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Investigator-initiated Phase 1 clinical trial to evaluate the safety of Pitavastatin PLGA nano-particle in patients with retinitis pigmentosa
(Phase 1 clinical trial to evaluate the safety of Pitavastatin PLGA nano-particle in patients with retinitis pigmentosa)

Safety Evaluation Study of Pitavastatin PLGA nano-particle for Retinitis Pigmentosa (RP CLARITI trial)

Murakami Yusuke

Kyushu University Hospital

3-1-1 Maidashi Higashi-ku Fukuoka 812-8582,Japan

murakami.yusuke.407@m.kyushu-u.ac.jp

Murakami Yusuke

Kyushu University Hospital

3-1-1 Maidashi Higashi-ku Fukuoka 812-8582,Japan

+81-92-642-5648

murakami.yusuke.407@m.kyushu-u.ac.jp

Pending

June. 01, 2026

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

1. Patients diagnosed with typical retinitis pigmentosa by two ophthalmologists according to the Clinical Practice Guidelines for Retinitis Pigmentosa (genomic diagnosis will not be performed).
2. Patients aged 18 years or older and 70 years or younger at the time of informed consent.
3. Patients with a mean retinal sensitivity of 10 dB or higher within the central 4 degrees (12 points) of the Humphrey 10-2 visual field.
4. Patients whose difference in mean retinal sensitivity (MD value) between two Humphrey 10-2 examinations at screening is within 3 dB for both eyes. If the criteria are not met in the second examination, a third measurement will be performed within 14 days of the second examination, and the difference between the third measurement and the first or second measurement must be within 3 dB.
5. Patients with a foveal retinal thickness of 250 micrometer or less as measured by optical coherence tomography.
6. Female patients of childbearing potential who agree to use appropriate contraception from the time of informed consent until 180 days after the last dose of the investigational product.
7. Male patients who agree to use appropriate contraception for 3 days from each administration of the investigational product.
8. Patients who can provide written informed consent.

1. Patients currently taking statins for hyperlipidemia.
2. Patients who have received helenien, unoprostone, or calcium channel blockers for the treatment of eye diseases within 30 days prior to informed consent.
3. Patients scheduled for ophthalmic surgery during the study period.
4. Patients with concomitant glaucoma or ocular hypertension.
5. Patients with concomitant uveitis or optic neuritis.
6. Patients with retinal lesions not attributable to retinitis pigmentosa (e.g., fundus hemorrhage, retinal edema, proliferative tissue, etc.) observed on fundus examination.
7. Patients with a history of hypersensitivity or severe adverse reactions to pitavastatin calcium or any component of the investigational product.
8. Patients with severe allergies or a history thereof.
9. Patients with severe renal impairment.
10. Patients with severe cardiac dysfunction or heart failure.
11. Patients with severe hepatic impairment.
12. Patients with biliary obstruction.
13. Patients with active inflammatory diseases or infections (e.g., active collagen disease, rheumatoid arthritis, ulcerative colitis, sepsis, etc.).
14. Patients with a history of cerebral hemorrhage, cerebral infarction, or ischemic heart disease within 6 months prior to informed consent.
15. Patients with hematological disorders (e.g., severe anemia, leukemia, aplastic anemia, etc.).
16. Patients with alcohol dependence, drug dependence, or mental disorders that may interfere with study participation.
17. Patients with concomitant malignant tumors or who have received treatment for malignant tumors within the past 3 years.
18. Patients currently receiving cyclosporine.
19. Patients currently receiving fibrate drugs (e.g., clofibrate, fenofibrate, bezafibrate, etc.) who cannot discontinue fibrate drugs from 3 days prior to the start of investigational product administration.
20. Patients currently receiving nicotinic acid, erythromycin, or rifampicin who cannot discontinue these drugs from 3 days prior to the start of investigational product administration.
21. Patients currently participating in other clinical trials or studies.
22. Pregnant women, women suspected of being pregnant, or lactating women.
23. Any other patient judged unsuitable by the principal investigator or sub-investigator.

Both

Retinitis Pigmentosa

Part 1 (Single Dose): Dissolve 2 mg, 4 mg, or 8 mg of pitavastatin calcium in 100 mL of physiological saline and infuse intravenously over 1 hour.
Part 2 (Multiple Doses): Infuse the maximum dose confirmed safe in Part 1 intravenously once a month for 3 months, over 1 hour.

retina, degeneration

Infusions, Intravenous

D012174

D007262

Incidence of adverse events

Part 1:
1. Time course of drug concentrations of pitavastatin and pitavastatin lactone in plasma.
2. Urinary excretion of pitavastatin, pitavastatin lactone, and pitavastatin conjugates in urine.
3. Changes from baseline (pre-investigational product administration) in body temperature, blood pressure, pulse rate, and transcutaneous arterial oxygen saturation at 8 hours, 24 hours, and 48 hours after the start of administration, and on Day 28.
4. Changes from baseline (pre-investigational product administration) in each clinical laboratory value at 8 hours, 24 hours, and 48 hours after the start of administration, and on Day 7 and Day 28 (blood tests only at 8 hours, Day 7, and Day 28; blood and urine tests at 24 hours and 48 hours).
5. Changes from baseline (at screening) in ETDRS visual acuity letter score at 24 hours after the start of administration, and on Day 7 and Day 28.
6. Changes from baseline (at screening) in OCT Ellipsoid Zone length at 24 hours after the start of administration, and on Day 7 and Day 28.
7. Changes from baseline (at screening) in autofluorescence examination / fluorescent ring area at 24 hours after the start of administration, and on Day 7 and Day 28.
8. Changes from baseline (at screening) in Humphrey 10-2 visual field analyzer / central 4-point mean sensitivity at 24 hours after the start of administration, and on Day 7 and Day 28.
9. Changes from baseline (at screening) in Humphrey 10-2 visual field analyzer / central 12-point mean sensitivity at 24 hours after the start of administration, and on Day 7 and Day 28.
10. Changes from baseline (at screening) in Humphrey 10-2 visual field analyzer / MD value at 24 hours after the start of administration, and on Day 7 and Day 28.
11. Changes from baseline (at screening) in Humphrey 10-2 visual field analyzer / functional transition points (FTP) retinal sensitivity at 24 hours after the start of administration, and on Day 7 and Day 28.
12. Changes from baseline (at screening) in anterior chamber flare value at 24 hours after the start of administration, and on Day 7 and Day 28.

Part 2:
1. Time course of drug concentrations of pitavastatin and pitavastatin lactone in plasma after each investigational product administration.
2. Urinary excretion of pitavastatin, pitavastatin lactone, and pitavastatin conjugates in urine after each investigational product administration.
3. Changes from baseline (pre-investigational product administration) in body temperature, blood pressure, pulse rate, and transcutaneous arterial oxygen saturation at 8 hours, 24 hours, and 48 hours after the start of each investigational product administration, and on Day 28.
4. Changes from baseline (pre-investigational product administration) in each clinical laboratory value at 8 hours, 24 hours, and 48 hours after the start of each investigational product administration, and on Day 7 and Day 28 (blood tests only at 8 hours, Day 7, and Day 28; blood and urine tests at 24 hours and 48 hours).
5. Changes from baseline (at screening) in ETDRS visual acuity letter score at 24 hours after the start of the first administration, and on Day 7, Day 28, Day 56, and Day 84.
6. Changes from baseline (at screening) in OCT Ellipsoid Zone length at 24 hours after the start of the first administration, and on Day 7, Day 28, Day 56, and Day 84.
7. Changes from baseline (at screening) in autofluorescence examination / fluorescent ring area at 24 hours after the start of the first administration, and on Day 7, Day 28, Day 56, and Day 84.
8. Changes from baseline (at screening) in Humphrey 10-2 visual field analyzer / central 4-point mean sensitivity at 24 hours after the start of the first administration, and on Day 7, Day 28, Day 56, and Day 84.
9. Changes from baseline (at screening) in Humphrey 10-2 visual field analyzer / central 12-point mean sensitivity at 24 hours after the start of administration, and on Day 7, Day 28, Day 56, and Day 84.
10. Changes from baseline (at screening) in Humphrey 10-2 visual field analyzer / MD value at 24 hours after the start of the first administration, and on Day 7, Day 28, Day 56, and Day 84.
11. Changes from baseline (at screening) in Humphrey 10-2 visual field analyzer / FTP retinal sensitivity at 24 hours after the start of the first administration, and on Day 7, Day 28, Day 56, and Day 84.
12. Changes from baseline (at screening) in anterior chamber flare value at 24 hours after the start of the first administration, and on Day 7, Day 28, Day 56, and Day 84.

Jan. 23, 2026

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