OPA1 Gene MutationPhase 1NCT06461286
SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)
A Phase 1 study testing PYC-001 in people with OPA1 Gene Mutation and Autosomal Dominant Optic Atrophy, run by PYC Therapeutics at 2 sites in Australia.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy, Hereditary Optic Atrophies
- What is being tested?
- PYC-001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 2 sitesAustralia
- Study dates
- 31 Oct 2024to Nov 2029
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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";
Show all 23Show fewer
- 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;
- Systolic BP in the range of 90 to 160mmHg and diastolic BP in the range of 50 to 95 mmHg after 5 minutes in supine of semi-supine position;
- Heart rate in the range of 45 to 100 bpm after 5 minutes rest in supine or semi-supine position;
- Body temperature (tympanic), between 35.5°C and 37.7°C;
- ECG without clinically significant abnormalities including QT interval corrected for Fredericia (QTcF) < 450 msec for male subjects and < 470 msec for female subjects;
- No clinically significant findings in clinical chemistry, hematology, coagulation and urinalysis tests at screening.
- Female volunteers must be of non-child-bearing potential, i.e., surgically sterilized (hysterectomy, bilateral salpingectomy, bilateral oophorectomy at least 6 weeks before the screening visit) or postmenopausal (where postmenopausal is defined as no menses for 12 months without an alternative medical cause and a follicle stimulating hormone [FSH] level consistent with postmenopausal status, per local laboratory guidelines). Females receiving hormone replacement therapy (HRT) may be considered for inclusion if the need for HRT is for no other medical reason than to treat symptoms associated with menopause. If female participants are of child-bearing potential, they must:
- Have a negative pregnancy test at the screening visit, on study Day -1 and study Day 1 prior to dosing;
- Agree not to attempt to become pregnant or donate ova from signing the consent form until at least 130 days after IVT administration of PYC-001 and at least 30 days after the final dose of ANX776;
- Agree to use adequate contraception (defined as use of a condom by the male partner combined with use of a highly effective method of contraception from one month prior to screening until at least 30 days after the final dose of ANX776 (study Day 336), if not exclusively in a same-sex relationship or abstinent as a committed lifestyle.
- Male volunteers must:
- Agree not to donate sperm from signing the consent form until at least 190 days after IVT administration of PYC-001 and at least 90 days after the final dose of ANX776;
- If engaging in sexual intercourse with a female partner who could become pregnant, agree to use adequate contraception (defined as use of a condom combined with use of a highly effective method of contraception) from signing the consent form until at least 190 days after IVT administration of PYC-001 and at least 90 days after the final dose of ANX776;
- If engaging in sexual intercourse with a female partner who is not of child-bearing potential or a same-sex partner, agree to use a condom from signing the consent form until at least 190 days after IVT administration of PYC-001 and at least 90 days after the final dose of ANX776;
- Have suitable venous access for blood sampling
- Willing and able to comply with all study assessments and adhere to the protocol schedule and restrictions.
Who cannot join
- 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;
- 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;
Show all 15Show fewer
- 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;
- Have ocular media opacity or poor pupillary dilation prohibiting quality ophthalmic evaluation or photography, as assessed by the Investigator;
- Macular edema (intraretinal, sub-retinal or other fluid) requiring regular treatment at a frequency of less than every 6 weeks; macular edema must be stable for at least 3 months prior to screening. Note, the above conditions will be permitted for inclusion if, in the opinion of the Investigator and in consultation with the SMM, they will not interfere with study evaluations, or have resolved by study Day -1 prior to dose administration;
- Have used any investigational drug or device within 90 days or 5 estimated half-lives of the investigational drug or device (whichever is longer) prior to study Day -1, or plan to participate in another study of a drug or device during the study period. Participation in observational trials is allowed based on Investigator discretion and consultation with the Sponsor's Medical Representative;
- Have a recent history (< 6 months) of or current excessive recreational drug or alcohol use, in the opinion of the Investigator. Note: excessive alcohol use is defined as regular consumption of > 10 standard drinks per week or > 4 standard drinks per day, where 1 standard drink is defined as 10 grams of pure alcohol;
- Positive alcohol breath test as assessed at screening, and on study Day -1 and study Day 1;
- Positive urine drugs of abuse as assessed at screening and on study Day -1 and study Day 1;
- Any retinal pathology other than ADOA or any other condition or prior therapy that in the opinion of the Investigator would make the volunteer unsuitable for this study, including inability to cooperate fully with the requirements of the study protocol or likelihood of noncompliance with any study requirements.
Trial locations and contacts
2 sites in 1 country.
Australia2 sites
Center for Eye Research Australia (CERA)
East Melbourne, Victoria, 3002
Save Sight Institute - Sydney Eye Hospital
Sydney, New South Wales, 2000
About this study
Official title: A Phase 1a Open-Label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered PYC-001 in Participants With Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy
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.
Read the detailed description
This is a First-In-Human multi-centre, prospective, Phase 1a, single ascending dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.
Following confirmation of eligibility on Day-1, one(1) eye will be selected for study participation (the "study eye"), and the other eye will be designated as the "fellow eye". Selection of the "Study eye" will be the eye with worse vision. If both eyes have similar visual acuity and visual field information, the choice of study eye will be determined at the discretion of the Investigator in consultation with the participant.
Each eligible participant will receive a single intravitreal (IVT) injection of PYC-001 in their study eye on Day 1, and will be monitored for dose limiting toxicities (DLTs) for 4 weeks. The study will use a 3+3 escalation scheme and will involve up to three PYC-001 dose groups. Cohorts of 3 participants will be initially enrolled at each dose level, and then expanded to 6 participants per cohort in the event of a DLT or any >> Grade 2 adverse events (AEs) that are deemed related to study treatment. Within each cohort, dosing will be staggered with a 7-day interval between the first participant receiving investigational product (IP), PYC-001, and the remaining 2 participants receiving IP.
If >> 2 DLTs are observed in 6 participants in any cohort, and the previous lower dose cohort was not previously expanded to 6 participants per the 3+3 design rules, the lower dose cohort will be expanded to include evaluation of 6 participants. If this lower dose level has << 1 DLTs it will be considered the maximum tolerated dose (MTD). Alternatively, a dose half-way between the previous lower dose, and the dose with >> 2 participants showing DLTs may be selected for evaluation.
If no DLTs or any >> Grade 2 AEs are observed in the first 3 participants treated within a cohort, then escalation to the next dose cohort can proceed following review of the collated 4-week safety data by the safety review committee (SRC).
- Conditions
- OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy, Hereditary Optic Atrophies, Kjer Optic Atrophy
- Being tested
- PYC-001
- Medical subject headings
- Optic Atrophy, Autosomal Dominant, Optic Atrophies, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PYC-001Drug
Single Group Assignment
| Study group | Role | What they receive |
|---|---|---|
| Single arm dose escalation study of PYC-001 | Experimental | Drug: PYC-001
Phase 1a Open-Label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered PYC-001 in participants with Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy Receives: Drug: PYC-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, type, severity and relationship of ocular treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (SAEs) in the study eye
Measured: 24 weeks
Incidence, type, severity and relationship of ocular treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (SAEs) in the study eye
Measured: 48 weeks
Incidence, type, severity and relationship of ocular TEAEs, and treatment-emergent SAEs in the fellow eye
Measured: 24 weeks
Incidence, type, severity and relationship of ocular TEAEs, and treatment-emergent SAEs in the fellow eye
Measured: 48 weeks
Incidence, type, severity and relationship of non-ocular TEAEs, and treatment-emergent SAEs
Measured: 24 weeks
Incidence, type, severity and relationship of non-ocular TEAEs, and treatment-emergent SAEs
Measured: 48 weeks
Change from baseline for vital signs measurements (heart rate [HR], systolic and diastolic blood pressure [BP], tympanic temperature, respiratory rate [RR])
Measured: 48 weeks
Change from baseline for 12-lead electrocardiogram (ECG) parameters
Twelve-lead ECG (including but not limited to the measurements of ventricular HR, PR interval, QRS duration, QT interval and QtcF) will be performed. ECGs will be performed in triplicate at screening only with each replicate separated by at least 1 minute and the full set of triplicates completed within 5 minutes. The mean value for the triplicate will be utilized. Single ECGs will be collected at all other timepoints.
Measured: 48 weeks
Change from baseline for clinical laboratory results - hematology
Hematology Screening parameters: Hematocrit, Hemoglobin, Mean Corpuscular hemoglobin, mean corpuscular hemoglobin concentration, mean corpuscular volume, mean platelet volume, packed cell volume, platelet count, red blood cell count, reticulocyte count, white blood cell count with differential neutrophil count, eosinophil count, basophil count, lymphocyte count, monocyte count.
Measured: 48 weeks
Change from baseline for clinical laboratory results - clinical chemistry
Clinical Chemistry screening parameters: Albumin, alkaline phosphatase, alanine aminotransferase, amylase, anion gap, aspartate aminotransferase, bicarbonate, calcium, ionized calcium, chloride, conjugated (direct) and unconjugated bilirubin, creatinine, eGFR, Creatinine kinase, Gamma glutamyl transferase, Globulin, Glucose, High density lipoprotein, Lactate dehydrogenase, Lipase, Low density lipoprotein, Magnesium, Phosphate, Potassium, Sodium, Total bilirubin, Total cholesterol, Total protein, Triglycerides, Urate, Urea
Measured: 48 weeks
Change from baseline for clinical laboratory results - coagulation
Coagulation screening parameters: Activated partial thromboplastin time, International normalized ratio/Prothrombin time, Fibrinogen
Measured: 48 weeks
Change from baseline for clinical laboratory results - urinalysis
Urinalysis screening parameters: Bilirubin, Blood, Glucose, Ketones, Leukocyte esterase, Nitrites, pH, Protein, Specific gravity, Urobilinogen
Measured: 48 weeks
Secondary outcomes (11)
Change from baseline for Best-corrected visual acuity (BCVA) letter score using Early Treatment of Diabetic Retinopathy Study(ETDRS)
Measured: Week 4, Week 12, Week 24, Week 48
Change from baseline for Low contrast visual acuity (LCVA)
Measured: Week 4, Week 12, Week 24, Week 48
Change from baseline for Perimetry
Measured: Week 4, Week 12, Week 24, Week 48
Change from baseline for posterior eye healthy by fundus examination, using ultrawide funduscopy
Measured: Week 4, Week 12, Week 24, Week 48
Change from baseline for color vision by Hardy Rand Rittler test
Measured: Week 4, Week 12, Week 24, Week 48
Change from baseline for contrast sensitivity by Pelli Robson Chart
Measured: Week 4, Week 12, Week 24, Week 48
Change from baseline for visual field sensitivity by Static Perimetry
Measured: Week 4, Week 12, Week 24, Week 48
Change from baseline for Multifocal Visual Evoked Potential (mfVEP)
Measured: Week 4, Week 12, Week 24, Week 48
Change from baseline for retinal nerve fiber layer (RNFL) and Ganglion Cell Layer (GCL) thickness by Spectral domain optical coherence tomography (SD-OCT)
Measured: Week 4, Week 12, Week 24, Week 48
Change from baseline for mitochondrial function test by flavoprotein fluorescence (FPF) analyzer (Ocumet Beacon)
Measured: Week 4, Week 12, Week 24, Week 48
Change from baseline for Detection of Apoptosis in Retinal Cells (DARC)
Measured: Week 4, Week 12, Week 24, Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2024
- Study start31 Oct 2024
- Primary completionOct 2029 (estimated)
- Study completionNov 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for OPA1 Gene MutationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- PYC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Aishwarya Kundu, Study chair, PYC Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06461286
- Sponsor's protocol ID
- PYC-001-101
- Status last verified
- Jan 2026
- First submitted
- 5 Jun 2024
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1a Open-Label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered PYC-001 in Participants With Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy
- ClinicalTrials.gov ID
- NCT06461286
- Acronym / sponsor ID
- PYC-001-101
- Conditions
- OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy, Hereditary Optic Atrophies, Kjer Optic Atrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- PYC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 31 Oct 2024 (actual)
- Primary completion
- Oct 2029 (estimated)
- Study completion
- Nov 2029 (estimated)
- First posted
- 14 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a First-In-Human multi-centre, prospective, Phase 1a, single ascending dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.
Following confirmation of eligibility on Day-1, one(1) eye will be selected for study participation (the "study eye"), and the other eye will be designated as the "fellow eye". Selection of the "Study eye" will be the eye with worse vision. If both eyes have similar visual acuity and visual field information, the choice of study eye will be determined at the discretion of the Investigator in consultation with the participant.
Each eligible participant will receive a single intravitreal (IVT) injection of PYC-001 in their study eye on Day 1, and will be monitored for dose limiting toxicities (DLTs) for 4 weeks. The study will use a 3+3 escalation scheme and will involve up to three PYC-001 dose groups. Cohorts of 3 participants will be initially enrolled at each dose level, and then expanded to 6 participants per cohort in the event of a DLT or any >> Grade 2 adverse events (AEs) that are deemed related to study treatment. Within each cohort, dosing will be staggered with a 7-day interval between the first participant receiving investigational product (IP), PYC-001, and the remaining 2 participants receiving IP.
If >> 2 DLTs are observed in 6 participants in any cohort, and the previous lower dose cohort was not previously expanded to 6 participants per the 3+3 design rules, the lower dose cohort will be expanded to include evaluation of 6 participants. If this lower dose level has << 1 DLTs it will be considered the maximum tolerated dose (MTD). Alternatively, a dose half-way between the previous lower dose, and the dose with >> 2 participants showing DLTs may be selected for evaluation.
If no DLTs or any >> Grade 2 AEs are observed in the first 3 participants treated within a cohort, then escalation to the next dose cohort can proceed following review of the collated 4-week safety data by the safety review committee (SRC).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm dose escalation study of PYC-001 | Experimental | Drug: PYC-001Drug: PYC-001 Phase 1a Open-Label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered PYC-001 in participants with Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- 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;
Key exclusion criteria (15 listed)
- 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;
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PYC-001Single Group Assignment | Drug | Single arm dose escalation study of PYC-001 |
Primary outcome
Incidence, type, severity and relationship of ocular treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (SAEs) in the study eye
Time frame: 24 weeks
Incidence, type, severity and relationship of ocular treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (SAEs) in the study eye
Time frame: 48 weeks
Incidence, type, severity and relationship of ocular TEAEs, and treatment-emergent SAEs in the fellow eye
Time frame: 24 weeks
Incidence, type, severity and relationship of ocular TEAEs, and treatment-emergent SAEs in the fellow eye
Time frame: 48 weeks
Incidence, type, severity and relationship of non-ocular TEAEs, and treatment-emergent SAEs
Time frame: 24 weeks
Incidence, type, severity and relationship of non-ocular TEAEs, and treatment-emergent SAEs
Time frame: 48 weeks
Change from baseline for vital signs measurements (heart rate [HR], systolic and diastolic blood pressure [BP], tympanic temperature, respiratory rate [RR])
Time frame: 48 weeks
Change from baseline for 12-lead electrocardiogram (ECG) parameters
Time frame: 48 weeks
Twelve-lead ECG (including but not limited to the measurements of ventricular HR, PR interval, QRS duration, QT interval and QtcF) will be performed. ECGs will be performed in triplicate at screening only with each replicate separated by at least 1 minute and the full set of triplicates completed within 5 minutes. The mean value for the triplicate will be utilized. Single ECGs will be collected at all other timepoints.
Change from baseline for clinical laboratory results - hematology
Time frame: 48 weeks
Hematology Screening parameters: Hematocrit, Hemoglobin, Mean Corpuscular hemoglobin, mean corpuscular hemoglobin concentration, mean corpuscular volume, mean platelet volume, packed cell volume, platelet count, red blood cell count, reticulocyte count, white blood cell count with differential neutrophil count, eosinophil count, basophil count, lymphocyte count, monocyte count.
Change from baseline for clinical laboratory results - clinical chemistry
Time frame: 48 weeks
Clinical Chemistry screening parameters: Albumin, alkaline phosphatase, alanine aminotransferase, amylase, anion gap, aspartate aminotransferase, bicarbonate, calcium, ionized calcium, chloride, conjugated (direct) and unconjugated bilirubin, creatinine, eGFR, Creatinine kinase, Gamma glutamyl transferase, Globulin, Glucose, High density lipoprotein, Lactate dehydrogenase, Lipase, Low density lipoprotein, Magnesium, Phosphate, Potassium, Sodium, Total bilirubin, Total cholesterol, Total protein, Triglycerides, Urate, Urea
Change from baseline for clinical laboratory results - coagulation
Time frame: 48 weeks
Coagulation screening parameters: Activated partial thromboplastin time, International normalized ratio/Prothrombin time, Fibrinogen
Change from baseline for clinical laboratory results - urinalysis
Time frame: 48 weeks
Urinalysis screening parameters: Bilirubin, Blood, Glucose, Ketones, Leukocyte esterase, Nitrites, pH, Protein, Specific gravity, Urobilinogen
11 secondary outcomes
Change from baseline for Best-corrected visual acuity (BCVA) letter score using Early Treatment of Diabetic Retinopathy Study(ETDRS)
Time frame: Week 4, Week 12, Week 24, Week 48
Change from baseline for Low contrast visual acuity (LCVA)
Time frame: Week 4, Week 12, Week 24, Week 48
Change from baseline for Perimetry
Time frame: Week 4, Week 12, Week 24, Week 48
Change from baseline for posterior eye healthy by fundus examination, using ultrawide funduscopy
Time frame: Week 4, Week 12, Week 24, Week 48
Change from baseline for color vision by Hardy Rand Rittler test
Time frame: Week 4, Week 12, Week 24, Week 48
Change from baseline for contrast sensitivity by Pelli Robson Chart
Time frame: Week 4, Week 12, Week 24, Week 48
Change from baseline for visual field sensitivity by Static Perimetry
Time frame: Week 4, Week 12, Week 24, Week 48
Change from baseline for Multifocal Visual Evoked Potential (mfVEP)
Time frame: Week 4, Week 12, Week 24, Week 48
Change from baseline for retinal nerve fiber layer (RNFL) and Ganglion Cell Layer (GCL) thickness by Spectral domain optical coherence tomography (SD-OCT)
Time frame: Week 4, Week 12, Week 24, Week 48
Change from baseline for mitochondrial function test by flavoprotein fluorescence (FPF) analyzer (Ocumet Beacon)
Time frame: Week 4, Week 12, Week 24, Week 48
Change from baseline for Detection of Apoptosis in Retinal Cells (DARC)
Time frame: Week 4, Week 12, Week 24, Week 48
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2026.
Related trials for OPA1 Gene Mutation
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingObservational study
A Longitudinal Natural History Study of OPA1-Associated Autosomal-Dominant Optic Atrophy
OPA1 Gene Mutation, Optic Atrophy, Autosomal Dominant
RecruitingPhase 1/2
Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)
OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Aishwarya Kundu study chair
PYC Therapeutics
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PYC-001 in people with OPA1 Gene Mutation and Autosomal Dominant Optic Atrophy, run by PYC Therapeutics at 2 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 23 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence, type, severity and relationship of ocular treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (SAEs) in the study eye (time frame: 24 weeks). The study lists 12 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2026.