Screeningsperioden är upp till 8 veckor och behandlingsperioderna är 12 veckor (del 1) och 50 veckor (del 2).

Studieöversikt

Status

Avslutad

Betingelser

Intervention / Behandling

Studietyp

Interventionell

Inskrivning (Faktisk)

34

Fas

  • Fas 2

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studieorter

  • Bulgarien
      • Sofia, Bulgarien, 1680
        • Study Site
  • Förenta staterna
    • Alabama
      • Birmingham, Alabama, Förenta staterna, 35209
        • Study Site
    • Arizona
      • Paradise Valley, Arizona, Förenta staterna, 85253
        • Study Site
    • Missouri
      • St Louis, Missouri, Förenta staterna, 63141
        • Study Site
  • Irland
      • Dublin, Irland, D08NHY1
        • Study Site
  • Italien
      • Milan, Italien, 20157
        • Study Site
      • Palermo, Italien, 0146
        • Study Site
    • PD
      • Padua, PD, Italien, 35128
        • Study Site
  • Kanada
    • Ontario
      • Ottawa, Ontario, Kanada, K1H 1E4
        • Study Site
    • Quebec
      • Montreal, Quebec, Kanada, H2W 1R7
        • Study Site
  • Polen
      • Krakow, Polen
        • Study Site
  • Storbritannien
    • England
      • Brighton, England, Storbritannien, BN2 1ES
        • Study Site
      • Bristol, England, Storbritannien, BS10 5NB
        • Study Site
      • Cambridge, England, Storbritannien, CB2 0QQ
        • Study Site
      • London, England, Storbritannien, E1 1FR
        • Study Site
      • Southampton, England, Storbritannien, SO16 6YD
        • Study Site
  • Tyskland
      • Berlin, Tyskland, 10117
        • Study Site
      • Frankfurt, Tyskland, 60323
        • Study Site
  • Österrike
      • Vienna, Österrike, A-1090
        • Study Site

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

18 år till 75 år (Vuxen, Äldre vuxen)

Tar emot friska volontärer

Nej

Beskrivning

Inklusionskriterier:

  • Undertecknad och daterad blankett för informerat samtycke
  • Diagnos av HAE typ I eller II
  • Dokumenterad historia av minst 3 HAE-attacker under de senaste 3 månaderna i följd före screening, eller minst 2 HAE-attacker under screeningsperioden
  • Tillförlitlig tillgång och erfarenhet för att använda standardmediciner för akuta attacker

Exklusions kriterier:

  • Graviditet eller amning
  • Kliniskt signifikant onormalt elektrokardiogram
  • Varje annan systemisk sjukdom eller betydande sjukdom eller störning som skulle störa patientens säkerhet eller förmåga att delta i studien
  • Användning av C1-esterashämmare, orala kallikreinhämmare, försvagade androgener, antifibrinolytika eller monoklonal HAE-terapi inom en definierad period före inskrivning
  • Onormal leverfunktion
  • Onormal njurfunktion
  • Historik av alkohol- eller drogmissbruk inom en definierad period, eller aktuella bevis på drogberoende eller missbruk
  • Deltagande i någon annan läkemedelsstudie inom en definierad period

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Förebyggande
  • Tilldelning: Randomiserad
  • Interventionsmodell: Parallellt uppdrag
  • Maskning: Fyrdubbla

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Placebo-jämförare: Del 1: Placebo
BID placebo
Läkemedel: Placebo
Matchande placebokapslar för oral användning
Experimentell: Del 1: Låg dos
BID låg dos av deucrictibant
Läkemedel: Deucrictibant låg dos
Deucrictibant softgel kapslar för oral användning (PHVS416)
Experimentell: Del 1: Hög dos
BID hög dos av deucrictibant
Läkemedel: Deucrictibant hög dos
Deucrictibant softgel kapslar för oral användning (PHVS416)
Experimentell: Del 2: Open-label
BID hög dos av deucrictibant
Läkemedel: Deucrictibant hög dos
Deucrictibant softgel kapslar för oral användning (PHVS416)

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Number of Investigator-confirmed Hereditary Angioedema (HAE) Attacks (Expressed as the Normalized Number of Attacks Per Month [4 Weeks] of Exposure) During the Treatment Period in Part 1
Tidsram: Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily electronic patient-reported outcome (ePRO) diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days, multiplied by 28. Mean rate ratio, confidence interval (CI), and p-value were estimated using a generalized linear model with Poisson distribution, with baseline attack rate included as a factor.
Up to Day 84

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Number of Investigator-confirmed Moderate or Severe HAE Attacks During the Treatment Period in Part 1
Tidsram: Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days×28. Moderate HAE attack defined as which limits/interferes with the participant's ability to attend work/school or participate in family life and social/recreational activities whereas Severe HAE attack significantly limits the participant's ability.
Up to Day 84
Number of Investigator-confirmed HAE Attacks Requiring Acute Treatment During the Treatment Period in Part 1
Tidsram: Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days, multiplied by 28.
Up to Day 84
Number of Participants Achieving ≥50%, ≥70% and ≥90% Reduction in Attack Rate Relative to Baseline During the Treatment Period in Part 1
Tidsram: Up to Day 84
Baseline attack rate was defined as each participant's normalized HAE attack rate (attacks per 4 weeks) prior to randomization, based on documented history or confirmed during screening. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days, multiplied by 28. Percent reduction from baseline was calculated for each participant by comparing the normalized treatment-period attack rate to the baseline attack rate. Participants were classified as responders if they achieved ≥50%, ≥70%, or ≥90% reduction in attack rate relative to baseline during Part 1.
Up to Day 84
Number of Participants Who Were Investigator-confirmed Attack-free During the Treatment Period in Part 1
Tidsram: Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Participants reporting no confirmed attacks over the treatment period were considered attack-free.
Up to Day 84
Number of Days With Investigator-confirmed HAE Symptoms During the Treatment Period in Part 1
Tidsram: Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). HAE symptom days were identified from daily participant entries in the ePRO diary and were confirmed by the investigator.
Up to Day 84
Proportion of Days With Investigator-confirmed HAE Symptoms During the Treatment Period in Part 1
Tidsram: Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). HAE symptom days were identified from daily participant entries in the ePRO diary and were confirmed by the investigator.
Up to Day 84
Time to First Investigator-confirmed HAE Attack in the Treatment Period in Part 1
Tidsram: Up to Day 84
Time to first investigator-confirmed HAE attack was defined as days from date of first administration of study drug until the onset date of first Investigator-confirmed HAE attack. Participants without an Investigator-confirmed HAE attack during the treatment period in Part 1 were censored at the date of the last dose of study drug +1 in Part 1 or last day of the study, whichever came sooner.
Up to Day 84
Number of Investigator-confirmed HAE Attacks Resulting in a Visit to the Emergency Department or an Admission to Hospital During the Treatment Period in Part 1
Tidsram: Up to Day 84
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days, multiplied by 28. Only Investigator-confirmed attacks resulting in emergency department visits or hospital admissions were included.
Up to Day 84
Number of Investigator-confirmed HAE Attacks During the Treatment Period in Part 2
Tidsram: Day 84 to Day 938
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days, multiplied by 28. An attack will be counted as in Part 2 if it occurs after the first IMP administration in Part 2 and within 24 hours after last IMP administration in Part 2.
Day 84 to Day 938
Number of Investigator-confirmed Moderate or Severe HAE Attacks During the Treatment Period in Part 2
Tidsram: Day 84 to Day 938
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days×28. Moderate HAE attack defined as which limits/interferes with the participant's ability to attend work/school or participate in family life and social/recreational activities whereas Severe HAE attack significantly limits the participant's ability. LSM and SE were estimated using a generalized linear model with Poisson distribution.
Day 84 to Day 938
Number of Investigator-confirmed HAE Attacks Requiring Acute Treatment During the Treatment Period in Part 2
Tidsram: Day 84 to Day 938
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). A new attack was counted if symptoms occurred ≥24 hours after complete resolution of a prior attack. Attacks were recorded via daily ePRO diaries and confirmed by the investigator. Monthly attack rate was calculated as number of attacks during treatment divided by treatment days, multiplied by 28. LSM and SE were estimated using a generalized linear model with Poisson distribution.
Day 84 to Day 938
Number of Days With Investigator-confirmed HAE Symptoms During the Treatment Period in Part 2
Tidsram: Day 84 to Day 938
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). HAE symptom days were identified from daily participant entries in the ePRO diary and were confirmed by the investigator.
Day 84 to Day 938
Proportion of Days With Investigator-confirmed HAE Symptoms During the Treatment Period in Part 2
Tidsram: Day 84 to Day 938
HAE attack was defined as signs or symptoms in ≥1 location: peripheral angioedema (cutaneous swelling involving an extremity, face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain with or without abdominal distension, nausea, vomiting, or diarrhea), or upper airway angioedema (lump sensation, difficulty swallowing, throat tightening, difficulty speaking, hoarseness, stridor, dyspnea, or swelling of tongue, palate, uvula, or larynx). HAE symptom days were identified from daily participant entries in the ePRO diary and were confirmed by the investigator.
Day 84 to Day 938

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Pharvaris Netherlands B.V.

Utredare

  • Huvudutredare: Marc Riedl, MD, UC San Diego, La Jolla, California, United States
  • Huvudutredare: Emel Aygören-Pürsün, MD, University Hospital Frankfurt - Goethe University, Frankfurt, Germany

Publikationer och användbara länkar

Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.

Allmänna publikationer

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

19 april 2022

Primärt slutförande (Faktisk)

30 juni 2025

Avslutad studie (Faktisk)

30 juni 2025

Studieregistreringsdatum

Först inskickad

7 september 2021

Först inskickad som uppfyllde QC-kriterierna

7 september 2021

Första postat (Faktisk)

17 september 2021

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

17 augusti 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

12 augusti 2026

Senast verifierad

1 augusti 2026

Mer information

Termer relaterade till denna studie

Nyckelord

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • PHA022121-C301
  • 2021-000227-13 (EudraCT-nummer)

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

NEJ

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Ja

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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