OPA1 Gene MutationPhase 1/2PYC-001-CL-102NCT06970106
Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)
A Phase 1/2 study testing PYC-001 in people with OPA1 Gene Mutation and Autosomal Dominant Optic Atrophy, run by PYC Therapeutics at 3 sites in 2 countries.
Trial summary
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… 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
- 24 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 30 Sep 2025to Nov 2029
- Phase
- Phase 1/2
- 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 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.
- Treatment naïve participants with best-corrected visual acuity (BCVA) of between ≤20/40 (≤70 Early Treatment of Diabetic Retinopathy Study [ETDRS] letters) and ≥20/200 (≥35 ETDRS letters).
- Treatment Naïve participants with mild to moderate visual field loss and retinal nerve fiber layer (RNFL) loss in the study eye only as determined by the Spectralis Glaucoma Module Premium Edition (GMPE) RNFL \& visual field structure function data (map)
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- Medically healthy (in the opinion of the PI), as determined by pre-study medical history
- Female participants must be of non-childbearing potential or if female participants are of childbearing potential, they must:
- Have a negative pregnancy test at the screening visit and on study Day -1;
- Agree not to attempt to become pregnant or donate ova from signing of the consent form until at least 130 days after final IVT dose administration of PYC-001;
- Agree to use adequate contraception
- Male participants must:
- Agree not to donate sperm
- If engaging in sexual intercourse with a female partner who could become pregnant, agree to use adequate contraception
- If engaging in sexual intercourse with a female partner who is not of childbearing potential or a same-sex partner, agree to use a condom
- Willing and able to comply with all study assessments and protocol schedule/ restrictions
Who cannot join
- 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.
- Have received any prior cell or gene therapy for a retinal condition, excluding participation in study PYC-001-101;
- Within three months prior to study Day -1, have undergone any vitreoretinal surgery or any other ocular surgery in the study eye.
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- Within three months prior to study Day -1, have placement of an Ozurdex® implant. T
- Within three years 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, ;
- Macular edema (intraretinal, sub-retinal or other fluid) in the study eye requiring treatment.
- History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation;
- Have used within 30 days of the Screening visit or is using any investigational drug or over-the-counter drug such as Idebenone, Vitamin B6, Vitamin B12, A decision will be made on a case-by-case basis by the PI in consultation with the Sponsor.
- Over-the-counter drugs like CoQ10 and other Nutraceutical usage will require a washout by five half-lives prior to baseline visit.
- Have a recent history (<6 months) of or current excessive recreational drug or alcohol use, in the opinion of the PI.
- 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 PI would make the volunteer unsuitable for this study
- Presence of illness or pathology that, per investigator, include symptoms and/or the associated treatments that can alter visual function including current ocular infection.
- Positive test for human immunodeficiency virus, hepatitis B or C virus;
- Clinically significant findings in clinical chemistry, hematology, coagulation and urinalysis tests at screening
Trial locations and contacts
3 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
3 sites in 2 countries
Australia2 sites, 2 recruiting
- Recruiting
Cerulea Clinical Trials
East Melbourne
Stefanie Mulabegovic, +61 3 9959 0063, smulabegovic@ceruleaclinicaltrials.org.au
Doron Hickey
- Recruiting
Save Sight Institute - Sydney Eye Hospital
Sydney, New South Wales, 2000
Chanel Ling, +61 02 9382 7309, myrtle.mrg.coordinator@groups.sydney.edu.au
Clare Fraser
New Zealand1 site, 1 recruiting
- Recruiting
Retina Specialists
Auckland, 1052
Zanelle Angelo, +6493072020, nurses@retinaspecialists.co.nz
Andrea Vincent
About this study
Official title: A Phase 1b Open-Label, Randomized, Single Dose and Repeat Dose Study to Evaluate the Single and Repeat Dose Safety and Tolerability of Intravitreally Administered PYC-001 in Participants With Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy
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:
- A single 60ug dose of PYC-001
- Three doses of 10ug PYC-001 at an interval of 8 weeks
- Three doses of 10ug PYC-001 at an interval of 12 weeks
- Three doses of 30ug PYC-001 at an interval of 8 weeks
- 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:
- Three doses of 60ug PYC-001 at an interval of 12 weeks
Read the detailed description
This is a phase 1b open-label, randomized, single and repeat dose study to evaluate the safety and tolerability of IVT administered PYC-001 in participants with confirmed OPA1 mutation-associated ADOA.
The primary objective of this study is to gather safety data and determine the optimal dosing regimen for PYC-001.
The exploratory objectives of this study include evaluating the ocular structural and functional changes following multiple doses of IVT administered PYC-001. The PK profile of PYC-001 following multiple doses will also be assessed.
In this open-label study, PYC-001 will be injected in a single eye and ocular safety will be assessed in both eyes.
- 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
PYC-001 is a peptide-phosphorodiamidate morpholino oligonucleotide administered intravitreally
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Single Dose of 60ug | Experimental | A single dose of 60ug of PYC-001 administered intravitreally Receives: Drug: PYC-001 |
| Cohort 2: 10ug of PYC-001, 8 weeks | Experimental | Up to 12 doses of 10ug PYC-001, administered intravitreally in 8 weeks interval. Receives: Drug: PYC-001 |
| Cohort 3: 10ug of PYC-001, 12 weeks | Experimental | Up to 12 doses of 10ug PYC-001, administered intravitreally in 12 weeks interval Receives: Drug: PYC-001 |
| Cohort 4: 30ug of PYC-001, 8 weeks | Experimental | Up to 12 doses of 30ug PYC-001, administered intravitreally in 8 weeks interval Receives: Drug: PYC-001 |
| Cohort 5: 30ug of PYC-001, 12 weeks | Experimental | Up to 12 doses of 30ug PYC-001, administered intravitreally in 12 weeks interval Receives: Drug: PYC-001 |
| Cohort 6: 60ug of PYC-001, 12 weeks | Experimental | Following SRC, Single dose participant can continue to receive Up to 12 doses of 60ug PYC-001, administered intravitreally at 12 weeks interval; alternatively, treatment naive participants can be enrolled (once SRC is complete) to receive up to 12 doses Receives: Drug: PYC-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
[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.
Measured: Up to 164 weeks
[All Cohorts] Changes from baseline in vital signs (heart rate)
Measured: Up to 164 weeks
[All Cohorts] Changes from baseline in vital signs (systolic and diastolic blood pressure)
Measured: Up to 164 weeks
[All Cohorts] Changes from baseline in vital signs (tympanic temperature)
Measured: Up to 164 weeks
[All Cohorts] Changes from baseline in vital signs (respiratory rate)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in white blood cells (WBC), platelets, white blood cell count with differential neutrophil count, eosinophil count, basophil count, lymphocyte count, monocyte count
All measured in 10x^9L
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in red blood cells
Units x 10^12L
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in hemoglobin and mean corpuscular hemaglobin concentration
Units: grams per liter (g/L)
Measured: Up to164 weeks
[All Cohorts] Change from baseline in mean corpuscular volume and mean platelet volume
measured in femtoliter (fL)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in hematocrit
Units: litres per litre (L/L)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in mean corpuscular hemoglobin
Units: picograms (pg)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in neutrophils, lymphocytes, monocytes, eosinophils, basophils and reticulocyte count
Units: %
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in Alanine Transaminase, Alkaline Phosphatase, Aspartate aminotransferase, Creatine Phosphokinase, Gamma Glutamyl Transferase, Lactate Dehydrogenase, Amylase and Lipase
Units: units/Liter (U/L)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in Bilirubin (total, direct and indirect) and creatinine
Units: micromoles per liter (mcmol/L)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in protein, albumin, globulin and fibrinogen
Units:grams per liter (g/L)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in Blood Urea Nitrogen (BUN), Sodium, Potassium, Chloride, Bicarbonate, Calcium, Magnesium, Phosphorous, Total Cholesterol, Triglyceride, HDL, LD. Uric Acid and Glucose
Units: millimoles per liter (mmol/L)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in Anion Gap
Units: milliequivalents per liter (mEq/L)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in Estimated Glomerular Filtration Rate (eGFR)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in Prothrombin Time and Partial Thromboplastin Time, Activated (APTT)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in Prothrombin Time (INR)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in urinalysis (Protein, glucose, ketones, blood, bilirubin, leucocyte esterase and nitrites
Presence (positive, negative, trace) captured
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in urinalysis (pH)
Measured: Up to 164 weeks
[All Cohorts] Change from baseline in urinalysis (specific gravity)
Measured: Up to 164 weeks
Secondary outcomes (11)
[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.
Measured: Up to 164 weeks
[All Cohorts] Change from Baseline in Visual field sensitivity by photopic static perimetry
Measured: Up to 164 weeks
[All Cohorts] Change from Baseline in posterior eye health by fundus examination using ultrawide fundoscopy
Measured: Up to 164 weeks
[All Cohorts] Change from Baseline in Color vision
Measured: Up to 164 weeks
[All Cohorts] Change from Baseline in Contrast sensitivity by Pelli Robson chart
Measured: Up to 164 weeks
[All Cohorts] Change from Baseline in Multifocal visual evoked potential OR Full field electroretinogram
Measured: Up to 164 weeks
[All Cohorts] Change from Baseline in Retinal nerve fiber layer (RNFL) by spectral domain optical coherence tomography
Measured: Up to 164 weeks
[All Cohorts] Change from Baseline for Ganglion Cell Layer (GCL) thickness determined by Spectral domain optical coherence tomography
Measured: Up to 164 Weeks
[All Cohorts] Change from Baseline for Bruch's membrane opening (BMO) disc size, as determined by spectral domain optical coherence tomography
Measured: Up to 164 weeks
[All Cohorts] Change from Baseline in Mitochondrial function test via flavoprotein fluorescence
Measured: Up to 164 weeks
[Repeat Dose Cohorts] Plasma concentrations of PYC-001 following multiple dose intravitreally administered PYC-001
Measured: Up to 164 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2025
- Study start30 Sep 2025
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 10mcg and 30mcg dose groups will be enrolling in parallel. 60mcg dose enrollment will be sequential - SAD cohort will be filled first followed by 60mcg MAD (once safety review is completed)
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- PYC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06970106
- Sponsor's protocol ID
- PYC-001-CL-102
- Acronym
- PYC-001-CL-102
- Status last verified
- Jun 2026
- First submitted
- 28 Apr 2025
- Last updated
- 29 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b Open-Label, Randomized, Single Dose and Repeat Dose Study to Evaluate the Single and Repeat Dose Safety and Tolerability of Intravitreally Administered PYC-001 in Participants With Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy
- ClinicalTrials.gov ID
- NCT06970106
- Acronym / sponsor ID
- PYC-001-CL-102 / PYC-001-CL-102
- Conditions
- OPA1 Gene Mutation, Autosomal Dominant Optic Atrophy, Hereditary Optic Atrophies, Kjer Optic Atrophy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- PYC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 30 Sep 2025 (actual)
- Primary completion
- Oct 2029 (estimated)
- Study completion
- Nov 2029 (estimated)
- First posted
- 14 May 2025 (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.
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:
- A single 60ug dose of PYC-001
- Three doses of 10ug PYC-001 at an interval of 8 weeks
- Three doses of 10ug PYC-001 at an interval of 12 weeks
- Three doses of 30ug PYC-001 at an interval of 8 weeks
- 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:
- Three doses of 60ug PYC-001 at an interval of 12 weeks
Detailed description
This is a phase 1b open-label, randomized, single and repeat dose study to evaluate the safety and tolerability of IVT administered PYC-001 in participants with confirmed OPA1 mutation-associated ADOA.
The primary objective of this study is to gather safety data and determine the optimal dosing regimen for PYC-001.
The exploratory objectives of this study include evaluating the ocular structural and functional changes following multiple doses of IVT administered PYC-001. The PK profile of PYC-001 following multiple doses will also be assessed.
In this open-label study, PYC-001 will be injected in a single eye and ocular safety will be assessed in both eyes.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
- Model description
- 10mcg and 30mcg dose groups will be enrolling in parallel. 60mcg dose enrollment will be sequential - SAD cohort will be filled first followed by 60mcg MAD (once safety review is completed)
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: Single Dose of 60ug | Experimental | Drug: PYC-001A single dose of 60ug of PYC-001 administered intravitreally |
| Cohort 2: 10ug of PYC-001, 8 weeks | Experimental | Drug: PYC-001Up to 12 doses of 10ug PYC-001, administered intravitreally in 8 weeks interval. |
| Cohort 3: 10ug of PYC-001, 12 weeks | Experimental | Drug: PYC-001Up to 12 doses of 10ug PYC-001, administered intravitreally in 12 weeks interval |
| Cohort 4: 30ug of PYC-001, 8 weeks | Experimental | Drug: PYC-001Up to 12 doses of 30ug PYC-001, administered intravitreally in 8 weeks interval |
| Cohort 5: 30ug of PYC-001, 12 weeks | Experimental | Drug: PYC-001Up to 12 doses of 30ug PYC-001, administered intravitreally in 12 weeks interval |
| Cohort 6: 60ug of PYC-001, 12 weeks | Experimental | Drug: PYC-001Following SRC, Single dose participant can continue to receive Up to 12 doses of 60ug PYC-001, administered intravitreally at 12 weeks interval; alternatively, treatment naive participants can be enrolled (once SRC is complete) to receive up to 12 doses |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- 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.
- Treatment naïve participants with best-corrected visual acuity (BCVA) of between ≤20/40 (≤70 Early Treatment of Diabetic Retinopathy Study [ETDRS] letters) and ≥20/200 (≥35 ETDRS letters).
Key exclusion criteria (20 listed)
- 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.
- Have received any prior cell or gene therapy for a retinal condition, excluding participation in study PYC-001-101;
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-001PYC-001 is a peptide-phosphorodiamidate morpholino oligonucleotide administered intravitreally | Drug | Cohort 1: Single Dose of 60ug, Cohort 2: 10ug of PYC-001, 8 weeks, Cohort 3: 10ug of PYC-001, 12 weeks, Cohort 4: 30ug of PYC-001, 8 weeks, Cohort 5: 30ug of PYC-001, 12 weeks, Cohort 6: 60ug of PYC-001, 12 weeks |
Primary outcome
[All Cohorts] Number of participants experiencing treatment emergent adverse events
Time frame: Up to 164 weeks
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)
Time frame: Up to 164 weeks
[All Cohorts] Changes from baseline in vital signs (systolic and diastolic blood pressure)
Time frame: Up to 164 weeks
[All Cohorts] Changes from baseline in vital signs (tympanic temperature)
Time frame: Up to 164 weeks
[All Cohorts] Changes from baseline in vital signs (respiratory rate)
Time frame: Up to 164 weeks
[All Cohorts] Change from baseline in white blood cells (WBC), platelets, white blood cell count with differential neutrophil count, eosinophil count, basophil count, lymphocyte count, monocyte count
Time frame: Up to 164 weeks
All measured in 10x^9L
[All Cohorts] Change from baseline in red blood cells
Time frame: Up to 164 weeks
Units x 10^12L
[All Cohorts] Change from baseline in hemoglobin and mean corpuscular hemaglobin concentration
Time frame: Up to164 weeks
Units: grams per liter (g/L)
[All Cohorts] Change from baseline in mean corpuscular volume and mean platelet volume
Time frame: Up to 164 weeks
measured in femtoliter (fL)
[All Cohorts] Change from baseline in hematocrit
Time frame: Up to 164 weeks
Units: litres per litre (L/L)
[All Cohorts] Change from baseline in mean corpuscular hemoglobin
Time frame: Up to 164 weeks
Units: picograms (pg)
[All Cohorts] Change from baseline in neutrophils, lymphocytes, monocytes, eosinophils, basophils and reticulocyte count
Time frame: Up to 164 weeks
Units: %
[All Cohorts] Change from baseline in Alanine Transaminase, Alkaline Phosphatase, Aspartate aminotransferase, Creatine Phosphokinase, Gamma Glutamyl Transferase, Lactate Dehydrogenase, Amylase and Lipase
Time frame: Up to 164 weeks
Units: units/Liter (U/L)
[All Cohorts] Change from baseline in Bilirubin (total, direct and indirect) and creatinine
Time frame: Up to 164 weeks
Units: micromoles per liter (mcmol/L)
[All Cohorts] Change from baseline in protein, albumin, globulin and fibrinogen
Time frame: Up to 164 weeks
Units:grams per liter (g/L)
[All Cohorts] Change from baseline in Blood Urea Nitrogen (BUN), Sodium, Potassium, Chloride, Bicarbonate, Calcium, Magnesium, Phosphorous, Total Cholesterol, Triglyceride, HDL, LD. Uric Acid and Glucose
Time frame: Up to 164 weeks
Units: millimoles per liter (mmol/L)
[All Cohorts] Change from baseline in Anion Gap
Time frame: Up to 164 weeks
Units: milliequivalents per liter (mEq/L)
[All Cohorts] Change from baseline in Estimated Glomerular Filtration Rate (eGFR)
Time frame: Up to 164 weeks
[All Cohorts] Change from baseline in Prothrombin Time and Partial Thromboplastin Time, Activated (APTT)
Time frame: Up to 164 weeks
[All Cohorts] Change from baseline in Prothrombin Time (INR)
Time frame: Up to 164 weeks
[All Cohorts] Change from baseline in urinalysis (Protein, glucose, ketones, blood, bilirubin, leucocyte esterase and nitrites
Time frame: Up to 164 weeks
Presence (positive, negative, trace) captured
[All Cohorts] Change from baseline in urinalysis (pH)
Time frame: Up to 164 weeks
[All Cohorts] Change from baseline in urinalysis (specific gravity)
Time frame: Up to 164 weeks
11 secondary outcomes
[All Cohorts] Change from Baseline in Best Corrected Visual Acuity (BCVA)/ High Contrast Visual Acuity (HCVA) and Low Contrast Visual Acuity (LCVA) scores
Time frame: Up to 164 weeks
[All Cohorts] Change from Baseline in Visual field sensitivity by photopic static perimetry
Time frame: Up to 164 weeks
[All Cohorts] Change from Baseline in posterior eye health by fundus examination using ultrawide fundoscopy
Time frame: Up to 164 weeks
[All Cohorts] Change from Baseline in Color vision
Time frame: Up to 164 weeks
[All Cohorts] Change from Baseline in Contrast sensitivity by Pelli Robson chart
Time frame: Up to 164 weeks
[All Cohorts] Change from Baseline in Multifocal visual evoked potential OR Full field electroretinogram
Time frame: Up to 164 weeks
[All Cohorts] Change from Baseline in Retinal nerve fiber layer (RNFL) by spectral domain optical coherence tomography
Time frame: Up to 164 weeks
[All Cohorts] Change from Baseline for Ganglion Cell Layer (GCL) thickness determined by Spectral domain optical coherence tomography
Time frame: Up to 164 Weeks
[All Cohorts] Change from Baseline for Bruch's membrane opening (BMO) disc size, as determined by spectral domain optical coherence tomography
Time frame: Up to 164 weeks
[All Cohorts] Change from Baseline in Mitochondrial function test via flavoprotein fluorescence
Time frame: Up to 164 weeks
[Repeat Dose Cohorts] Plasma concentrations of PYC-001 following multiple dose intravitreally administered PYC-001
Time frame: Up to 164 weeks
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 29 Jun 2026.
- Individual participant dataSharing planned: No
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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.
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PYC-001 in people with OPA1 Gene Mutation and Autosomal Dominant Optic Atrophy, run by PYC Therapeutics at 3 sites in 2 countries.
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 16 inclusion and 20 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: looking for participants now, as of the sponsor's last update on 29 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Australia, New Zealand. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Contact Jessica Stevenson (+61 8 6151 0992, adoa@pyctx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
[All Cohorts] Number of participants experiencing treatment emergent adverse events (time frame: Up to 164 weeks). The study lists 23 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 29 Jun 2026.