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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05282953




Registration number
NCT05282953
Ethics application status
Date submitted
8/03/2022
Date registered
16/03/2022

Titles & IDs
Public title
A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)
Scientific title
A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)
Secondary ID [1] 0 0
KIO-301-1101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Retinitis Pigmentosa 0 0
Choroideremia 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Stereotactic radiosurgery
Treatment: Drugs - KIO-301

Experimental: Patients with Retinitis Pigmentosa and Choroideremia -


Treatment: Other: Stereotactic radiosurgery
HyperArc is a radiotherapy treatment with a structured workflow to plan and deliver stereotactic radiosurgery (SRS).

Treatment: Drugs: KIO-301
KIO-301 intravitreal injection at ascending doses
Intervention code [1] 0 0
Treatment: Other
Intervention code [2] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with treatment-related adverse events as assessed by overall changes in retinal thickness and appearance, clinical chemistry and hematology, and other eye assessments.
Timepoint [1] 0 0
84 days

Eligibility
Key inclusion criteria
1. Have a clinical diagnosis of retinitis pigmentosa (Cohorts 1 - 3) or choroideremia (Cohort 3 only).
2. Have a visual acuity of:

1. no light perception for Cohort 3 or
2. no light perception or bare light perception for Cohort 1 confirmed with a LogMar >2.9 using the Berkeley Rudimentary Vision Test (BRVT), or
3. count fingers or hand motion for Cohort 2 as confirmed by a LogMar = 2.9 and > 1.6 using the BRVT.
3. Have similar visual acuity in both eyes as defined as a LogMar difference between eyes of < 0.05 using the BRVT.
Minimum age
18 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Have evidence of material/substantial optic nerve disease.
2. Have a history of retinal detachments.
3. Have clinically significant ocular disease (e.g., corneal oedema, uveitis, severe keratoconjunctivitis sicca) which might interfere with the study or clinically significant opacities of the media.
4. Have high intraocular pressure (IOP) >22 mm Hg.
5. Have had a previous intraocular surgery (excluding phakocataract surgery).
6. Have aphakia.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
10/11/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/01/2025
Actual
Sample size
Target
12
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Royal Adeliade Hospital - Adelaide
Recruitment hospital [2] 0 0
Harley Eye Clinic - North Adelaide
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
5006 - North Adelaide

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Kiora Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Robert Casson
Address 0 0
Royal Adelaide Hospital
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Eric Daniels, M.D., MBA
Address 0 0
Country 0 0
Phone 0 0
61 448787315
Fax 0 0
Email 0 0
edaniels@kiorapharma.com
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/NCT05282953