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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06628947




Registration number
NCT06628947
Ethics application status
Date submitted
3/10/2024
Date registered
8/10/2024
Date last updated
9/09/2025

Titles & IDs
Public title
A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
Scientific title
A Phase II, Randomised, Controlled, Double Masked, Multiple Dose Study of the Safety, Tolerability and Efficacy of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
Secondary ID [1] 0 0
KIO-301-2101
Universal Trial Number (UTN)
Trial acronym
ABACUS-2
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Retinitis Pigmentosa 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
Other interventions - Placebo (Sterile Saline)
Treatment: Drugs - 100 µg KIO-301
Treatment: Drugs - 50 µg KIO-301

Experimental: 50 µg KIO-301 - 50 µg KIO-301 or placebo administered by IVT injection bilaterally (OU) once every 6 weeks for 3 administrations per participant.

Experimental: 100 µg KIO-301 - 100 µg KIO-301 or placebo administered by IVT injection OU once every 6 weeks for 3 administrations per participant.


Other interventions: Placebo (Sterile Saline)
A control 50 µl injection of clear sterile saline liquid

Treatment: Drugs: 100 µg KIO-301
KIO-301 drug product is an ophthalmic formulation of the drug substance KIO-300-Cl in sulfobutylether-ß-cyclodextrin, sucrose, phosphate buffer salts and water suitable for IVT injection. The drug substance KIO-300-Cl is a quaternary ammonium chloride salt of the active compound KIO-300.

Treatment: Drugs: 50 µg KIO-301
KIO-301 drug product is an ophthalmic formulation of the drug substance KIO-300-Cl in sulfobutylether-ß-cyclodextrin, sucrose, phosphate buffer salts and water suitable for IVT injection. The drug substance KIO-300-Cl is a quaternary ammonium chloride salt of the active compound KIO-300.
Intervention code [1] 0 0
Other interventions
Intervention code [2] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To assess the safety and tolerability of multiple doses of KIO-301 administered by IVT injection of KIO-301 in patients with late-stage RP.
Timepoint [1] 0 0
Baseline (Week 1/pre-dose) to Week 25 (12 weeks post 3rd IVT administration of KIO-301)

Eligibility
Key inclusion criteria
1. Be aged 18 years or older at the time of consent.
2. Provide informed consent prior to any study procedures, as stipulated by local laws, Ethics Committee (EC) and Regulatory Authority (RA) guidelines.
3. Be willing and able to follow all study instructions, attend all study visits, and complete all study assessments.
4. Have a clinical diagnosis of non-syndromic RP, with the exception of Usher's Syndrome Type II (USH2) which is allowed.
5. Have a visual acuity at Screening of:

* NLP OU confirmed by inability to see pen torch light at 30cm in each eye (OU) (assigned logMAR of 4.0 as per the Berkeley Rudimentary Vision Test (BRVT).
* LP or ULV OU limited to logMAR > 1.6 and < 4.0 as determined by the BRVT.
6. Other than intravitreal corticosteroids, participants must not receive intravitreal concomitant medications from Screening until end of study.
7. Must fail at least one functional vision Multi-luminance Orientation and Mobility (MLOM) assessment at a light level between 100 and 980 lux at Screening.
8. Must agree to follow appropriate contraception requirements from Screening until 3 months after the last dose of IMP.

* Participants assigned female at birth who are of child-bearing potential (OCBP) must agree to a pregnancy test at Visit 1 and use an acceptable method of birth control including oral, transdermal, injectable, or implantable hormonal contraception, intrauterine device, abstinence from intercourse with partner assigned male at birth, or surgical sterilisation of partner assigned male at birth. Participants assigned female at birth are not OCBP if they have had a hysterectomy, bilateral oophorectomy, bilateral tubal ligation, or are post-menopausal by at least 12 months.
* Participants assigned male at birth with a partner OCBP must be surgically sterile for at least 3 months prior to starting study drug, or ensure their partner uses contraception as outlined above, and must use a male condom. Participants assigned male at birth must not donate sperm from Screening until 3 months after the last dose of IMP.
* Participants who have practiced true abstinence for at least 1 year due to usual and preferred lifestyle choice are exempt from contraceptive requirements. If a participant who is abstinent becomes sexually active, they must agree to use appropriate contraception as described above.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Pregnant or breast-feeding, or plan to become pregnant during the study.
2. Have evidence of material/substantial optic nerve disease.
3. Have a history of one or more retinal detachments.
4. Other than RP related macular pathologies, have clinically significant ocular disease (e.g., corneal oedema, uveitis, severe keratoconjunctivitis sicca), or clinically significant opacities of the media which might interfere with the study assessments, or the ability of the participant to complete the study.
5. Have a history of high myopia (at least > 6 diopters)
6. Have uncontrolled severe glaucoma defined as intraocular pressure (IOP) of >26 mmHg when on 2 or more IOP lowering medications and cup disc ratio of >0.8, as diagnosed by an ophthalmologist.
7. Have had a previous intraocular surgery (excluding phacoemulsification cataract surgery and YAG capsulotomy more than 12 months prior to first study drug administration)
8. Have aphakia or a subluxed intraocular lens, or have evidence of zonular weakness that in the opinion of the investigator would result in light obfuscation.
9. Have a psychiatric condition that, in the investigator's opinion, precludes compliance with the protocol; past or present psychoses; past or present bipolar disorder; disorder requiring lithium; or within five years prior to screening, a history of suicide plan.
10. Have any clinically significant abnormality at screening determined by medical history, vital signs, clinical biochemistry, haematology, urinalysis, or a 12-lead electrocardiogram (ECG), as assessed by the investigator, which might interfere with the study assessments or the ability of the participant to complete the study.
11. Have any other medical condition or significant co-morbidities, or any finding during screening, which in the view of the Investigator is likely to interfere with the study or put the Participant at risk, confound study data, or interfere significantly with study participation.
12. Have clinical signs of active ocular or systemic infection and/or a temperature greater than 38.0°C at the time of screening. Study entry must be deferred at least 14 days from resolution.
13. Have participated in any investigational study within 30 days prior to screening, prior exposure to an investigational product within 5 elimination half-lives, or planned used of an investigational product or device during the study.
14. Have known or suspected hypersensitivity to any of the study drug excipients.
15. Are taking any medications that are known to be toxic to the retina or optic nerve.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
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
29/08/2025
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2027
Actual
Sample size
Target
36
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Save Sight Institute - Sydney
Recruitment hospital [2] 0 0
Queensland Eye Institute - Woolloongabba
Recruitment hospital [3] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [4] 0 0
Cerulea Clinical Trials - East Melbourne
Recruitment hospital [5] 0 0
Lions Eye Institute - Nedlands
Recruitment postcode(s) [1] 0 0
2000 - Sydney
Recruitment postcode(s) [2] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [3] 0 0
5000 - Adelaide
Recruitment postcode(s) [4] 0 0
3002 - East Melbourne
Recruitment postcode(s) [5] 0 0
6009 - Nedlands

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
Doron Hickey, MBChB
Address 0 0
The Centre for Eye Research Australia (CERA)
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Eric Daniels, MD
Address 0 0
Country 0 0
Phone 0 0
+61 448 787 315
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)?
Undecided
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/NCT06628947