UGX202 Injection in Patients With Advanced Retinitis Pigmentosa
ConditionRetinitis Pigmentosa (RP)
Trial statusNot yet recruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorSuzhou UgeneX Therapeutics Co., Ltd.
About this trial
The primary objective of this clinical trial is to evaluate the safety and tolerability of a single intravitreal injection of the gene therapy drug UGX202 in patients with advanced RP. The secondary objective is, to assess the preliminary efficacy of a single intravitreal injection of the gene therapy drug UGX202 in treating patients with advanced RP.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Provide written informed consent form (ICF).
Age ≥18 years at ICF signing.
Diagnosed as non-syndromic RP；
BCVA > logMAR 1.9 (assessed by FrACT) in the study eye.
Disqualifiers
Prior gene therapy in either eye.
Received any interventional investigational drug within 90 days prior to screening.
Any Study eye disease or systemic disease judged by the investigator to affect visual function assessment.
Hypersensitivity to corticosteroids, intolerance to corticosteroid regimen, active concurrent infection contraindicating treatment.
Trial design
Design model
Sequential
Treatments tested in this trial
UGX202 injection
GeneticComparison of different dosages of UGX202
Treatment groups
6 Participants
are divided into 2 treatment groupsGroup A: Low dose of UGX202 groupExperimental treatment 1 intervention
Group B: High dose of UGX202 groupExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Incidence of adverse events and serious adverse events
From the time of administration of UGX202 injection until the 52nd week, based on the topical and systemic safety data, the incidence rates of AEs, TEAEs during treatment, TRAEs related to the study drug, TRAEs related to the study procedures, SAEs, TRSAEs related to the study drug, and TRSAEs related to the study procedures during the study period were summarized by the investigators, and the correlations between AEs and the study drug and study procedures were determined.
Time frame
baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 522
The average change in IOP
The average change in IOP of the study eyes and non-study eyes from after treatment to the 52nd week compared to the baseline. The IOP was measured three times consecutively at each visit and the average value was taken.
Time frame
baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52Secondary outcomes
1
The changes in BCVA
The changes in BCVA of the study eyes and non-study eyes at each follow-up visit compared to the baseline were evaluated. BCVA was assessed using the Freiburg Vision Test (FrACT) system. If the subjects had no light perception at the baseline: the proportion of subjects whose BCVA improved to having light perception after treatment was evaluated, and the change in their vision compared to the baseline was assessed;
Time frame
baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 522
The changes in the average stimulus threshold
The changes in the average stimulus threshold measured by the full-field stimulus threshold test (FST) of the study eyes and/or non-study eyes at each follow-up visit compared to the baseline;
Time frame
baseline to Week 4, Week 12, Week 24, Week 523
The changes in the visual function questionnaire (VFQ-25) scores
The changes in the visual function questionnaire (VFQ-25) scores of the subjects at each follow-up visit after the treatment compared to the baseline.
Time frame
baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52Other outcomes
1
The change in Latency of N2, latency of P2, N2-P2 amplitude difference will be evaluated in VEP
Latency of N2, latency of P2, N2-P2 amplitude difference will be evaluated in VEP both in the study eye and non-study eyes at each visits compared with baseline.
Time frame
Baseline, week4, week 8, week 12, week 24, week 52/EoS2
The change in dark-adapted 0.01 ERG, dark- adapted 3.0 ERG, dark-adapted 30.0 ERG and light-adapted 3.0 ERG
Dark-adapted 0.01 ERG, dark- adapted 3.0 ERG, dark-adapted 30.0 ERG and light-adapted 3.0 ERG will be evaluated in electroretinogram both in the study eye and non-study eyes at each visits compared with baseline.
Time frame
Baseline, week4, week 8, week 12, week 24, week 52/EoS3
Change of mean defect (MD) in the visual fields
Change of mean defect (MD) in the visual fields of the study eyes and non-study eyes
Time frame
Baseline, week 24, week 52/EoS4
Change of visual field index (VFI) in the visual fields
Change of visual field index (VFI) in the visual fields of the study eyes and non-study eyes
Time frame
Baseline, week 24, week 52/EoSSponsors and contacts
Click on the lead sponsor to view all of their trials.
Suzhou UgeneX Therapeutics Co., Ltd.
Lead sponsor
Eye & ENT Hospital of Fudan University
Collaborator
Source ClinicalTrials.gov
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