X-Linked Retinitis Pigmentosa
Conditions
Brief summary
A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).
Interventions
GENETICFT-002
Comparison of different dosages of FT-002
Sponsors
Eye & ENT Hospital of Fudan University
Peking Union Medical College Hospital
Henan Provincial People's Hospital
Frontera Therapeutics
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
X-linked retinitis pigmentosa patients
Eligibility
Sex/Gender
MALE
Age
8 Years to 45 Years
Healthy volunteers
No
Inclusion criteria
1.Subjects that are willing and able to follow study procedures; 2.Males aged 8-45 years old at the time of signing the Informed Consent Form; 4.Subjects who are confirmed with variants of RPGR ;
Exclusion criteria
1.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 |
|---|---|---|
| Incidence of AEs | from FT-002 administration through up to 1 years | Number and proportion of Adverse Events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in visual function | from FT-002 administration through up to 1 years | Change in retinal function as assessed by mean retinal sensitivity within the 30-degree visual field |
| Change in retinal structure as assessed by Optical Coherence Tomography | from FT-002 administration through up to 1 years | Change in Outer Nuclear Layer Thickness from baseline as assessed by Optical Coherence Tomography |
Countries
China
Contacts
Primary ContactXinyan Li
Backup ContactMinghui Xue
Outcome results
None listed