Health TrialsbyHealthPanel

DIAG723 in Adults With Hereditary Hemorrhagic Telangiectasia

ConditionsHereditary Hemorrhagic TelangiectasiaPulmonary Arterial Hypertension
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorDiagonal Therapeutics, Inc.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Adult patients ≥18 years with a clinical or genetic diagnosis of HHT

Adequate hepatic and renal function

Part B: Epistaxis and anemia or transfusion/iron history

Part C: HHT with documented pre-capillary pulmonary arterial hypertension

Disqualifiers

Active or recent systemic infection

Recent thromboembolic events

Use of anti-angiogenic drugs within 6 weeks

Pregnancy or lactation

Trial design

Design model

Parallel

Treatments tested in this trial

  • DIAG723

    Biological/Vaccine

    Bispecific agonist monoclonal antibody targeting ALK-1 and BMPRII, administered subcutaneously as: Single ascending dose in Part A; Multiple doses (7 doses over 13 weeks) in Parts B and C

  • Placebo

    Other intervention

    Sterile normal saline (0.9% NaCl) administered subcutaneously in volumes matched to DIAG723 to maintain study blinding.

Treatment groups

93 Participants
are divided into 2 treatment groups
Group A: DIAG723Experimental treatment 1 intervention
Group B: Placebo ComparatorPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of Treatment-Emergent Adverse Events (TEAEs) - Part A (Single Dose)

Number and proportion of participants experiencing TEAEs following single-dose administration of DIAG723.

Time frame
From first dose through Day 28
2

Incidence of Serious Adverse Events (SAEs) - Part A (Single Dose)

Number and proportion of participants experiencing SAEs following single-dose administration of DIAG723.

Time frame
From first dose through Day 28
3

Incidence of Dose-Limiting Toxicities (DLTs) - Part A (Single Dose)

Number and proportion of participants experiencing DLTs during the dose-escalation period following single-dose administration.

Time frame
From first dose through Day 28
4

Number of participants with abnormal laboratory tests results - Part A (Single Dose)

Laboratory assessments include hematology (complete blood count), clinical chemistry (basic metabolic panel; liver function tests; renal function; electrolytes; thyroid-stimulating hormone, free T4, free T3; cortisol; luteinizing hormone; lipase; amylase), coagulation (prothrombin time/international normalized ratio, activated partial thromboplastin time), and urinalysis.

Time frame
Baseline through Day 28

Secondary outcomes

1

Area Under the Plasma Concentration-Time Curve (AUC) of DIAG723 - Part A

Plasma AUC following single-dose administration of DIAG723.

Time frame
Pre-dose through Day 28
2

Maximum Observed Plasma Concentration (Cmax) of DIAG723 - Part A

Maximum observed plasma concentration following single-dose administration.

Time frame
Pre-dose through Day 28
3

Time to Maximum Plasma Concentration (Tmax) of DIAG723 - Part A

Time to reach maximum plasma concentration following single-dose administration.

Time frame
Pre-dose through Day 28
4

Area Under the Plasma Concentration-Time Curve (AUC) of DIAG723 - Part B (Multiple Dose)

Plasma AUC following repeated dosing of DIAG723.

Time frame
Pre-dose through 28 days after final dose

Other outcomes

Sponsors and contacts

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

Diagonal Therapeutics, Inc.

Lead sponsor

Source ClinicalTrials.gov
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