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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04850118




Registration number
NCT04850118
Ethics application status
Date submitted
5/04/2021
Date registered
20/04/2021

Titles & IDs
Public title
A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP
Scientific title
A Randomized, Controlled, Masked, Multi-center Study Evaluating the Efficacy, Safety, and Tolerability of Two Doses of AGTC-501 Compared to an Untreated Control Group in Male Participants With X-linked Retinitis Pigmentosa
Secondary ID [1] 0 0
AGTC-RPGR-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
X-Linked 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
Treatment: Other - rAAV2tYF-GRK1-hRPGRco
Treatment: Drugs - Control

Active comparator: Group 1: Dose - Male participants 12-50 years of age treated by subretinal injection with the of AGTC-501

Active comparator: Group 2: Dose - Male participants 12-50 years of age treated by subretinal injection with the dose of AGTC-501

Other: Group 3: Control - Male participants 12-50 years of age in the untreated control group. Participants in the control group will be followed for a minimum of 24 months. After all participants have reached Month 12, participants in the control group will be given the option to receive the study drug in the fellow eye, if eligible.


Treatment: Other: rAAV2tYF-GRK1-hRPGRco
Adeno-associated virus vector expressing a human RPGR gene

Treatment: Drugs: Control
Untreated Control Group 3
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
The proportion of participants with a =15 letter increase from baseline in LLVA
Timepoint [1] 0 0
Day 0 - Month 12
Secondary outcome [1] 0 0
Change from baseline in mobility test score at Month 12
Timepoint [1] 0 0
Day 0 - Month 12
Secondary outcome [2] 0 0
Change from baseline in mean sensitivity across the whole grid, as measured by MAIA microperimetry
Timepoint [2] 0 0
Day 0 - Month 18
Secondary outcome [3] 0 0
Visual sensitivity improvement from baseline in at least 5 loci
Timepoint [3] 0 0
Month 12
Secondary outcome [4] 0 0
Change from baseline in mobility test score
Timepoint [4] 0 0
Month 12
Secondary outcome [5] 0 0
Change from baseline in full-field stimulus threshold (FST)
Timepoint [5] 0 0
Month 12
Secondary outcome [6] 0 0
Change from baseline in mean sensitivity across the central 4 loci
Timepoint [6] 0 0
Month 12
Secondary outcome [7] 0 0
Proportion of participants with a =15 letter increase from baseline
Timepoint [7] 0 0
Month 18 and Month 24
Secondary outcome [8] 0 0
Change in low-luminance deficit (LLD), defined as the difference between BCVA and LLVA
Timepoint [8] 0 0
Month 12
Secondary outcome [9] 0 0
Change from baseline in BCVA over time as assessed by an Early Treatment of Diabetic Retinopathy (ETDRS) or Tumbling E chart at Month 12
Timepoint [9] 0 0
Month 12
Secondary outcome [10] 0 0
Response by Ora-VNC where response is defined as a mobility test score increase of 2 or more luminance levels at Month 12
Timepoint [10] 0 0
Month 12
Secondary outcome [11] 0 0
Change from baseline in mean sensitivity across the whole grid, as measured by MAIA microperimetry at Month 24
Timepoint [11] 0 0
Month 24
Secondary outcome [12] 0 0
Visual sensitivity improvement from baseline in at least 5 loci
Timepoint [12] 0 0
Month 18 and 24
Secondary outcome [13] 0 0
Change from baseline in full-field stimulus threshold (FST) at Month 24
Timepoint [13] 0 0
Month 24
Secondary outcome [14] 0 0
Change from baseline in mean sensitivity across the central 4 loci, as measured by MAIA microperimetry, at Month 18 and Month 24
Timepoint [14] 0 0
Month 18 and 24
Secondary outcome [15] 0 0
Proportion of participants with a =10 letter increase from baseline in LLVA at Month 12, 18 and 24
Timepoint [15] 0 0
Month 12, 18 and 24
Secondary outcome [16] 0 0
Change in low-luminance deficit (LLD), defined as the difference between BCVA and LLVA Month 18 and 24
Timepoint [16] 0 0
Month 18 and 24
Secondary outcome [17] 0 0
Change from baseline in BCVA over time as assessed by an Early Treatment of Diabetic Retinopathy (ETDRS) or Tumbling E chart at Month 18 and 24
Timepoint [17] 0 0
Month 18 and 24
Secondary outcome [18] 0 0
Change from baseline in mobility test score at Month 18 and 24 as measured by the Ora- VNC mobility course
Timepoint [18] 0 0
Month 18 and 24
Secondary outcome [19] 0 0
Response by Ora-VNC where response is defined as a mobility test score increase of 2 or more luminance levels at Month 18 and 24
Timepoint [19] 0 0
Month 18 and 24

Eligibility
Key inclusion criteria
General

1. Provide written informed consent or assent (per local regulation), prior to the conduct of any study-related procedure. Participants who provide assent must have a parent, guardian, or legal representative provide written informed consent.
2. Be between 12 and 50 years of age (inclusive) at the time of informed consent and assent (as applicable).
3. Be male (XY chromosome) and have at least one documented pathogenic or likely pathogenic variant in the RPGR gene.
4. Have a clinical diagnosis of XLRP.
5. Be able and willing, as assessed by the Investigator, to follow study instructions, complete study assessments, comply with the protocol, and attend study visits for the duration of the study.

Ocular Inclusion Criteria (Study Eye):
6. Have a BCVA = 78 letters (approximately Snellen, 20/32) and = 34 letters (approximately Snellen, 20/200)
7. Have a LLVA =64 letters (approximately Snellen 20/50) in the study eye
8. Be able to perform all tests of visual and retinal function and structure in both eyes based on the participant's reliability, and fixation, in the study eye per the Investigator's discretion.
9. Have detectable baseline mean macular sensitivity .
10. Have a detectable sub-foveal ellipsoid zone (EZ) line as assessed by SD-OCT in the study eye and confirmed by the CRC.
11. If study eye will be at the discretion of the Investigator and/or Surgeon.

General
Minimum age
12 Years
Maximum age
50 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
1. Have other known disease-causing mutations documented in the participant's medical history or identified through a retinal dystrophy gene panel, that in the opinion of the Investigator would interfere with the potential therapeutic effect of the study agent or the quality of the assessments.
2. For participants with herpes simplex virus (HSV):

1. Have history of oral or genital herpes and unable and/or unwilling to utilize prophylactic antiviral medication.
2. Have a history of ocular herpes.
3. Have active oral or genital herpes or are currently receiving treatment for HSV infection.
3. Have known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications.
4. Have used anti-coagulant agents that may alter coagulation (e.g., warfarin, heparin, apixaban, or high dose docosahexaenoic acid [DHA; fish oil]) within 7 days prior to study treatment administration (ibuprofen, aspirin, or similar are acceptable).
5. Have used systemic corticosteroids or other immunosuppressive medications within 3 months prior to screening and/or intend to use during screening. Corticosteroids used on an as-needed basis administered by insufflation, inhalation or local administration to the skin and mucosa such as Symbicort (budesonide/formoterol), Flonase (fluticasone propionate), and skin creams and ointments containing corticosteroids shall not be exclusionary.
6. If sexually active or planning to become sexually active, are unwilling to use barrier contraception for 3 months following treatment administration.
7. Are currently participating or recently participated in any other research
8. Have previously received any AAV gene therapy product, stem cell therapy, cell-based therapy, or similar biologics.
9. Have significant media opacity impacting evaluation of the retina or vitreous. administration.
10. Had intraocular surgery within 90 days of study treatment administration.
11. Have any active ocular/intraocular infection or inflammation (e.g., severe blepharitis, infectious conjunctivitis, keratitis, scleritis, endophthalmitis, idiopathic or autoimmune associated uveitis, or herpetic lesions).
12. Have a history of corticosteroid-induced raised IOP of >25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.
13. Have any artificial retinal implant or prosthesis.
14. Have absence of clear ocular media and/or inadequate pupil dilation to facilitate good quality SD-OCT images.
15. Have any history of rhegmatogenous retinal detachment.
16. Have myopia (spherical equivalent) exceeding -10 diopters (or axial length of >30 mm if the Principal Investigator [PI] deems it appropriate to measure) or presence of pathologic myopia in the study eye.
17. Have passed the Low Contrast Ora-VNC mobility course at =0.35 lux light level in either eye or binocularly at any screening visit.

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
14/03/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2029
Actual
Sample size
Target
75
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Sydney Eye Hospital - Sydney
Recruitment hospital [2] 0 0
Royal Victorian Eye & Ear Hospital - East Melbourne
Recruitment postcode(s) [1] 0 0
2000 - Sydney
Recruitment postcode(s) [2] 0 0
3002 - East Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Ohio
Country [5] 0 0
United States of America
State/province [5] 0 0
Oregon
Country [6] 0 0
United States of America
State/province [6] 0 0
Texas

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Beacon 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
Carrie Reichley
Address 0 0
Beacon Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Serva Health
Address 0 0
Country 0 0
Phone 0 0
855-467-2364
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/NCT04850118