UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000010260 |
|---|---|
| Receipt number | R000012009 |
| Scientific Title | 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 |
| Date of disclosure of the study information | 2013/03/26 |
| Last modified on | 2018/10/20 09:34:48 |
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Basic information
Public title
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
Acronym
The Clinical Study for Neuroprotective Gene Therapy to Treat Patients with Retinitis Pigmentosa
Scientific Title
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
Scientific Title:Acronym
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
| 1st name | |
| Middle name | |
| Last name | Koh-Hei Sonoda |
Organization
Kyushu University Hospital
Division name
Department of Ophthalmology
Zip code
Address
3-1-1 Maidashi, Higashi-ku, Fukuoka, Japan
TEL
092-642-5648
sonodak@med.kyushu-u.ac.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Yasuhiro Ikeda |
Organization
Kyushu University Hospital
Division name
Department of Ophthalmology
Zip code
Address
3-1-1 Maidashi, Higashi-ku, Fukuoka, Japan
TEL
092-642-5648
Homepage URL
ymocl@pathol1.med.kyushu-u.ac.jp
Sponsor or person
Institute
Kyushu University Hospital
Institute
Department
Personal name
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)
IRB Contact (For public release)
Organization
Address
Tel
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 |
Related information
URL releasing protocol
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Completed
Date of protocol fixation
| 2012 | Year | 08 | Month | 23 | Day |
Date of IRB
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
Other
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000012009
