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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06140329




Registration number
NCT06140329
Ethics application status
Date submitted
15/11/2023
Date registered
18/11/2023

Titles & IDs
Public title
Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation
Scientific title
A Natural History Study in Patients With Genetically Confirmed Diagnosis of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation
Secondary ID [1] 0 0
PYC-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Autosomal Dominant Optic Atrophy 0 0
Optic Atrophy, Autosomal Dominant 0 0
Optic Atrophies, Hereditary 0 0
Kjer Optic Atrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Eye 0 0 0 0
Diseases / disorders of the eye
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Neurodegenerative diseases
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Best-corrected High Contrast Visual Acuity (HCVA)
Timepoint [1] 0 0
Baseline through Year 2
Primary outcome [2] 0 0
Low Contrast Visual Acuity (LCVA)
Timepoint [2] 0 0
Screening through Year 2
Primary outcome [3] 0 0
Contrast Sensitivity
Timepoint [3] 0 0
Baseline through Year 2
Primary outcome [4] 0 0
Color Vision
Timepoint [4] 0 0
Baseline through Year 2
Primary outcome [5] 0 0
Retinal Thickness
Timepoint [5] 0 0
Baseline through Year 2
Primary outcome [6] 0 0
Ellipsoid Zone (EZ) Volume
Timepoint [6] 0 0
Baseline through Year 2
Primary outcome [7] 0 0
Ellipsoid Zone (EZ) Area
Timepoint [7] 0 0
Baseline through Year 2
Primary outcome [8] 0 0
Visual Field Sensitivity
Timepoint [8] 0 0
Baseline through Year 2
Primary outcome [9] 0 0
Multifocal Visual Evoked Potential (mfVEP)
Timepoint [9] 0 0
Baseline through Year 2
Primary outcome [10] 0 0
Pregnancy Test
Timepoint [10] 0 0
Baseline
Primary outcome [11] 0 0
DARC (Detection of Apoptosing Retinal Cells)
Timepoint [11] 0 0
Baseline through Year 2
Primary outcome [12] 0 0
Flavoprotein Fluorescence (FPF)
Timepoint [12] 0 0
Baseline through Year 2
Primary outcome [13] 0 0
Retinal Abnormalities
Timepoint [13] 0 0
Baseline through Year 2
Primary outcome [14] 0 0
Adverse Events (AEs)
Timepoint [14] 0 0
Screening through Year 2
Primary outcome [15] 0 0
Genomic Analysis for Study Eligibility
Timepoint [15] 0 0
Screening
Primary outcome [16] 0 0
Vital signs
Timepoint [16] 0 0
Baseline through Year 2
Secondary outcome [1] 0 0
To determine the outcome measures that are associated with ADOA disease progression.
Timepoint [1] 0 0
Baseline through Year 2

Eligibility
Key inclusion criteria
* Participants and/or their parent(s)/guardian(s) 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. Assent, where appropriate, will be obtained according to institutional guidelines.
* Males and females, 8 years of age and above.
* Have a clinical diagnosis of OPA1 mutation (haploinsufficiency) associated ADOA.
* No other ocular pathology.
* Patients with best-corrected visual acuity (BCVA) of between 20/40 (70 Early Treatment of Diabetic Retinopathy Study [ETDRS] letters) and 20/160 (39-43 ETDRS letters)
* Willing and able to comply with all study assessments and adhere to the protocol schedule and restrictions.
* For sites performing the Detection of apoptosis in retinal cells (DARC) procedure, and in volunteers = 12 years only:

1. Female volunteers must:

I. Be of non-child-bearing potential 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), or

II. If of childbearing potential, must:
* Have a negative pregnancy test at the screening visit and prior to each administration of ANX776, and
* Agree not to attempt to become pregnant or donate ova from signing the consent form until at least 30 days after the last dose of ANX776, and
* Agree to use adequate contraception (defined as the use of a condom by the male partner combined with the use of a highly effective method of contraception from one month prior to screening until at least 30 days after the last dose of ANX776, if not exclusively in a same-sex relationship or abstinent as a committed lifestyle.
2. Male volunteers must:

* Agree not to donate sperm from signing the consent form until at least 90 days after the last dose of ANX776, and
* If engaging in sexual intercourse with a female partner who could become pregnant, agree to use adequate contraception (defined as the use of a condom combined with the use of a highly effective method of contraception) from signing the consent form until at least 90 days after the last dose of study drug.
Minimum age
8 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Participant has a known allergy to ANX776 or any of its excipients.
* Have any uncontrolled systemic disease that, in the opinion of the Investigator, would preclude participation in the study, which includes but is not limited to, infection, uncontrolled elevated blood pressure, cardiovascular disease, or glycemic control issues, or any other medical condition that may put the participant at risk due to study procedures. Note: comorbidities relevant to the pathogenesis of OPA1 associated ADOA (including hearing loss, peripheral neuropathy, myopathy, and ataxia) are acceptable.
* Have mutations in genes that cause ADOA, other than OPA1 (for example in case of dominant negative ADOA) and ADOA Plus.
* Within 3 months prior to Baseline (Visit 2), 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.
* Have ocular media opacity or poor pupillary dilation prohibiting quality ophthalmic evaluation or photography, as assessed by the Investigator.
* Have used any investigational drug or device within 90 days or 5 estimated half-lives of Visit 2, whichever is longer, or plan to participate in another study of a drug or device during the study period. Participation in observational trials is allowable based on Investigator discretion and consultation with the Medical Monitor. It is assumed that the observational trial evaluations would not interfere with participation in this study.
* Have received any prior cell or gene therapy for a retinal condition.
* 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.
* 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.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/02/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2026
Actual
Sample size
Target
40
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Sydney Eye Hospital - Sydney
Recruitment postcode(s) [1] 0 0
- Sydney

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
PYC Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Sreenivasu Mudumba, PhD
Address 0 0
PYC Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Alistair Mallard
Address 0 0
Country 0 0
Phone 0 0
+61-8 8249 4788
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06140329