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

15. maj 2026 opdateret af: 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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Detaljeret 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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

100

Fase

  • Fase 2
  • Fase 1

Kontakter og lokationer

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

Studiekontakt

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

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

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: Firedobbelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Part 1: 60 mg QD
Active drug, once daily
Medicin: ATV-1601
Administered orally, daily
Andre navne:
  • Active drug, capsule
Eksperimentel: Part 1: 100 mg QD
Active drug, once daily
Medicin: ATV-1601
Administered orally, daily
Andre navne:
  • Active drug, capsule
Eksperimentel: Part 1: 60 BID
Active drug, twice daily
Medicin: ATV-1601
Administered orally, daily
Andre navne:
  • Active drug, capsule
Eksperimentel: Part 1: Placebo
Control Arm
Medicin: Placebo
Administered orally, daily
Andre navne:
  • Inactive, Placebo

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
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
Foranstaltningsbeskrivelse
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

Samarbejdspartnere og efterforskere

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

Sponsor

Atavistik Bio, Inc

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)

1. juni 2026

Primær færdiggørelse (Anslået)

1. december 2027

Studieafslutning (Anslået)

1. marts 2030

Datoer for studieregistrering

Først indsendt

12. maj 2026

Først indsendt, der opfyldte QC-kriterier

15. maj 2026

Først opslået (Faktiske)

22. maj 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

22. maj 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

15. maj 2026

Sidst verificeret

1. maj 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

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

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

INGEN

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

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Kliniske forsøg med Arvelig hæmoragisk telangiektasi (HHT)

Kliniske forsøg med ATV-1601

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Betingelser

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Kosttilskud

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