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DIAG723 in Adults With Hereditary Hemorrhagic Telangiectasia (DIAMOND)

2026년 9월 2일 업데이트: Diagonal Therapeutics, Inc.

A Phase 1/2, First-in-Human, Multicenter, Ascending Single-Dose and Multi-Dose Study to Assess the Safety of DIAG723, a Novel Bispecific ALK-1 and BMPRII Agonist Antibody in Adult Patients With Hereditary Hemorrhagic Telangiectasia (DIAMOND 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.

연구 개요

상태

모병

정황

개입 / 치료

상세 설명

This is a Phase 1/2, randomized, double-blind, placebo-controlled, first-in-human, multicenter study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary clinical activity of DIAG723, a bispecific agonist monoclonal antibody targeting activin receptor-like kinase 1 (ALK-1) and bone morphogenetic protein receptor type II (BMPRII), in adult patients with hereditary hemorrhagic telangiectasia (HHT).

The study is conducted in three sequential parts (Parts A, B, and C), each evaluating different dosing strategies and patient populations.

Part A (Single Ascending DoseDose in HHT):

Part A is a dose-escalation phase evaluating ascending single-dose levels of DIAG723 administered subcutaneously in sequential cohorts. Within each cohort, participants are randomized to receive DIAG723 or placebo. Dose escalation proceeds in a stepwise manner following review of safety, tolerability, and pharmacokinetic data. Sentinel dosing is implemented to allow for early safety assessment prior to dosing additional participants. Participants are monitored in an inpatient setting following dosing, with continued outpatient follow-up through the end of the assessment period.

Part B (Multiple-Dose Expansion in HHT):

Part B evaluates the safety, tolerability, pharmacokinetics, and preliminary clinical activity of DIAG723 administered as a multiple-dose regimen in patients with HHT symptoms. Participants are randomized to receive DIAG723 or placebo and receive repeated subcutaneous administrations over a planned treatment period of approximately 13 weeks. Dose levels and regimens evaluated in Part B are informed by available safety, pharmacokinetic, and pharmacodynamic data from Part A. An independent data monitoring process is used to support dose selection and cohort progression.

Part C (Multiple-Dose Expansion in HHT with Pulmonary Arterial Hypertension):

Part C evaluates the safety, tolerability, pharmacokinetics, and clinical effects of DIAG723 in a population of patients with HHT and concomitant pulmonary arterial hypertension. Participants are randomized to receive DIAG723 or placebo and receive the same general multiple-dose treatment approach as in Part B. Dose selection for Part C is informed by cumulative data from Part A and Part B, with additional assessments conducted to characterize effects in this specific patient population.

Across all study parts, participants are randomized using an interactive response system, and study treatment is administered under double-blind conditions. A Safety Review Committee and an independent Data Safety Monitoring Board provide ongoing review of safety data and support dose-escalation and progression decisions throughout the study. Dose escalation and cohort progression are guided by predefined safety criteria and overall risk-benefit assessment.

All doses are administered by subcutaneous injection, and participants undergo safety monitoring, pharmacokinetic sampling, and clinical assessments at scheduled study visits. The study includes both inpatient and outpatient components depending on the study part and stage of participation.

This study is designed to characterize the safety profile and pharmacokinetic properties of DIAG723 and to support selection of appropriate doses and regimens for further clinical development in HHT.

연구 유형

중재적

등록 (추정된)

93

단계

  • 2 단계
  • 1단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

연구 장소

  • 뉴질랜드
    • Auckland
      • Grafton, Auckland, 뉴질랜드, 1010
        • 모병
        • Diagonal Investigational Site
  • 캐나다
    • Ontario
      • Toronto, Ontario, 캐나다, M5B1W8
        • 아직 모집하지 않음
        • Diagonal Investigational Site
      • Toronto, Ontario, 캐나다, M9L 3A2
        • 아직 모집하지 않음
        • Diagonal Investigational Site
    • Quebec
      • Laval, Quebec, 캐나다, H7V 4B3
        • 아직 모집하지 않음
        • Diagonal Investigational Site
  • 호주
    • New South Wales
      • Camperdown, New South Wales, 호주, 2050
        • 아직 모집하지 않음
        • Diagonal Investigational Site
      • Randwick, New South Wales, 호주, 2031
        • 모병
        • Diagonal Investigational Site
    • Queensland
      • Herston, Queensland, 호주, 4029
        • 아직 모집하지 않음
        • Diagonal Investigational Site
      • Herston, Queensland, 호주, 4029
        • 빼는
        • Diagonal Investigational Site
    • Victoria
      • East Melbourne, Victoria, 호주, 3002
        • 모병
        • Diagonal Investigational Site
      • Parkville, Victoria, 호주, 3052
        • 아직 모집하지 않음
        • Diagonal Investigational Site
    • Western Australia
      • West Perth, Western Australia, 호주, 6005
        • 아직 모집하지 않음
        • Diagonal Investigational Site

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

  • 성인
  • 고령자

건강한 자원 봉사자를 받아들입니다

아니

설명

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • Active or recent systemic infection
  • Recent thromboembolic events
  • Use of anti-angiogenic drugs within 6 weeks
  • Pregnancy or lactation
  • Recent participation in another investigational study

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 무작위
  • 중재 모델: 병렬 할당
  • 마스킹: 삼루타

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: DIAG723

Participants receive DIAG723 administered subcutaneously.

Part A (dose-escalation): Single ascending dose across planned cohorts, randomized 3:1 DIAG723:placebo.

Part B (multi-dose HHT): Multiple-dose regimens over 13 weeks (7 doses administered every other week), randomized 2:1.

Part C (multi-dose HHT + PAH): Same 13-week multi-dose regimen in patients with pulmonary arterial hypertension, randomized 4:1.

Doses/regimens in Parts B and C are selected based on Part A safety and PK data.
생물학적: DIAG723

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
위약 비교기: Placebo Comparator

Participants receive placebo (sterile normal saline, 0.9% NaCl) administered subcutaneously in a volume matched to DIAG723 to maintain blinding.

Matching single-dose administration in Part A. Matching multi-dose regimens (every-other-week dosing for 13 weeks) in Parts B and C.

Randomization ratios consistent with each study part (3:1, 2:1, 4:1).
다른: Placebo
Sterile normal saline (0.9% NaCl) administered subcutaneously in volumes matched to DIAG723 to maintain study blinding.

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Incidence of Treatment-Emergent Adverse Events (TEAEs) - Part A (Single Dose)
기간: From first dose through Day 28
Number and proportion of participants experiencing TEAEs following single-dose administration of DIAG723.
From first dose through Day 28
Incidence of Serious Adverse Events (SAEs) - Part A (Single Dose)
기간: From first dose through Day 28
Number and proportion of participants experiencing SAEs following single-dose administration of DIAG723.
From first dose through Day 28
Incidence of Dose-Limiting Toxicities (DLTs) - Part A (Single Dose)
기간: From first dose through Day 28
Number and proportion of participants experiencing DLTs during the dose-escalation period following single-dose administration.
From first dose through Day 28
Number of participants with abnormal laboratory tests results - Part A (Single Dose)
기간: Baseline through Day 28
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.
Baseline through Day 28
Number of participants with abnormal vital signs - Part A (Single Dose)
기간: Baseline through Day 28
Vital sign measurements include body temperature, heart rate, respiratory rate, blood pressure, and oxygen saturation.
Baseline through Day 28
Change from Baseline in Electrocardiogram (ECG) Parameters - Part A (Single Dose)
기간: Baseline through Day 28
Change from baseline in ECG parameters, including QTc interval.
Baseline through Day 28
Incidence of Treatment-Emergent Adverse Events (TEAEs) - Part B (Multiple Dose)
기간: From first dose through 28 days after final dose
Number and proportion of participants experiencing TEAEs following multiple-dose administration of DIAG723.
From first dose through 28 days after final dose
Incidence of Serious Adverse Events (SAEs) - Part B (Multiple Dose)
기간: From first dose through 28 days after final dose
Number and proportion of participants experiencing SAEs following multiple-dose administration.
From first dose through 28 days after final dose
Incidence of Dose-Limiting Toxicities (DLTs) - Part B (Multiple Dose)
기간: From first dose through 28 days after final dose
Number and proportion of participants experiencing DLTs during multiple-dose treatment.
From first dose through 28 days after final dose
Number of participants with abnormal laboratory tests results - Part B (Multiple Dose)
기간: Baseline through Week 15
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.
Baseline through Week 15
Number of participants with abnormal vital signs - Part B (Multiple Dose)
기간: Baseline through Week 17
Vital sign measurements include body temperature, heart rate, respiratory rate, blood pressure, and oxygen saturation.
Baseline through Week 17
Change from Baseline in Electrocardiogram (ECG) Parameters - Part B (Multiple Dose)
기간: Baseline through Week 17
Change from baseline in ECG parameters, including QTc interval.
Baseline through Week 17
Incidence of Treatment-Emergent Adverse Events (TEAEs) - Part C (Multiple Dose, HHT with PAH)
기간: From first dose through 28 days after final dose
Number and proportion of participants experiencing TEAEs following multiple-dose administration in participants with HHT and pulmonary arterial hypertension.
From first dose through 28 days after final dose
Incidence of Serious Adverse Events (SAEs) - Part C (Multiple Dose, HHT with PAH)
기간: From first dose through 28 days after final dose
Number and proportion of participants experiencing SAEs in this population.
From first dose through 28 days after final dose
Incidence of Dose-Limiting Toxicities (DLTs) - Part C (Multiple Dose, HHT with PAH)
기간: From first dose through 28 days after final dose
Number and proportion of participants experiencing DLTs during treatment.
From first dose through 28 days after final dose
Number of participants with abnormal laboratory tests results - Part C (Multiple Dose, HHT with PAH)
기간: Baseline through Week 15
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.
Baseline through Week 15
Number of participants with abnormal vital signs - Part C (Multiple Dose, HHT with PAH)
기간: Baseline through Week 17
Vital sign measurements include body temperature, heart rate, respiratory rate, blood pressure, and oxygen saturation.
Baseline through Week 17
Change from Baseline in Electrocardiogram (ECG) Parameters - Part C (Multiple Dose, HHT with PAH)
기간: Baseline through Week 17
Change from baseline in ECG parameters, including QTc interval.
Baseline through Week 17

2차 결과 측정

결과 측정
측정값 설명
기간
Area Under the Plasma Concentration-Time Curve (AUC) of DIAG723 - Part A
기간: Pre-dose through Day 28
Plasma AUC following single-dose administration of DIAG723.
Pre-dose through Day 28
Maximum Observed Plasma Concentration (Cmax) of DIAG723 - Part A
기간: Pre-dose through Day 28
Maximum observed plasma concentration following single-dose administration.
Pre-dose through Day 28
Time to Maximum Plasma Concentration (Tmax) of DIAG723 - Part A
기간: Pre-dose through Day 28
Time to reach maximum plasma concentration following single-dose administration.
Pre-dose through Day 28
Area Under the Plasma Concentration-Time Curve (AUC) of DIAG723 - Part B (Multiple Dose)
기간: Pre-dose through 28 days after final dose
Plasma AUC following repeated dosing of DIAG723.
Pre-dose through 28 days after final dose
Maximum Observed Plasma Concentration (Cmax) of DIAG723 - Part B (Multiple Dose)
기간: Pre-dose through 28 days after final dose
Maximum observed plasma concentration following multiple-dose administration.
Pre-dose through 28 days after final dose
Time to Maximum Plasma Concentration (Tmax) of DIAG723 - Part B (Multiple Dose)
기간: Pre-dose through 28 days after final dose
Time to reach maximum plasma concentration following multiple-dose administration.
Pre-dose through 28 days after final dose
Incidence of Anti-Drug Antibodies (ADA) to DIAG723 - Part A (Single Dose)
기간: From first dose through Day 28
Number and proportion of participants with detectable anti-drug antibodies following single-dose administration.
From first dose through Day 28
Incidence of Anti-Drug Antibodies (ADA) to DIAG723 - Part B (Multiple Dose)
기간: From first dose through 28 days after final dose
Number and proportion of participants with detectable anti-drug antibodies following multiple-dose administration.
From first dose through 28 days after final dose
Change from Baseline in Epistaxis Frequency - Part B (Multiple Dose)
기간: Baseline through end of treatment (13 weeks)
Change from baseline in frequency of epistaxis events as recorded in a participant-reported electronic daily diary.
Baseline through end of treatment (13 weeks)
Change from Baseline in Epistaxis Flow Intensity, Duration, and Intensity-Adjusted Duration - Part B (Multiple Dose)
기간: Baseline through end of treatment (13 weeks)
Change from baseline in epistaxis flow intensity, event duration, and intensity-adjusted duration as recorded in an electronic daily diary.
Baseline through end of treatment (13 weeks)
Change from Baseline in Hemoglobin - Part B (Multiple Dose)
기간: Baseline through Week 15
Change from baseline in hemoglobin concentration.
Baseline through Week 15
Change from Baseline in Hematocrit - Part B (Multiple Dose)
기간: Baseline through Week 15
Change from baseline in hematocrit.
Baseline through Week 15
Change in Mean Corpuscular Volume (MCV) Following Multi-dose Treatment With DIAG723 (Part B)
기간: Baseline through Week 15
Change from baseline in mean corpuscular volume (MCV, fL) during Part B following multi-dose treatment with DIAG723.
Baseline through Week 15
Change from Baseline in Red Blood Cell Count Following Multi-dose Treatment With DIAG723
기간: Baseline through Week 15
Baseline through Week 15
Change from Baseline in White Blood Cell Count Following Multi-dose Treatment With DIAG723 (Part B)
기간: Baseline through Week 15
Baseline through Week 15
Change from Baseline in Platelet Count Following Multi-dose Treatment With DIAG723 (Part B)
기간: Baseline through Week 15
Baseline through Week 15
Change in Red Blood Cell (RBC) Units Transfused During Treatment With DIAG723 (Part B)
기간: Baseline through Week 15
Change from baseline in the number of RBC units transfused, comparing a pre-treatment baseline period to the on-treatment period during Part B.
Baseline through Week 15
Change in Elemental Iron Infused During Treatment With DIAG723 (Part B)
기간: Baseline through Week 15
Change from baseline in total milligrams of elemental iron administered via infusion, comparing a pre-treatment baseline period to the on-treatment period during Part B.
Baseline through Week 15
Change in Hematologic Support Score (HSS) Following Treatment With DIAG723 (Part B)
기간: Baseline through Week 17
The Hematologic Support Score ranges from 0 to 100, with higher scores indicating greater transfusion and iron supplementation burden and worse outcomes.
Baseline through Week 17
Change in Hematologic Impact Score (HIS) Following Treatment With DIAG723 (Part B)
기간: Baseline through Week 17
The Hematologic Impact Score ranges from 0 to 100, with higher scores indicating greater hematologic disease burden and worse outcomes.
Baseline through Week 17

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Diagonal Therapeutics, Inc.

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2026년 6월 4일

기본 완료 (추정된)

2027년 11월 30일

연구 완료 (추정된)

2027년 12월 31일

연구 등록 날짜

최초 제출

2026년 5월 19일

QC 기준을 충족하는 최초 제출

2026년 5월 28일

처음 게시됨 (실제)

2026년 6월 3일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 9월 8일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 9월 2일

마지막으로 확인됨

2026년 9월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

  • DIAG723-PT-CL-001

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

아니요

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

아니

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

구독하다