Back to resultsSafety and Efficacy of Zuretinol Acetate in Subjects With Inherited Retinal Disease
Primary Purpose
Retinal Disorder
Status
Withdrawn
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Placebos
ZA Low dose
ZA high dose
Sponsored by
About this trial
This is an interventional treatment trial for Retinal Disorder
Eligibility Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
- 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.
Sites / Locations
- NY NY
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Placebo Comparator
Active Comparator
Active Comparator
Arm Label
ZA placebo
ZA low dose
ZA high dose
Arm Description
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04311112
Brief Title
Safety and Efficacy of Zuretinol Acetate in Subjects With Inherited Retinal Disease
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
Study Type
Interventional
2. Study Status
Record Verification Date
October 2020
Overall Recruitment Status
Withdrawn
Why Stopped
The asset was transferred to another company
Study Start Date
December 2020 (Anticipated)
Primary Completion Date
February 2023 (Anticipated)
Study Completion Date
September 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Retinagenix Holdings
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief 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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinal Disorder
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
ZA placebo
Arm Type
Placebo Comparator
Arm Title
ZA low dose
Arm Type
Active Comparator
Arm Title
ZA high dose
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
Placebos
Intervention Description
Placebo
Intervention Type
Drug
Intervention Name(s)
ZA Low dose
Intervention Description
ZA low dose
Intervention Type
Drug
Intervention Name(s)
ZA high dose
Intervention Description
ZA high dose
Primary Outcome Measure Information:
Title
Change in functional vision at Week 12 using a visual navigation course at different ambient illumination levels
Description
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.
Time Frame
week 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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.
Exclusion Criteria:
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.
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.
Facility Information:
Facility Name
NY NY
City
New York
State/Province
New York
ZIP/Postal Code
10001
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Safety and Efficacy of Zuretinol Acetate in Subjects With Inherited Retinal Disease
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