Retinal DisorderPhase 2/3NCT04311112
Safety and Efficacy of Zuretinol Acetate in Subjects With Inherited Retinal Disease
A Phase 2/3 study testing ZA Low dose and ZA high dose in people with Retinal Disorder, run by Retinagenix Holdings at 1 site in United States.
Trial summary
To evaluate the efficacy of ZA oral solution in subjects with IRD caused by biallelic recessive RPE65 or LRAT gene mutations and phenotypically diagnosed as Leber's Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP). Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Retinal Disorder
- What is being tested?
- ZA Low dose and ZA high dose
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2020to Sep 2023
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Have read, understood and signed the informed consent form (ICF).
- Be aged 6 years or older.
- Have a diagnosis of IRD phenotypically diagnosed as LCA or RP by an ocular geneticist or ophthalmologist and caused by pathologic biallelic autosomal recessive mutation in RPE65 or LRAT as determined by a fully accredited certified central genotyping laboratory.
- Be naïve to gene therapy, surgical implantation of prosthetic retinal chips, or subretinal injections.
- If previously administered ZA , have at least > 3 years since last administration of ZA.
- Pregnancy testing and contraception before study treatment: Women of childbearing potential must not be pregnant or lactating.
Who cannot join
- Have a presence of concurrent ocular disease that in the opinion of the Investigator would put the subject at greater risk during the study or significantly affect study results.
- Have had ocular surgery within 3 months of Screening, including cataract or laser procedures.
- Have taken any prescription or investigational oral retinoid medication (e.g., isotretinoin or acitretin) within 6 months of Screening; subjects who did not tolerate their previous oral retinoid medication will be excluded regardless of the time of last exposure.
- Have taken any supplements containing ≥ 10,000 IU vitamin A within 60 days of Screening.
- Have taken any medication that affects bone metabolism within 6 months of Screening.
- Have circulating 25-hydroxy vitamin D < 20 ng/mL.
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- Use of medications that may interact with a retinoid, including tetracycline, ketoconazole and methotrexate within 60 days of Screening.
- Use of intraocular or periocular corticosteroids within 90 days of Screening; use of corticosteroid implants within 3 years of Screening; use of systemic corticosteroids unless these are at a steady low dose with low dose level in effect prior to or at Screening; or use of intraocular or periocular anti-vascular endothelial growth factor agents within 2 months of Screening.
- Have a known and documented allergy to soy.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Safety and Efficacy of Zuretinol Acetate Oral Solution in Subjects With Inherited Retinal Disease Caused by Mutations in Retinal Pigment Epithelium Protein 65 or Lecithin:Retinol Acyltransferase
To evaluate the efficacy of ZA oral solution in subjects with IRD caused by biallelic recessive RPE65 or LRAT gene mutations and phenotypically diagnosed as Leber's Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP).
- Conditions
- Retinal Disorder
- Being tested
- Placebos, ZA Low dose, ZA high dose
- Medical subject headings
- Retinal Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlacebosDrug
Placebo
ZA Low doseDrug
ZA low dose
ZA high doseDrug
ZA high dose
| Study group | Role | What they receive |
|---|---|---|
| ZA placebo | Placebo comparator | Not described Receives: Drug: Placebos |
| ZA low dose | Active comparator | Not described Receives: Drug: ZA Low dose |
| ZA high dose | Active comparator | Not described Receives: Drug: ZA high dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in functional vision at Week 12 using a visual navigation course at different ambient illumination levels
The primary efficacy endpoint (PEE) will be a measure of functional vision assessed at Week 12 employing mobility testing (Ora Inc, Andover, MA) via their Visual Navigation Challenge (VNC) course. The VNC measures functional vision by evaluating the subject's ability to navigate a maze accurately and successfully at different levels of ambient illumination. The PEE is a comparison between groups in their mean change from randomization to week 12, that is, the difference in VNC score from baseline performance to week 12 performance. Performance on the VNC is assessed using each eye individually and both eyes together at 1 or more levels of illumination that range from 0.35 lux to 500 lux. A subject's VNC score is the lowest level of luminance at which the subject can navigate and correctly pass through the maze.
Measured: week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2020
- Study startDec 2020 (estimated)
- Primary completionFeb 2023 (estimated)
- Study completionSep 2023 (estimated)
Why it stopped: The asset was transferred to another company
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinal DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Retinagenix Holdings
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04311112
- Sponsor's protocol ID
- RG201
- Status last verified
- Oct 2020
- First submitted
- 12 Mar 2020
- Last updated
- 24 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Zuretinol Acetate Oral Solution in Subjects With Inherited Retinal Disease Caused by Mutations in Retinal Pigment Epithelium Protein 65 or Lecithin:Retinol Acyltransferase
- ClinicalTrials.gov ID
- NCT04311112
- Acronym / sponsor ID
- RG201
- Conditions
- Retinal Disorder
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The asset was transferred to another company
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Retinagenix HoldingsIndustry (pharma, biotech, devices)
- Study start
- Dec 2020 (estimated)
- Primary completion
- Feb 2023 (estimated)
- Study completion
- Sep 2023 (estimated)
- First posted
- 17 Mar 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the efficacy of ZA oral solution in subjects with IRD caused by biallelic recessive RPE65 or LRAT gene mutations and phenotypically diagnosed as Leber's Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ZA placebo | Placebo comparator | Drug: Placebos |
| ZA low dose | Active comparator | Drug: ZA Low dose |
| ZA high dose | Active comparator | Drug: ZA high dose |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Have read, understood and signed the informed consent form (ICF).
- Be aged 6 years or older.
- Have a diagnosis of IRD phenotypically diagnosed as LCA or RP by an ocular geneticist or ophthalmologist and caused by pathologic biallelic autosomal recessive mutation in RPE65 or LRAT as determined by a fully accredited certified central genotyping laboratory.
- Be naïve to gene therapy, surgical implantation of prosthetic retinal chips, or subretinal injections.
- If previously administered ZA , have at least > 3 years since last administration of ZA.
Key exclusion criteria (9 listed)
- Have a presence of concurrent ocular disease that in the opinion of the Investigator would put the subject at greater risk during the study or significantly affect study results.
- Have had ocular surgery within 3 months of Screening, including cataract or laser procedures.
- Have taken any prescription or investigational oral retinoid medication (e.g., isotretinoin or acitretin) within 6 months of Screening; subjects who did not tolerate their previous oral retinoid medication will be excluded regardless of the time of last exposure.
- Have taken any supplements containing ≥ 10,000 IU vitamin A within 60 days of Screening.
- Have taken any medication that affects bone metabolism within 6 months of Screening.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PlacebosPlacebo | Drug | ZA placebo |
| ZA Low doseZA low dose | Drug | ZA low dose |
| ZA high doseZA high dose | Drug | ZA high dose |
Primary outcome
Change in functional vision at Week 12 using a visual navigation course at different ambient illumination levels
Time frame: week 12
The primary efficacy endpoint (PEE) will be a measure of functional vision assessed at Week 12 employing mobility testing (Ora Inc, Andover, MA) via their Visual Navigation Challenge (VNC) course. The VNC measures functional vision by evaluating the subject's ability to navigate a maze accurately and successfully at different levels of ambient illumination. The PEE is a comparison between groups in their mean change from randomization to week 12, that is, the difference in VNC score from baseline performance to week 12 performance. Performance on the VNC is assessed using each eye individually and both eyes together at 1 or more levels of illumination that range from 0.35 lux to 500 lux. A subject's VNC score is the lowest level of luminance at which the subject can navigate and correctly pass through the maze.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing ZA Low dose and ZA high dose in people with Retinal Disorder, run by Retinagenix Holdings at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 24 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in functional vision at Week 12 using a visual navigation course at different ambient illumination levels (time frame: week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Feb 2023.