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Tests treatment safety and results for OPA1 Gene Mutation
Official title Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)
ClinicalTrials.gov ID: NCT06970106
What this study is testing
What is PYC-001?
PYC-001 is an investigational medicine, being studied as a potential treatment for opa1 gene mutation.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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...
- Treatment naïve participants with best-corrected visual acuity (BCVA) of between ≤20/40 (≤70 Early Treatment of Diabetic Retinopathy Study [ETDRS]...
You likely can't join if
- 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...
- 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...
- 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.
See the full eligibility criteria
- 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...
- 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
- 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...
- 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.
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Sydney, New South Wales, Australia
- East Melbourne, Australia
- Auckland, New Zealand
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Sydney, New South Wales, Australia; East Melbourne, Australia; Auckland, New Zealand. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.