UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	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
Title of the study (Brief title)	The Clinical Study for Neuroprotective Gene Therapy to Treat Patients with Retinitis Pigmentosa
Region	Japan

Condition
Condition	Retinitis Pigmentosa
Classification by specialty	Ophthalmology Adult
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	To evaluate the safety of the gene therapy for patients with retinitis pigmentosa via the 3rd generation of recombinant simian immunodeficiency virus (SIV) vector expressing human pigment epithelium-derived factor (hPEDF) gene
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2	Explanatory
Developmental phase	Phase I

Assessment
Primary outcomes	1. Physical examination 2. Blood and urine analyses 3. Vital signs 4. Ophthalmic examination 5. Adverse events 6. Viral shedding
Key secondary outcomes	1. Best corrected visual acuity 2. Visual field measurement testing 3. VFQ25

Base
Study type	Interventional

Study design
Basic design	Parallel
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	No treatment
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	2
Purpose of intervention	Treatment
Type of intervention	Gene
Interventions/Control_1	Subretinal injection of the 3rd generation of recombinant simian immunodeficiency virus (SIV) vector expressing human pigment epithelium-derived factor (hPEDF) gene (low titer group)
Interventions/Control_2	Subretinal injection of the 3rd generation of recombinant simian immunodeficiency virus (SIV) vector expressing human pigment epithelium-derived factor (hPEDF) gene (high titer group)
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	40 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key 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
Key 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
Target sample size	20

Research contact person
Name of lead principal investigator	Koh-Hei Sonoda
Organization	Kyushu University Hospital
Division name	Department of Ophthalmology
Address	3-1-1 Maidashi, Higashi-ku, Fukuoka, Japan
TEL	092-642-5648
Email	sonodak@med.kyushu-u.ac.jp

Public contact
Name of contact person	Yasuhiro Ikeda
Organization	Kyushu University Hospital
Division name	Department of Ophthalmology
Address	3-1-1 Maidashi, Higashi-ku, Fukuoka, Japan
TEL	092-642-5648
Homepage URL
Email	ymocl@pathol1.med.kyushu-u.ac.jp

Sponsor
Institute	Kyushu University Hospital
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2013 Year 03 Month 26 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2012 Year 08 Month 23 Day
Anticipated trial start date	2013 Year 03 Month 21 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete	2018 Year 09 Month 19 Day
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2013 Year 03 Month 17 Day
Last modified on	2018 Year 10 Month 20 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000012009