RECRUITING

A Study to Evaluate the Efficacy and Safety of Orally Administered VX-01

Conditions

Diabetic Retinopathy NPDR - Non Proliferative Diabetic Retinopathy NPDR

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

A Phase II, Double-Masked, Randomised, Placebo-Controlled, Parallel Design Study to Evaluate the Efficacy and Safety of Orally Administered VX-01 in Diabetic Retinopathy of Non-Proliferative Type (NPDR)

Quick Facts

Study Start:2025-02-11

Study Completion:2027-03

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06770933

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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. * 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.	* 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. * 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.

Inclusion Criteria

Exclusion 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.
* 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.

* 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.
* 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.

Contacts and Locations

Study Contact

Steffy George

CONTACT

+44 7450953382

steffy.george@vantage-biosciences.com

Study Locations (Sites)

Retina-Vitreous Associates Medical Group

Beverly Hills, California, 90211

United States

Stanford Byers Eye Institute

Palo Alto, California, 94303

United States

California Retina Consultants- Santa Barbara

Santa Barbara, California, 93103

United States

Florida Retina Institute - Jacksonville Southside

Jacksonville, Florida, 32216

United States

Retina Associates

Elmhurst, Illinois, 60126

United States

Cumberland Valley Retina Consultants

Hagerstown, Maryland, 21740-5940

United States

Erie Retina Research

Erie, Pennsylvania, 16507

United States

Piedmont Eye Center

Lynchburg, Virginia, 24502

United States

Collaborators and Investigators

Sponsor: Vantage Biosciences Ltd

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-02-11

Study Completion Date2027-03

Study Record Updates

Study Start Date2025-02-11

Study Completion Date2027-03

Terms related to this study

Keywords Provided by Researchers

NPDR
Diabetic Retinopathy

Additional Relevant MeSH Terms

Diabetic Retinopathy
NPDR - Non Proliferative Diabetic Retinopathy