This study aims to gather safety data and determine the optimal dosing regimen for PYC-001 in participants with confirmed OPA1 mutation-associated ADOA. Approximately 18 participants from Australia, New Zealand, and other APAC countries are expected to be enrolled, depending on safety review committee (SRC) throughout the course of the study.
Participants may be assigned to any of the following:
1. A single 60ug dose of PYC-001 2. Three doses of 10ug PYC-001 at an interval of 8 weeks 3. Three doses of 10ug PYC-001 at an interval of 12 weeks 4. Three doses of 30ug PYC-001 at an interval of 8 weeks 5. Three doses of 30ug PYC-001 at an interval of 12 weeks
Following completion of the 4 week safety review of the single 60ug of PYC-001 cohort, and if the 60 μg dose level is deemed safe by the SRC, the following cohorts will also be available: 6. Three doses of 60ug PYC-001 at an interval of 12 weeks
Must give written informed consent before any study-related activity is carried out
Adult males and females, aged 18 years and above at screening;
Body mass index ≥18.0 and ≤35.0 kg/m2
Have a recent (within five years) genetic diagnosis of OPA1 mutation-associated (haploinsufficiency) ADOA and/or confirmed diagnosis during pre-screening or screening, as determined by the PI.
Participant has a known allergy to PYC-001 or any of its excipients;
Demonstrated clinically significant co-morbidities, which, in the opinion of the PI, would interfere with the participant's ability to participate in the study and/or confound study outcomes;
Females who are breastfeeding or planning to breastfeed;
Based on recent genetic testing, the participant has mutations in genes that cause ADOA, other than OPA1 (for example in case of dominant negative ADOA and ADOA Plus) or has other pathological variants that result in an ADOA-like optic atrophic phenotype or other pathologic genetic findings indicating presence of additional confounding ocular diseases based on comprehensive genetic screening.
Parallel
PYC-001
DrugPYC-001 is a peptide-phosphorodiamidate morpholino oligonucleotide administered intravitreally
6
Treatment groupsSee each treatment group below.
[All Cohorts] Number of participants experiencing treatment emergent adverse events
The incidence, type, severity, and relationship of treatment emergent ocular and non-ocular adverse events and treatment-emergent Serious Adverse Events will be recorded. An AE is any untoward medical occurrence in a clinical study participant that either occurs during the study or, if present predose, worsens during the study, and which does not necessarily have to have a causal relationship with the study treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease associated with study participation, whether or not considered related to the study treatment.
[All Cohorts] Changes from baseline in vital signs (heart rate)
[All Cohorts] Changes from baseline in vital signs (systolic and diastolic blood pressure)
[All Cohorts] Changes from baseline in vital signs (tympanic temperature)
[All Cohorts] Change from Baseline in Best Corrected Visual Acuity (BCVA)/ High Contrast Visual Acuity (HCVA) and Low Contrast Visual Acuity (LCVA) scores
Early Treatment Diabetic Retinopathy Study (ETDRS) optotypes used, letter score will be captured for BCVA/HCVa and LCVA.
[All Cohorts] Change from Baseline in Visual field sensitivity by photopic static perimetry
[All Cohorts] Change from Baseline in posterior eye health by fundus examination using ultrawide fundoscopy
[All Cohorts] Change from Baseline in Color vision
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