UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000034081 |
|---|---|
| Receipt number | R000038854 |
| Scientific Title | An investigator-initiated phase I/IIa trial of subretinal injection of DVC1-0401 as neuroprotective gene therapy for retinitis pigmentosa |
| Date of disclosure of the study information | 2018/11/01 |
| Last modified on | 2022/09/06 10:22:07 |
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Basic information
Public title
An investigator-initiated phase I/IIa trial of subretinal injection of DVC1-0401 as neuroprotective gene therapy for retinitis pigmentosa
Acronym
An investigator-initiated trial of DVC1-0401 as gene therapy for retinitis pigmentosa
Scientific Title
An investigator-initiated phase I/IIa trial of subretinal injection of DVC1-0401 as neuroprotective gene therapy for retinitis pigmentosa
Scientific Title:Acronym
An investigator-initiated trial of DVC1-0401 as gene therapy for retinitis pigmentosa
Region
| Japan |
Condition
Condition
Retinitis pigmentosa
Classification by specialty
| Ophthalmology |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
For patients with retinitis pigmentosa accompanied with decreased central retinal sensitivity,
1) To examine safety of subretinal injection of DVC1-0401
2) To explore the inhibitory effect on declining visual function
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Explanatory
Developmental phase
Phase I,II
Assessment
Primary outcomes
Incidence of adverse events
Key secondary outcomes
1) Rate of change of the mean retinal sensitivity (central 12 points)
: Primary outcome measure of efficiency
2) Rate of change of the mean retinal sensitivity (central 4 points)
: Secondary outcome measure of efficiency-1
3) Rate of change of the visual acuity (logMAR converted value)
: Secondary outcome measure of efficiency-2
4) Amount of hPEDF protein in the aqueous humor
: Exploratory outcome measure
Base
Study type
Interventional
Study design
Basic design
Parallel
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
No treatment
Stratification
NO
Dynamic allocation
NO
Institution consideration
Institution is not considered as adjustment factor.
Blocking
NO
Concealment
No need to know
Intervention
No. of arms
3
Purpose of intervention
Treatment
Type of intervention
| Gene |
Interventions/Control_1
Subretinal injection of low-dose DCV1-0401 (Simian immunodeficiency virus vector expressing human pigment epithelium-derived factor (hPEDF))
Interventions/Control_2
Subretinal injection of medium-dose DCV1-0401
Interventions/Control_3
Subretinal injection of high-dose DCV1-0401
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
1) Subjects with retinitis pigmentosa diagnosed by two or more ophthalmic specialists
2) 40 years and above, and 70 years or younger
3) The mean retinal sensitivity measured by Humphrey perimeter (10-2 program) is less than 30 dB (central 4 points)
4) Interocular difference in visual acuity is less than or equal to 0.21 (logMAR converted value)
5) Interocular difference in visual field is less than or equal to twofold (Goldmann perimeter V-4e)
6) Interocular difference in the MD value measured by Humphrey perimeter (10-2 program) is less than or equal to 5 dB
7) Interocular difference in central retinal thickness measured by optical coherence tomography is less than or equal to 50 um
8) No interocular difference in the structure of outer retinal layer (ellipsoid zone) observed by optical coherence tomography
9) In case of subjects who are followed up over three years, interocular difference in the rate of change of visual acuity (logMAR converted value) and the MD value measured by Humphrey perimeter (10-2 program) is less than or equal to twofold
10) Difference in the mean retinal sensitivity measured in repeating tests using Humphrey perimeter (10-2 program) within 60 days before administration is binocularly less than or equal to 3 dB. If results from first and second tests don't fulfill the above criteria, the result from third test performed within 7 days from the second test is acceptable.
11) No significant pigmentation in ophthalmoscopic examination
12) Subjects who can give consent in writing by oneself (If subjects can not read the document because of reduction in visual function, both oral consent of the subject and signature of witness are necessary.)
Key exclusion criteria
1) HIV antibody-positive
2) Decimal visual acuities of both eyes are less than 0.1, or lateral or bilateral blindness
3) Cone-rod dystrophy
4) Interocular difference in the condition of lens
5) Co-existence of glaucoma or ocular hypertension (intraocular pressure is more than or equal to 22 mmHg)
6) Co-existence of uveitis or optic neuritis
7) Retinal or subretinal pathological change observed by fundus examination (including fluorescein fundus angiography, laser scanning fundus photography), that is independent of retinitis pigmentosa
8) Visual field measured by Goldmann perimeter V-4e is less than 10 degree at the center area of vision
9) Loss or massive structural irregularity of the layer of retinal pigment epithelium on OCT (optical coherence tomography) at the supposed site of administration
10) Having severe allergy or previous history
11) Receiving chronic hemodialysis therapy
12) Severe heart dysfunction or heart failure
13) Sever hepatic dysfunction or liver cirrhosis
14) Active inflammatory diseases
15) History of cerebral hemorrhage or infarction within 6 months before agreement
16) Hematopoietic disorders
17) Alcoholism and/or drug dependence
18) Female subjects with pregnant or doubt of pregnancy. Breast-feeding female.
19) Subjects who are diagnosed with cancer or suspected to have cancer. History of treatment of malignancies within 5 years.
20) Disapprove of birth control during at least 12 months after the administration
21) Administration of Helenien, Unoprostone, and/or Ca antagonist intended for treatment of eye disease within 30 days before agreement
22) Taking the antiviral drug at the registration
23) Plan of operation other than administration of the investigational drug during this clinical trial
24) Entry into other clinical trial and/or clinical study, or history of entry within 6 months before agreement
25) Others: Subjects who are judged to be inappropriate by principal investigator or subinvestigator
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
8128582
Address
3-1-1 Maidashi, Higashi-ku, Fukuoka, Japan
TEL
092-642-5648
ymuraka3@pathol1.med.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
8128582
Address
3-1-1 Maidashi, Higashi-ku, Fukuoka, Japan
TEL
092-642-5648
Homepage URL
ymuraka3@pathol1.med.kyushu-u.ac.jp
Sponsor or person
Institute
Kyushu University Hospital
Institute
Department
Personal name
Funding Source
Organization
AMED
Organization
Division
Category of Funding Organization
Japanese Governmental office
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
University of Miyazaki Hospital
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Kyushu University Hospital IRB
Address
3-1-1, Maidashi, Higashi-ku
Tel
+81926425774
bysirb@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
九州大学病院（福岡県）、宮崎大学医学部附属病院（宮崎県）
Other administrative information
Date of disclosure of the study information
| 2018 | Year | 11 | Month | 01 | 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
12
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
No longer recruiting
Date of protocol fixation
| 2018 | Year | 07 | Month | 31 | Day |
Date of IRB
| 2018 | Year | 09 | Month | 21 | Day |
Anticipated trial start date
| 2019 | Year | 02 | Month | 14 | Day |
Last follow-up date
| 2023 | Year | 08 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2018 | Year | 09 | Month | 10 | Day |
Last modified on
| 2022 | Year | 09 | Month | 06 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000038854
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