This is a Phase 1/2, randomized, double-blind, placebo-controlled, first-in-human study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of subcutaneously administered DIAG723 in adult patients with hereditary hemorrhagic telangiectasia (HHT). The study consists of three parts: Part A (dose escalation): Single ascending subcutaneous doses of DIAG723 are evaluated in sequential cohorts to assess safety, tolerability, and pharmacokinetics. Part B (dose expansion): Multiple doses of DIAG723 administered over 13 weeks are evaluated in patients with HHT to assess safety and preliminary efficacy. Part C (dose expansion): Multiple doses of DIAG723 administered over 13 weeks are evaluated in patients with HHT and concomitant pulmonary arterial hypertension to assess safety and exploratory clinical effects in this population. Participants will be randomized within each study part to receive DIAG723 or placebo. The study includes dose escalation in Part A and dose expansion in Parts B and C.
Age range
18 Years
Sex
ALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Incidence of Treatment-Emergent Adverse Events (TEAEs) - Part A (Single Dose)
Timeframe: From first dose through Day 28
Incidence of Serious Adverse Events (SAEs) - Part A (Single Dose)
Timeframe: From first dose through Day 28
Incidence of Dose-Limiting Toxicities (DLTs) - Part A (Single Dose)
Timeframe: From first dose through Day 28
Number of participants with abnormal laboratory tests results - Part A (Single Dose)
Timeframe: Baseline through Day 28
Number of participants with abnormal vital signs - Part A (Single Dose)
Timeframe: Baseline through Day 28
Change from Baseline in Electrocardiogram (ECG) Parameters - Part A (Single Dose)
Timeframe: Baseline through Day 28
Incidence of Treatment-Emergent Adverse Events (TEAEs) - Part B (Multiple Dose)
Timeframe: From first dose through 28 days after final dose
Incidence of Serious Adverse Events (SAEs) - Part B (Multiple Dose)
Timeframe: From first dose through 28 days after final dose
Incidence of Dose-Limiting Toxicities (DLTs) - Part B (Multiple Dose)
Timeframe: From first dose through 28 days after final dose
Number of participants with abnormal laboratory tests results - Part B (Multiple Dose)
Timeframe: Baseline through Week 15
Number of participants with abnormal vital signs - Part B (Multiple Dose)
Timeframe: Baseline through Week 17
Change from Baseline in Electrocardiogram (ECG) Parameters - Part B (Multiple Dose)
Timeframe: Baseline through Week 17
Incidence of Treatment-Emergent Adverse Events (TEAEs) - Part C (Multiple Dose, HHT with PAH)
Timeframe: From first dose through 28 days after final dose
Incidence of Serious Adverse Events (SAEs) - Part C (Multiple Dose, HHT with PAH)
Timeframe: From first dose through 28 days after final dose
Incidence of Dose-Limiting Toxicities (DLTs) - Part C (Multiple Dose, HHT with PAH)
Timeframe: From first dose through 28 days after final dose
Number of participants with abnormal laboratory tests results - Part C (Multiple Dose, HHT with PAH)
Timeframe: Baseline through Week 15
Number of participants with abnormal vital signs - Part C (Multiple Dose, HHT with PAH)
Timeframe: Baseline through Week 17
Change from Baseline in Electrocardiogram (ECG) Parameters - Part C (Multiple Dose, HHT with PAH)
Timeframe: Baseline through Week 17