Trial Review

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06770933




Registration number
NCT06770933
Ethics application status
Date submitted
16/12/2024
Date registered
13/01/2025
Date last updated
13/01/2025

Titles & IDs
Public title
A Study to Evaluate the Efficacy and Safety of Orally Administered VX-01
Scientific 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)
Secondary ID [1] 0 0
VX01-DR-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Diabetic Retinopathy 0 0
NPDR - Non Proliferative Diabetic Retinopathy 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye
Metabolic and Endocrine 0 0 0 0
Diabetes

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - VX-01
Treatment: Drugs - Placebo

Experimental: 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.

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


Treatment: Drugs: 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.

Treatment: Drugs: 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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Evaluate the efficacy of oral doses of VX-01 in subjects compared to placebo following 1 year of treatment.
Timepoint [1] 0 0
From enrollment to the end of treatment at week 52
Secondary outcome [1] 0 0
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.
Timepoint [1] 0 0
From enrollment to the end of treatment at week 52
Secondary outcome [2] 0 0
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.
Timepoint [2] 0 0
From enrollment to the end of treatment at week 52
Secondary outcome [3] 0 0
Incidence of Treatment-Emergent Adverse Events (TEAEs) when taking multiple oral doses of VX-01.
Timepoint [3] 0 0
From enrollment to the end of treatment at week 52
Secondary outcome [4] 0 0
Number of subjects developing moderate to high risk proliferative diabetic retinopathy
Timepoint [4] 0 0
From enrollment to the end of treatment at week 52

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/01/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/03/2027
Actual
Sample size
Target
100
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Retina And Eye Consultants Hurstville - Hurstville
Recruitment postcode(s) [1] 0 0
2220 - Hurstville

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Vantage Biosciences Ltd
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Vantage Biosciences Australia Pty Ltd
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/study/NCT06770933
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Steffy George
Address 0 0
Country 0 0
Phone 0 0
+44 7450953382
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06770933