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Safety and Efficacy of Zuretinol Acetate in Subjects With Inherited Retinal Disease

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 evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04311112
Recruitment Status : Suspended (Company dissolved)
First Posted : March 17, 2020
Last Update Posted : October 9, 2020
Sponsor:
Information provided by (Responsible Party):
Retinagenix Therapeutics

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).

Condition or disease Intervention/treatment Phase
Retinal Disorder Drug: Placebos Drug: ZA Low dose Drug: ZA high dose Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 45 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Estimated Study Start Date : December 2020
Estimated Primary Completion Date : February 2023
Estimated Study Completion Date : September 2023

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Placebo Comparator: ZA placebo Drug: Placebos
Placebo

Active Comparator: ZA low dose Drug: ZA Low dose
ZA low dose

Active Comparator: ZA high dose Drug: ZA high dose
ZA high dose




Primary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Have read, understood and signed the informed consent form (ICF).
  2. Be aged 6 years or older.
  3. 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.
  4. Be naïve to gene therapy, surgical implantation of prosthetic retinal chips, or subretinal injections.
  5. If previously administered ZA , have at least > 3 years since last administration of ZA.
  6. Pregnancy testing and contraception before study treatment: Women of childbearing potential must not be pregnant or lactating.

Exclusion Criteria:

  1. 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.
  2. Have had ocular surgery within 3 months of Screening, including cataract or laser procedures.
  3. 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.
  4. Have taken any supplements containing ≥ 10,000 IU vitamin A within 60 days of Screening.
  5. Have taken any medication that affects bone metabolism within 6 months of Screening.
  6. Have circulating 25-hydroxy vitamin D < 20 ng/mL.
  7. Use of medications that may interact with a retinoid, including tetracycline, ketoconazole and methotrexate within 60 days of Screening.
  8. 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.
  9. Have a known and documented allergy to soy.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04311112


Locations
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United States, New York
NY NY
New York, New York, United States, 10001
Sponsors and Collaborators
Retinagenix Therapeutics
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Responsible Party: Retinagenix Therapeutics
ClinicalTrials.gov Identifier: NCT04311112    
Other Study ID Numbers: RG201
First Posted: March 17, 2020    Key Record Dates
Last Update Posted: October 9, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Retinal Diseases
Eye Diseases