Phase III Efficacy and Safety Clinical Study of UF-021 for Treatment of Retinitis Pigmentosa

• Register: ClinicalTrials.gov
• Last refreshed on: 8 February 2016
• Main ID: NCT01786395
• Date of registration: 23/01/2013
• Prospective Registration: Yes
• Primary sponsor: R-Tech Ueno, Ltd.
• Public title: Phase III Efficacy and Safety Clinical Study of UF-021 for Treatment of Retinitis Pigmentosa
• Scientific title: Phase III Clinical Study of UF-021 for Retinitis Pigmentosa - Evaluation for a Comparative Double Masked Placebo Controlled Study Period and a Continuous Administration Period
• Date of first enrolment: March 2013
• Target sample size: 202
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01786395
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

Japan

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Age: 20 years or more, 70 years or less (at obtaining informed consent)

2. Medical examination classification: Outpatients

3. Observation, examination, and surveillance in accordance with the study protocol are
judged to be feasible.

4. ETDRS visual acuity test is judged to be feasible at a distance of 4 meters.

5. HFA (10-2) test is judged to be feasible by investigator.

6. Goldmann perimetry shows concentric central visual field loss (including a ring
scotoma) with the central 30 degrees or less.

7. The difference in the mean retinal sensitivity at four central points must be less
than 3 dB between two reliable measurements with HFA (10-2) (SITA-Standard) conducted
within 31 days and both values are worse than 30 dB.(When this criterion is not met
after two tests, a 3rd measurement will be implemented within 31 days from the 2nd
test. The difference between the 3rd reliable measurement value and the 1st or 2nd
measurements must be less than 3 dB, and both values are worse than 30 dB; the most
recent data will be regarded as the data acquired before instillation.)

Exclusion Criteria:

1. Judged to have difficulty by investigator for visiting the hospital and returning
home safely over the study period.

2. Planning to undergo an ophthalmic operation for eye for efficacy evaluation during
study period.

3. Current treatment for glaucoma or ocular hypertension.

4. Prior ophthalmectomy or evisceration of an eye

5. Intraocular surgery within the past five months.

6. History of allergy to drugs (instillation narcotics, fluorescein, etc.) that will be
used during the clinical study, and to drugs similar to the investigational product

7. Complications of diabetic retinopathy.

8. Complications of external eye inflammation, infectious diseases, or severe dry eye.

9. Use of isopropyl unoprostone in the past or present.

10. Use of the following drugs within 31 days before obtaining informed consent. Calcium
antagonists, Dark adaptation improvement drug (helenien)

11. Participation in UF-021 phase ? trial (including subjects assigned to the placebo
group).

12. Participation in other clinical studies within the past 6 months (However, any
subject who has not been administered an investigational product will be accepted)

13. Pregnancy or the possibility of becoming pregnant. Currently breastfeeding.
Childbearing patients who wish to become pregnant during the clinical study period
and are not using appropriate contraceptive measures.

14. Cone-rod dystrophy where cone function was primarily impaired

15. History of optic nerve disease in the eye for efficacy evaluation

16. Complications of a moderate or more severe (grade 3 of the Emery classification)
central cataract, an anterior subcapsular cataract, a posterior subcapsular cataract,
and posterior capsule opacification that may exert a major influence on visual acuity
in the eye for efficacy evaluation



Age minimum: 20 Years
Age maximum: 70 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Retinitis Pigmentosa

Intervention(s)

Drug: Placebo

Drug: UF-021

Primary Outcome(s)

Changes in the value of the mean retinal sensitivity at four central points through HFA (10-2) [Time Frame: baseline and 1 year]

Secondary Outcome(s)

- Changes in the value of the ETDRS visual acuity [Time Frame: baseline and 1 year]

- Changes in the Goldmann visual field area within V4e isopter [Time Frame: baseline and 1 year]

- Changes in the value of the retinal sensitivity through HFA (10-2) (MD value, mean retinal sensitivity at 12 central points /24 central points /68 central points) [Time Frame: baseline and 1 year]

- Changes in the value of retinal thickness through OCT [Time Frame: baseline and 1 year]

- Changes in the VFQ-25 (composite 8) value [Time Frame: baseline and 1 year]

Secondary ID(s)

UF-021-C003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors