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

ConditionsHereditary Hemorrhagic Telangiectasia (HHT)Osler Weber Rendu Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorAtavistik Bio, Inc

About this trial

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.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

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

Disqualifiers

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)

Trial design

Design model

Parallel

Treatments tested in this trial

  • ATV-1601

    Drug

    Administered orally, daily

  • Placebo

    Drug

    Administered orally, daily

Treatment groups

100 Participants
are divided into 4 treatment groups
Group A: Part 1: 60 mg QDExperimental treatment 1 intervention
Group B: Part 1: 100 mg QDExperimental treatment 1 intervention
Group C: Part 1: 60 BIDExperimental treatment 1 intervention
Group D: Part 1: PlaceboExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Part 1: Safety and tolerability

Number and severity of treatment-emergent adverse events (TEAEs) and study drug-related TEAEs

Time frame
16 weeks
2

Part 2: Safety and tolerability

Type, incidence, severity, timing, seriousness and relatedness of AEs and laboratory abnormalities

Time frame
24 months

Secondary outcomes

1

Part 1: Change in Epistaxis duration

28-day total duration compared to baseline

Time frame
16 weeks
2

Part 1: Epistaxis frequency

28-day frequency of nosebleeds compared to baseline

Time frame
16 weeks
3

Part 1: Epistaxis intensity

28-day average epistaxis intensity (6-point scale) of nosebleeds compared to baseline

Time frame
16 weeks
4

Part 1: Intensity-weighted epistaxis duration

28-day intensity-weighted duration of nosebleeds

Time frame
16 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Atavistik Bio, Inc

Lead sponsor

Source ClinicalTrials.gov
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