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Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)

28. august 2026 oppdatert av: Atavistik Bio, Inc

A Randomized, Placebo-Controlled, Double-Blind, Proof-of-Concept Study of ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)

This is a 2-part study evaluating ATV-1601 in participants with moderate to severe HHT. Part 1 is a randomized, double-blind, placebo-controlled study evaluating 3 dosing regimens of ATV-1601. Patients completing Part 1 may participate in the Part 2 open-label extension to receive ATV-1601.

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

Part 1: This is a Phase 1/2 proof-of-concept, double-blind, multicenter, placebo-controlled study to evaluate the safety, pharmacokinetics and efficacy of 3 oral dosing regimens of ATV-1601. Participants who meet eligibility requirements will be randomized in a double-blind manner to one of 3 doses of ATV-1601 or placebo. Participants will receive double-blind study treatment for a 16-week period.

Part 2: Eligible participants who complete Part 1 may enroll in an open-label extension study to receive up to 2 years of additional treatment. All participants in the open-label extension will receive ATV-1601. Once the recommended Phase 2 dose (RP2D) is determined based on Part 1, all participants in Part 2 will have the option to switch to the RP2D.

Studietype

Intervensjonell

Registrering (Antatt)

100

Fase

  • Fase 2
  • Fase 1

Kontakter og plasseringer

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

Studiekontakt

Studiesteder

  • Forente stater
    • Arizona
      • Phoenix, Arizona, Forente stater, 85013
        • Rekruttering
        • Arizona Pulmonary Specialists
        • Ta kontakt med:
          • Study Director
    • Massachusetts
      • Boston, Massachusetts, Forente stater, 02114
        • Rekruttering
        • Massachusetts General Hospital
        • Ta kontakt med:
          • Study Director
    • Minnesota
      • Rochester, Minnesota, Forente stater, 55905
        • Rekruttering
        • Mayo Clinic
        • Ta kontakt med:
          • Study Director

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

Ja

Beskrivelse

Inclusion Criteria:

  • Ability to provide informed consent prior to any study-specific procedures
  • Confirmed diagnosis of hereditary hemorrhagic telangiectasia (HHT) based on Curaçao criteria
  • Moderate to severe HHT with an ESS ≥ 4
  • Anemia at Screening and/or requirement for at least 1 red-cell unit (RUE) in the previous 6 months
  • Adequate hematologic, renal, and hepatic function per protocol-defined laboratory criteria
  • Use highly effective contraception during the study and for a protocol-defined period after last dose

Exclusion Criteria:

  • Clinically significant abnormalities of glucose metabolism including diagnosed Type 1 or uncontrolled Type 2 diabetes
  • Chronic cardiac disease, or cardiac rhythm abnormalities
  • History of significant cardiovascular, hepatic, renal, or hematologic disease not related to HHT that may confound study results
  • Use of prohibited concomitant medications within a protocol-defined washout period prior to first dose (including strong CYP modulators and certain herbal supplements)
  • Recent (within 6 weeks) major surgery or local ablative procedures, or procedures on nasal telangiectasias
  • Prior AKT inhibitor
  • Pregnant or breastfeeding women

Additional Criteria for Open-Label Extension:

  • Participants must complete the double-blind treatment period (Part 1)

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: Firemannsrom

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Part 1: 60 mg QD
Active drug, once daily
Legemiddel: ATV-1601
Administered orally, daily
Andre navn:
  • Active drug, capsule
Eksperimentell: Part 1: 100 mg QD
Active drug, once daily
Legemiddel: ATV-1601
Administered orally, daily
Andre navn:
  • Active drug, capsule
Eksperimentell: Part 1: 60 BID
Active drug, twice daily
Legemiddel: ATV-1601
Administered orally, daily
Andre navn:
  • Active drug, capsule
Eksperimentell: Part 1: Placebo
Control Arm
Legemiddel: Placebo
Administered orally, daily
Andre navn:
  • Inactive, Placebo

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Part 1: Safety and tolerability
Tidsramme: 16 weeks
Number and severity of treatment-emergent adverse events (TEAEs) and study drug-related TEAEs
16 weeks
Part 2: Safety and tolerability
Tidsramme: 24 months
Type, incidence, severity, timing, seriousness and relatedness of AEs and laboratory abnormalities
24 months

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Part 1: Change in Epistaxis duration
Tidsramme: 16 weeks
28-day total duration compared to baseline
16 weeks
Part 1: Epistaxis frequency
Tidsramme: 16 weeks
28-day frequency of nosebleeds compared to baseline
16 weeks
Part 1: Epistaxis intensity
Tidsramme: 16 weeks
28-day average epistaxis intensity (6-point scale) of nosebleeds compared to baseline
16 weeks
Part 1: Intensity-weighted epistaxis duration
Tidsramme: 16 weeks
28-day intensity-weighted duration of nosebleeds
16 weeks
Part 1: Epistaxis Severity Score (ESS)
Tidsramme: 16 weeks
The Epistaxis Severity Score (ESS) is a validated 6-question instrument with scores ranging from 0 to 10, where higher scores indicate more severe epistaxis symptoms.
16 weeks
Part 1: Change in Hemoglobin
Tidsramme: 16 weeks
Hemoglobin levels compared to baseline
16 weeks
Part 1: Change in Parenteral iron use
Tidsramme: 16 weeks
Amount of parenteral iron administered compared to 16-weeks prior to treatment initiation
16 weeks
Part 1: Change in Blood transfusion requirements
Tidsramme: 16 Weeks
Amount of packed red blood cell (PRBC) transfusions and rate of transfusion independence compared to 16-weeks prior to treatment initiation
16 Weeks
Part 1: Pharmacokinetics - Maximum observed concentration (Cmax)
Tidsramme: 16 Weeks
Maximum plasma concentration
16 Weeks
Part 1: Pharmacokinetics - Area under the concentration-time curve over the dosing interval (AUCtau)
Tidsramme: 16 Weeks
Systemic exposure of ATV-1601 over the dosing interval
16 Weeks
Part 1: Pharmacokinetics - Area under the concentration-time curve extrapolated to infinity (AUCinf)
Tidsramme: 16 Weeks
Total systemic exposure of ATV-1601 extrapolated to infinite time
16 Weeks
Part 1: Pharmacokinetics - Time to maximum concentration (Tmax)
Tidsramme: 16 Weeks
Time to reach maximum plasma concentration
16 Weeks
Part 1: Pharmacokinetics - minimum concentration (Cmin)
Tidsramme: 16 Weeks
Pre-dose trough plasma concentration
16 Weeks
Part 1: Pharmacokinetics - Half-life (t½)
Tidsramme: 16 Weeks
Time required for plasma concentration to decrease by half
16 Weeks
Part 2: Epistaxis duration
Tidsramme: Up to 2 years
28-day total duration every 4 weeks
Up to 2 years
Part 2: Epistaxis frequency
Tidsramme: Up to 2 years
Total number of nosebleeds every 4 weeks
Up to 2 years
Part 2: Epistaxis Severity Score (ESS)
Tidsramme: At 12 weeks and every 12 weeks thereafter up to study completion
Severity of nosebleeds using a score of 0-10 automatically calculated based on responses to 6 questions.
At 12 weeks and every 12 weeks thereafter up to study completion
Part 2: Change in Hemoglobin
Tidsramme: Monthly during Part 2
Hemoglobin levels compared to baseline
Monthly during Part 2
Part 2: Parenteral iron use
Tidsramme: At 12 weeks and every 12 weeks thereafter up to study completion
Total amount of parenteral iron infused (mg) compared to baseline (12 weeks prior to treatment initiation
At 12 weeks and every 12 weeks thereafter up to study completion
Part 2: Blood transfusion requirements
Tidsramme: At 12 weeks and every 12 weeks thereafter during part 2
Total number of packed red blood cell (PRBC) transfusions (units) compared to baseline
At 12 weeks and every 12 weeks thereafter during part 2
Part 2: Transfusion independence
Tidsramme: At 12 weeks and every 12 weeks thereafter during part 2
Proportion of participants who do not require PRBC transfusions
At 12 weeks and every 12 weeks thereafter during part 2

Samarbeidspartnere og etterforskere

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

Sponsor

Atavistik Bio, Inc

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 (Faktiske)

27. juli 2026

Primær fullføring (Antatt)

1. desember 2027

Studiet fullført (Antatt)

1. mars 2030

Datoer for studieregistrering

Først innsendt

12. 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)

1. september 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

28. august 2026

Sist bekreftet

1. august 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • ATV-1601-102
  • Harmony-HHT (Annen identifikator: Atavistik Bio Inc)

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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