UMIN-ICDS Clinical Trial
| Unique ID issued by UMIN | UMIN000049825 |
|---|---|
| Receipt number | R000056748 |
| Scientific Title | Evaluation of long-term safety and efficacy after neuroprotective gene therapy with SIV-hPEDF for retinitis pigmentosa |
| Date of disclosure of the study information | 2023/01/04 |
| Last modified on | 2024/04/08 10:58:59 |
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Basic information
Public title
Evaluation of long-term safety and efficacy after neuroprotective gene therapy with SIV-hPEDF for retinitis pigmentosa
Acronym
Long-term safety and efficacy after SIV-hPEDF gene therapy for retinitis pigmentosa
Scientific Title
Evaluation of long-term safety and efficacy after neuroprotective gene therapy with SIV-hPEDF for retinitis pigmentosa
Scientific Title:Acronym
Long-term safety and efficacy after SIV-hPEDF gene therapy for retinitis pigmentosa
Region
| Japan |
Condition
Condition
Retinitis Pigmentosa
Classification by specialty
| Ophthalmology |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
Retinitis pigmentosa (RP) is an inherited retinal degenerative disease for which no effective treatment has yet been established. We have developed a simian immunodeficiency virus from African green monkey (SIVagm) vector carrying the human pigment epithelium-derived factor (PEDF) gene (SIV-hPEDF, DVC1-0401) that exhibits potent neuroprotective effects. The "Phase I/IIa investigator-initiated clinical trial of DVC1-0401 subretinal administration of neuroprotective gene therapy for retinitis pigmentosa" started in February 2019, with a one-year post-administration observation period. The trial is now underway with a one-year observation period after administration. In order to investigate the long-term safety and efficacy of this vector, this clinical study will continue the observation period after the completion of the clinical trial.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Not applicable
Assessment
Primary outcomes
Rate of change in visual acuity (logMAR equivalent)
Key secondary outcomes
1. Rate of change in mean retinal sensitivity at the 12 center points in HFA10-2 static perimetry tests
2. Rate of change in mean retinal sensitivity at the 4 center points in HFA10-2 static perimetry tests
3. Percentage of serious adverse events for which a causal relationship with DVC1-0401 cannot be ruled out
Base
Study type
Observational
Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
Interventions/Control_2
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
| 70 | years-old | >= |
Gender
Male and Female
Key inclusion criteria
Patients with retinitis pigmentosa who received DVC1-0401 in the "Phase I/IIa Investigator-Initiated Trial of Neuroprotective Gene Therapy for Retinitis Pigmentosa by Subretinal Administration of DVC1-0401"
Key exclusion criteria
Patients whom the investigator determines are not appropriate as research subjects
Target sample size
12
Research contact person
Name of lead principal investigator
| 1st name | Yusuke |
| Middle name | |
| Last name | Murakami |
Organization
Kyushu University Hospital
Division name
Department of Ophthalmology
Zip code
812-8582
Address
Maidashi 3-1-1, Higashi-ku, Fukuoka, Japan
TEL
+81-92-642-5648
murakami.yusuke.407@m.kyushu-u.ac.jp
Public contact
Name of contact person
| 1st name | Yusuke |
| Middle name | |
| Last name | Murakami |
Organization
Kyushu University Hospital
Division name
Department of Ophthalmology
Zip code
812-8582
Address
Maidashi 3-1-1, Higashi-ku, Fukuoka, Japan
TEL
+81-92-642-5648
Homepage URL
https://www.eye.med.kyushu-u.ac.jp/patient/clinicaltrial/index.html
murakami.yusuke.407@m.kyushu-u.ac.jp
Sponsor or person
Institute
Kyushu University
Institute
Department
Personal name
Funding Source
Organization
Others
Organization
Division
Category of Funding Organization
Other
Nationality of Funding Organization
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
IRB for observational clinical study in medical area of Kyushu University
Address
Maidashi 3-1-1, HIgashi-ku, Fukuoka
Tel
092-642-6254
ijkseimei@jimu.kyushu-u.ac.jp
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
| 2023 | Year | 01 | Month | 04 | 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
10
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
| 2020 | Year | 09 | Month | 09 | Day |
Date of IRB
| 2020 | Year | 09 | Month | 09 | Day |
Anticipated trial start date
| 2020 | Year | 11 | Month | 12 | Day |
Last follow-up date
| 2025 | Year | 03 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
| 2024 | Year | 03 | Month | 06 | Day |
Date analysis concluded
Other
Other related information
NA
Management information
Registered date
| 2022 | Year | 12 | Month | 19 | Day |
Last modified on
| 2024 | Year | 04 | Month | 08 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000056748
| Research Plan | |
|---|---|
| Registered date | File name |
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| Registered date | File name |
| Research case data | |
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