Actively Recruiting
Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)
Led by PYC Therapeutics · Updated on 2026-04-08
18
Participants Needed
3
Research Sites
117 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
CONDITIONS
Official Title
Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Must give written informed consent before any study-related activity
- Adult males and females aged 18 years and above at screening
- Body mass index between 18.0 and 35.0 kg/m2
- Recent (within five years) or confirmed diagnosis of OPA1 mutation-associated Autosomal Dominant Optic Atrophy
- Treatment-na�ve with best-corrected visual acuity between 20/40 and 20/200 in the study eye
- Mild to moderate visual field and retinal nerve fiber layer loss in the study eye
- Medically healthy as determined by the investigator
- Female participants must be non-childbearing or have negative pregnancy tests and agree to use contraception
- Male participants must agree not to donate sperm and use contraception if engaging with partners who could become pregnant
- Willing and able to comply with all study assessments and protocol requirements
You will not qualify if you...
- Allergy to PYC-001 or its excipients
- Significant co-morbidities interfering with study participation or outcomes
- Females who are breastfeeding or planning to breastfeed
- Genetic mutations causing ADOA other than OPA1 or other confounding ocular diseases
- Prior cell or gene therapy for retinal conditions (except specific study participation)
- Vitreoretinal or other ocular surgery in the study eye within 3 months
- Placement of Ozurdex implant within 3 months or Retisert/Iluvien implants within 3 years
- Ocular media opacity or poor pupil dilation preventing quality eye exams
- Macular edema requiring treatment in the study eye
- History of recurrent uveitis or active ocular inflammation
- Use of investigational or certain over-the-counter drugs within 30 days prior to screening
- Recent history of excessive recreational drug or alcohol use
- Positive alcohol breath or urine drug tests at screening and study days
- Any retinal pathology other than ADOA or conditions making participant unsuitable
- Illness or pathology affecting visual function or current ocular infection
- Positive tests for HIV, hepatitis B or C
- Abnormal clinical chemistry, hematology, coagulation, or urinalysis at screening
AI-Screening
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Trial Site Locations
Total: 3 locations
1
Save Sight Institute - Sydney Eye Hospital
Sydney, New South Wales, Australia, 2000
Actively Recruiting
2
Cerulea Clinical Trials
East Melbourne, Australia
Actively Recruiting
3
Retina Specialists
Auckland, New Zealand, 1052
Actively Recruiting
Research Team
S
Sreenivasu Mudumba
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
6
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