High importance
Sep 8, 2026
To evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of DIAG723 in adult patients with hereditary hemorrhagic telangiectasia (HHT).
The study consists of three parts: Part A involves dose escalation with single ascending doses of DIAG723; Part B assesses multiple doses over 13 weeks in HHT patients; Part C evaluates multiple doses in HHT patients with concomitant pulmonary arterial hypertension.
Preliminary results will assess the safety and tolerability of DIAG723, along with initial efficacy signals in the HHT population and those with associated pulmonary conditions.
As a first-in-human study, limitations include a small sample size, a short duration of treatment evaluation, and the inherent uncertainties in safety and efficacy observed in early-phase trials.
This study could provide critical insights into a novel therapeutic approach for HHT, which currently lacks effective treatment options, potentially improving management and outcomes for affected individuals.
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.