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경구투여 VX-01의 유효성 및 안전성을 평가하기 위한 연구

2026년 9월 28일 업데이트: Vantage Biosciences Ltd

비증식성 당뇨병성 망막병증(NPDR)에서 경구 투여되는 VX-01의 효능과 안전성을 평가하기 위한 제2상, 이중 마스크, 무작위, 위약 대조, 병렬 설계 연구

The goal of this clinical trial is to evaluate the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of VX-01 as stand-alone treatment for Diabetic Retinopathy of Non-Proliferative Type (NPDR).

The primary objective of the study is to evaluate the efficacy of daily oral doses of VX-01 versus placebo following 52 weeks of treatment.

연구 개요

상태

모집하지 않고 적극적으로

정황

개입 / 치료

상세 설명

This is a Phase 2, multi-center, double-masked, randomized, placebo-controlled, parallel group study to evaluate the efficacy of oral doses of VX-01 in subjects with moderate to severe NPDR, without CI-DME.

Approximately 100 male and female subjects aged ≥ 18 years with a documented diagnosis of Type 1 Diabetic Mellitus or Type 2 Diabetic Mellitus with moderate to severe NPDR (without CI-DME) will be enrolled, if they meet all the eligibility criteria for the study.

Subjects will be randomized 1:1 to 1 of 2 study cohorts:

  • Cohort 1 (n = 50): VX-01 (film-coated tablets, 150 mg administered BID)
  • Cohort 2 (n = 50): Placebo (film-coated tablets, administered BID)

Subjects will be stratified by the presence or absence of proliferative diabetic retinopathy (PDR) and by glycated hemoglobin (HbA1c) of ≥ 8.5% or < 8.5% at Screening. All subjects will take 1 tablet of VX-01 or placebo BID for 52 consecutive weeks. All subjects will be followed for 12 weeks after completion of treatment at Week 52.

The Sponsor, study site staff, monitors, personnel, and subjects will be masked to treatment assignment during the entirety of the study.

연구 유형

중재적

등록 (추정된)

100

단계

  • 2 단계

연락처 및 위치

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

연구 장소

  • 대한민국
      • Seongnam-si, 대한민국
        • Seoul National University Bundang Hospital
      • Seoul, 대한민국
        • Asan Medical Center
      • Seoul, 대한민국
        • Samsung Medical Center
  • 말레이시아
      • Selayang Baru Utara, 말레이시아
        • Hospital Selayang
    • Kuala Lumpur Federal Territory of Kuala Lumpur
      • Petaling Jaya, Kuala Lumpur Federal Territory of Kuala Lumpur, 말레이시아, 59100
        • University Malaya Medical Centre
    • Pulau Pinang
      • George Town, Pulau Pinang, 말레이시아, 10450
        • Hospital Pulau Pinang
    • Selangor
      • Shah Alam, Selangor, 말레이시아, 40000
        • Hospital Shah Alam
      • Sungai Buloh, Selangor, 말레이시아, 47000
        • Hospital Al-sultan Abdullah Uitm
  • 미국
    • California
      • Beverly Hills, California, 미국, 90211
        • Retina-Vitreous Associates Medical Group
      • Palo Alto, California, 미국, 94303
        • Stanford Byers Eye Institute
      • Santa Barbara, California, 미국, 93103
        • California Retina Consultants- Santa Barbara
    • Florida
      • Jacksonville, Florida, 미국, 32216
        • Florida Retina Institute - Jacksonville Southside
    • Georgia
      • Augusta, Georgia, 미국, 30909
        • Southeast Retina Center
    • Maryland
      • Hagerstown, Maryland, 미국, 21740-5940
        • Cumberland Valley Retina Consultants
    • Oregon
      • Eugene, Oregon, 미국, 97401
        • Sterling Vision, PC
    • Pennsylvania
      • Erie, Pennsylvania, 미국, 16507
        • Erie Retina Research
    • Virginia
      • Lynchburg, Virginia, 미국, 24502
        • Piedmont Eye Center
  • 호주
    • New South Wales
      • Albury, New South Wales, 호주, 2640
        • Eye Clinic Albury Wodonga
      • Hurstville, New South Wales, 호주, 2220
        • Retina And Eye Consultants Hurstville
      • Parramatta, New South Wales, 호주, 2150
        • Marsden Eye Specialists
      • Sydney, New South Wales, 호주, 2000
        • Sydney Eye Hospital
      • Sydney, New South Wales, 호주, 2000
        • Sydney Retina Clinic
      • Westmead, New South Wales, 호주, 2145
        • Sydney West Retina
    • Queensland
      • Birtinya, Queensland, 호주, 4575
        • University of the Sunshine Coast Clinical Trials (Birtinya)
    • South Australia
      • Adelaide, South Australia, 호주, 5000
        • Royal Adelaide Hospital
  • 홍콩
      • Shatin, 홍콩
        • Prince of Wales Hospital The Chinese University of Hong Kong
      • Wong Chuk Hang, 홍콩
        • HKU Eye Centre

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

18년 이상 (성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

모두

설명

Inclusion Criteria:

  • Written informed consent must be obtained from the subject prior to any study-related procedures.

  • Subject must be aged > 18 years at the time of Screening.

  • Subject must have a body mass index (BMI) of between 18 and 40 kg/m2, inclusive.

  • Subject has a documented diagnosis of T1DM or T2DM.

  • Subject has moderate to severe NPDR, as determined by a Central Reading Centre (CRC) using DRSS in at least one eye

  • Subject must have clear ocular media and be able to undergo adequate pupil dilation to allow adequate fundus imaging of both eyes.

  • Female subject must be either:

    1. Of non-childbearing potential: post-menopausal or documented surgically sterile post hysterectomy (at least 1 month prior to Screening)
    2. Or, if of childbearing potential, must have a negative serum pregnancy test at Screening and must use 2 acceptable forms of contraception, starting at Screening and throughout the study period and for 28 days after the final IP administration.

  • Female subject must not be breastfeeding at Screening or during the study period, and for 28 days after the final IP administration.

  • Male subject must be surgically sterile (> 30 days since vasectomy with no viable sperm), or if engaged in sexual relations with a female of childbearing potential, the couple should agree to use 2 acceptable contraceptive methods from Screening, during the study, and for 28 days after last IP administration.

Female subject must not donate ova or male subject must not donate sperm starting at Screening and throughout the study period, and for 28 days after the final IP administration.

  • Subject must have Best Corrected Visual Acuity (BCVA) assessed by Early Treatment Diabetic Retinopathy Study (ETDRS) protocol letters score of ≥ 70 letters in study eye, and ≥ 20 letters in the non-qualified fellow eye.
  • Subject must have the ability, in the opinion of the Investigator, and willingness to return for all scheduled visits and perform all assessments.
  • Subject agrees not to participate in another interventional study after signing the informed consent and until the End of Study (EOS) visit has been completed.

Exclusion Criteria:

Ophthalmic:

  • Presence of CI-DME (with central subfield thickness [CST] measured greater than 325 μm on spectral domain optical coherence tomography [SD-OCT]) threatening the center of the macula (within 1,000 μm of the foveal center) in either eye, or presence of DME requiring treatment.

  • Presence of moderate to high-risk PDR (DRSS level 65 or higher).

  • Any prior treatment (in either eye) with:

    1. Focal or grid laser photocoagulation within the past 6 months prior to Screening or pan-retinal photocoagulation (PRP) at any time.
    2. Systemic or intravitreal anti-vascular endothelial growth factor (VEGF) agents within the last 12 months prior to Screening.
    3. Intraocular, sub-tenon or periocular steroids, including triamcinolone and dexamethasone implant within the last 6 months, or suprachoroidal triamcinolone within the last 3 months prior to Screening.
    4. Fluocinolone implant within the last 3 years prior to Screening.
    5. Prior treatment for NPDR with any other treatment which is not labelled for NPDR within 1 year prior to Screening (e.g., calcium dobesilate, fibrate medication).
    6. Vitrectomy at any timepoint prior to Screening.
    7. Yttrium-Aluminium-Granate (YAG) capsulotomy within 3 months prior to Screening.

  • Active uveitis, vitritis, or infection in either eye including infectious conjunctivitis, keratitis, scleritis, or endophthalmitis.

  • History of corneal transplant and/or vitrectomy or any other ocular incisional surgery in either eye (e.g., shunt surgery). Note: Subjects who have had cataract or refractive surgery in either that was more than 3 months prior to Screening may be permitted at the discretion of the Investigator.

  • Uncontrolled glaucoma, as evidenced by intraocular pressure (IOP) > 25 mmHg despite up to 4 glaucoma medications, or evidence of glaucomatous visual field loss or has advanced glaucoma (e.g., prior shunt surgery) in either eye.

  • Clinically significant ocular disease in either eye that in the opinion of the Investigator would preclude participation in the study.

  • Presence of macular or retinal vascular disease including DME and/or retinopathy from causes other than diabetes, age-related macular degeneration, pattern dystrophy, choroidal neovascularisation of any cause, retinal vein occlusion, retinal artery occlusion in either eye.

  • History of retinal detachment or full-thickness macular hole post intraocular surgery in either eye, or idiopathic or autoimmune uveitis in either eye.

  • Any other ocular disease that may cause substantial reduction in BCVA.

Systemic:

  • Known, suspected hypersensitivity or contraindication to IP.
  • Uncontrolled diabetes mellitus with HbA1c of ≥ 12%.
  • Initiation of treatment with glucagon-like peptide-1 (GLP-1) modulators for glycaemic control and other indications within the last 3 months prior to Screening.
  • Initiation of intensive insulin treatment (a pump or multiple daily injections) within 3 months prior to Screening or plans to do so in the next 3 months.
  • Current use of coumarin anticoagulants (Coumadin/Warfarin).
  • On dialysis or an estimated glomerular filtration rate (eGFR) of < 30 mL/min/1.73m2 as per CKD-EPI evaluation at Screening. (Active Diabetic Ketoacidosis or Hyperglycemic Hyperosmolar Nonketotic State).
  • Hypertension with resting diastolic blood pressure (BP) > 100 mmHg or systolic BP > 180 mmHg on 2 consecutive measurements at least 5 minutes apart. Note: If the result is out of range, the assessment may be repeated once prior to randomisation for confirmation.
  • Resting heart rate outside the specified range (50 to 110 beats per minute). Note: If the result is out of range, the assessment may be repeated once prior to randomisation for confirmation.
  • History of chronic liver disease or presence of elevated (defined as > 3 × upper limit of normal) alanine aminotransferase (ALT) and aspartate aminotransferase (AST) consistent with such diagnosis.
  • Known to be immunocompromised or receiving immunosuppressive therapy. Note: Subjects receiving low dose corticosteroids may be eligible, at the discretion of the Investigator.
  • Currently receiving treatment with a strong inhibitor of the P-glycoprotein transporter (see Section 6.4.2), which may interfere with the IP.
  • History of allergy to fluorescein.
  • Any disease or medical condition that in the opinion of the Investigator would interfere with the study, prevent the subject from successfully participating in the study, or which might confound the study results.
  • Participation in any investigational study within 30 days prior to Screening or planning to participate in any other investigational drug or device clinical trials within 30 days of study completion.
  • History of blood transfusion or severe blood loss within 3 months prior to Screening, known hemoglobinopathy, and severe anaemia.

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: 네 배로

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: VX-01

Cohort 1 will include 50 subjects who will be randomized to take investigational drug VX-01 (film-coated tablets) at dose of 150 mg, administered BID.
의약품: VX-01

There is no physical difference in VX-01 and the placebo. The only difference lies in active ingredient found in VX-01, which is the compound that will be evaluated in the course of this study.
위약 비교기: Placebo

Cohort 2 will include 50 subjects who will be randomized to receive the placebo drug (film-coated tablets), that will be administered BID.
의약품: Placebo

Placebo will be supplied as a tablet identical to test drug but without VX-01. Placebo packaging will be identical to IP in order to keep study personnel and subjects masked.

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Evaluate the efficacy of oral doses of VX-01 in subjects compared to placebo following 1 year of treatment.
기간: From enrollment to the end of treatment at week 52

The endpoint of this objective is the proportion of subjects who do not develop a worsening from Baseline in binocular ETDRS DRSS at Week 52. The diabetic retinopathy severity scale (DRSS) is a scale healthcare professionals use to measure the severity and progression of a person's diabetic retinopathy. The main DRSS is the Early Treatment Diabetic Retinopathy Study (ETDRS) scale which will be used in this study.
From enrollment to the end of treatment at week 52

2차 결과 측정

결과 측정
측정값 설명
기간
To evaluate the efficacy of VX-01 in subjects with moderate to severe NPDR without CI-DME by determining the overall change from Baseline in BCVA letter scores.
기간: From enrollment to the end of treatment at week 52

Endpoint measurement of this objective will be improvement in Best Corrected Visual Acuity (BCVA), i.e. higher scoring at end of treatment compared to enrollment (baseline).
From enrollment to the end of treatment at week 52
To evaluate the efficacy of VX-01 in subjects with moderate to severe NPDR without CI-DME by determining the overall change from Baseline in the ETDRS DRSS scores.
기간: From enrollment to the end of treatment at week 52

Endpoint measurement of this objective will be improvement in the Early Treatment Diabetic Retinopathy score (ETDRS), i.e. higher score at end of treatment compared to enrollment (baseline measure)
From enrollment to the end of treatment at week 52
Incidence of Treatment-Emergent Adverse Events (TEAEs) when taking multiple oral doses of VX-01.
기간: From enrollment to the end of treatment at week 52

Endpoint measurement of this objective will include assessing the nature, frequency, and severity of systemic and ocular TEAEs across the duration of the study. A lower number of TEAEs would point towards a better safety and tolerability profile of VX-01
From enrollment to the end of treatment at week 52
Number of subjects developing moderate to high risk proliferative diabetic retinopathy
기간: From enrollment to the end of treatment at week 52

Measurement of proportion of subjects who develop moderate to high-risk proliferative diabetic retinopathy in either eye at Week 24 and also at Week 52 will be quantified. This will aid in understanding the safety of VX-01.
From enrollment to the end of treatment at week 52

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Vantage Biosciences Ltd

협력자

Vantage Biosciences Australia Pty Ltd

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)

2025년 2월 11일

기본 완료 (추정된)

2027년 7월 1일

연구 완료 (추정된)

2027년 10월 1일

연구 등록 날짜

최초 제출

2024년 12월 16일

QC 기준을 충족하는 최초 제출

2025년 1월 7일

처음 게시됨 (실제)

2025년 1월 13일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 10월 2일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 9월 28일

마지막으로 확인됨

2026년 9월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

  • VX01-DR-201

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

미정

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

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