An investigator-initiated trial of DVC1-0401 as gene therapy for retinitis pigmentosa

JRCT ID: jRCT2073180024

Registered date:22/02/2019

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Retinitis pigmentosa
• Date of first enrollment: 30/05/2019
• Target sample size: 12
• Study type: Interventional
• Intervention(s): Subretinal injection of low-dose DCV1-0401 (Simian immunodeficiency virus vector expressing human pigment epithelium-derived factor (hPEDF))

Outcome(s)

• Primary Outcome: Incidence of adverse events

Secondary Outcome

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

Key inclusion & exclusion criteria

• Age minimum: >= 40age old
• Age maximum: <= 70age old
• Gender: Both

Include 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.)

Exclude 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

Related Information

• Primary Sponsor: Ikeda Yasuhiro
• Source(s) of Monetary Support: AMED
• Secondary ID(s): UMIN000034081