The Clinical Study for Neuroprotective Gene Therapy to Treat Patients with Retinitis Pigmentosa via Subretinal Injection of The 3rd Generation of Recombinant Simian Immunodeficiency Virus (SIVagm) Vector Expressing Human Pigment Epithelium-Derived Factor (hPEDF) Gene

The Clinical Study for Neuroprotective Gene Therapy to Treat Patients with Retinitis Pigmentosa via Subretinal Injection of The 3rd Generation of Recombinant Simian Immunodeficiency Virus (SIVagm) Vector Expressing Human Pigment Epithelium-Derived Factor (hPEDF) Gene - The Clinical Study for Neuroprotective Gene Therapy to Treat Patients with Retinitis Pigmentosa

Status

Active, not recruiting

Phases

Phase 1

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000010260

Enrollment

Registered

2013-03-26

Start date

2013-03-21

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Retinitis Pigmentosa

Interventions

Subretinal injection of the 3rd generation of recombinant simian immunodeficiency virus (SIV) vector expressing human pigment epithelium-derived factor (hPEDF) gene (low titer group) Subretinal inject

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Subjects with retinitis pigmentosa 2. 40 years and above 3. Subjects who get a regular checkup at Kyushu University Hospital over 1 year

Exclusion criteria

Exclusion criteria: 1. Human immunodeficiency virus (HIV) antibody-positive subjects 2. Blindness 3. Subjects with a macular complication of retinitis pigmentosa (eg: macular degeneration,macular edema, etc.) 4. A past history of glaucoma 5. Subjects with retinal or subretinal disorders 6. Subjected with severe allergy or its history. 7. Subjects receiving chronic hemodialysis therapy 8. Subjects who have severe heart dysfunction or faiure 9. Subjects who have hepatic dysfunction or cirrhosis 10. Subject who have active inflammatory diseases 11. Subjects who have experienced celebral hemorrhage or infarction 6 months prior to treatment 12. Subjects with hematopoietic disorders 13. Alcoholism and/or drug dependence 14. Female subjects with pregnant or doubt of pregnancy 15. Subjects who are diagnosed with cancer or are suspected to cancer 16. Subject who have recieved operative resection of malignant neoplasm 5 years prior to treatment 17. Subjects who disapprove of birth control during at least 12 months after the vector administration 18. Subjects who take the antiviral drug at the registration 19. Others

Design outcomes

Primary

Measure	Time frame
1. Physical examination 2. Blood and urine analyses 3. Vital signs 4. Ophthalmic examination 5. Adverse events 6. Viral shedding	—

Secondary

Measure	Time frame
1. Best corrected visual acuity 2. Visual field measurement testing 3. VFQ25	—

Countries

Japan

Contacts

Public ContactYasuhiro Ikeda

Kyushu University Hospital Department of Ophthalmology

ymocl@pathol1.med.kyushu-u.ac.jp092-642-5648

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026