SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew) (NCT06461286) | Clinical Trial Compass
Active — Not RecruitingPhase 1
SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)
Australia18 participantsStarted 2024-10-31
Plain-language summary
A First-in-Human multi-centre, prospective, Phase1a, Single Ascending Dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Must have given written informed consent before any study-related activities are carried out and must be able to understand the full nature and purpose of the trial, including possible risks and adverse effects;
✓. Adult males and females, aged 18 years and above at screening;
✓. Body mass index (BMI) ≥18.0 and ≤32.0 kg/m2, with a body weight ≤ 100kg at screening;
✓. Have a molecular (genetic) diagnosis of OPA1 mutation at screening;
✓. Have a clinical diagnosis of OPA1 mutation-associated ADOA at screening;
✓. Participants with BCVA of between 20/40 (70 ETDRS letters) and 20/160 (39-43 ETDRS letters). If both eyes meet this eligibility criteria, the eye with worse BCVA will be selected as the study eye and the other eye will be designated as the "fellow eye";
✓. Medically healthy (in the opinion of the Investigator), as determined by pre-study medical history, and without clinically significant abnormalities including:
✓. Physical examination without any clinically relevant findings;
Exclusion criteria
✕. Participants has a known allergy to PYC-001 or any of its excipients;
✕. Demonstrated clinically significant co-morbidities, which, in the opinion of the Investigator, would interfere with the volunteer's ability to participate in the trial and/or confound study outcomes;
✕
What they're measuring
1
Incidence, type, severity and relationship of ocular treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (SAEs) in the study eye
Timeframe: 24 weeks
2
Incidence, type, severity and relationship of ocular treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (SAEs) in the study eye
Timeframe: 48 weeks
3
Incidence, type, severity and relationship of ocular TEAEs, and treatment-emergent SAEs in the fellow eye
Timeframe: 24 weeks
4
Incidence, type, severity and relationship of ocular TEAEs, and treatment-emergent SAEs in the fellow eye
Timeframe: 48 weeks
5
Incidence, type, severity and relationship of non-ocular TEAEs, and treatment-emergent SAEs
Timeframe: 24 weeks
6
Incidence, type, severity and relationship of non-ocular TEAEs, and treatment-emergent SAEs
Timeframe: 48 weeks
7
Change from baseline for vital signs measurements (heart rate [HR], systolic and diastolic blood pressure [BP], tympanic temperature, respiratory rate [RR])
. Females who are breastfeeding or planning to breastfeed;
✕. Have mutations in genes that cause ADOA, other than OPA1 (for example in case of dominant negative ADOA) and ADOA Plus;
✕. Have received any prior cell or gene therapy for a retinal condition;
✕. Within 3 months prior to study Day -1, have undergone any vitreoretinal surgery (scleral buckle, pars plana vitrectomy, retrieval of a dropped nucleus or intraocular lens, radial optic neurotomy, sheathotomy, cyclodestructive procedures or multiple filtration surgeries \[2 or more\]) or any other ocular surgery;
✕. Within 3 months prior to study Day -1, have placement of an Ozurdex® implant;
✕. Within 3 months prior to study Day -1 have placement of Retisert® of Iluvien® implants;
8
Change from baseline for 12-lead electrocardiogram (ECG) parameters
Timeframe: 48 weeks
9
Change from baseline for clinical laboratory results - hematology
Timeframe: 48 weeks
10
Change from baseline for clinical laboratory results - clinical chemistry
Timeframe: 48 weeks
11
Change from baseline for clinical laboratory results - coagulation
Timeframe: 48 weeks
12
Change from baseline for clinical laboratory results - urinalysis