X-Linked Retinitis Pigmentosa (XLRP)
Conditions
Keywords
X-Linked Retinitis Pigmentosa (XLRP), FT-002
Brief summary
The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation phase) and Phase II (dose expansion phase).
Interventions
GENETICFT-002
Intraocular injection of a single dose
Sponsors
Frontera Therapeutics
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
8 Years to 45 Years
Healthy volunteers
No
Inclusion criteria
* Subjects that are willing and able to follow study procedures including scheduled visits, treatment plan, and laboratory tests, and sign a written informed consent form; * Age: Phase I dose escalation stage, 18-45 years old male (including boundary value) at the time of signing the ICF; Phase II dose extension stage, males 8-45 years old (including boundary values) at the time of signing the ICF; * Clinically diagnosed XLRP, the main symptoms include but are not limited to night blindness, visual field loss, vision loss, etc.;
Exclusion criteria
* Have other retinal degenerative diseases, such as retinal degeneration caused by other known Inherited retinal disease gene variants or previously received an gene therapy product.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To evaluate the safety and tolerance of FT-002 | from FT-002 administration through up to 1 years | Incidence and severity of AEs |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To evaluate the efficacy of FT-002 | from FT-002 administration through up to 1 years | Changes in visual sensitivity /FST/BCVAfrom baseline |
Countries
China
Contacts
Primary ContactXinyan Li
Backup ContactMinghui Xue
Outcome results
None listed