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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01494805




Registration number
NCT01494805
Ethics application status
Date submitted
14/12/2011
Date registered
19/12/2011
Date last updated
1/09/2017

Titles & IDs
Public title
Safety and Efficacy Study of rAAV.sFlt-1 in Patients With Exudative Age-Related Macular Degeneration
Scientific title
A Phase I/II Controlled Dose-escalating Trial to Establish the Baseline Safety and Efficacy of a Single Subretinal Injection of rAAV.sFlt-1 Into Eyes of Patients With Exudative Age-related Macular Degeneration (AMD)
Secondary ID [1] 0 0
2008-135
Universal Trial Number (UTN)
Trial acronym
AMD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Macular Degeneration 0 0
Age-related Maculopathies 0 0
Age-related Maculopathy 0 0
Maculopathies,Age-related 0 0
Maculopathy,Age-related 0 0
Retinal Degeneration 0 0
Retinal Neovascularization 0 0
Eye Diseases 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - rAAV.sFlt-1
Treatment: Other - rAAV.sFlt-1
Other interventions - Control (ranibizumab alone)

Experimental: Low Dose rAAV.sFlt-1 -

Experimental: High Dose rAAV.sFlt-1 -

Active comparator: Control - ranibizumab only -


Treatment: Other: rAAV.sFlt-1
1 x 10\^10 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection

Treatment: Other: rAAV.sFlt-1
1 x 10\^11 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection

Other interventions: Control (ranibizumab alone)
Patients will not receive rAAV.sFlt-1, but will be eligible for retreatment with ranibizumab (Lucentis).
Intervention code [1] 0 0
Treatment: Other
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
No sign of unresolved ophthalmic complications, toxicity or systemic complications as measured by laboratory tests from 1 month post injection
Timepoint [1] 0 0
Primary endpoint at 1 month
Secondary outcome [1] 0 0
Maintenance or improvement of vision without the necessity of ranibizumab re-injections
Timepoint [1] 0 0
Up to 3 years

Eligibility
Key inclusion criteria
* Age greater than or equal to 55 years;
* Subfoveal CNV secondary to AMD and with best corrected visual acuity of 3/60 - 6/9 with 6/60 or better in the other eye;
* Fluorescein angiogram of the study eye must show evidence of a leaking subfoveal choroidal neovascular lesion, or CNV currently under active management with anti-VEGF therapy;
* Must be a candidate for anti-VEGF intravitreal injections;
* No previous retinal treatment of photodynamic therapy or laser;
* Able to provide informed consent;
* Able to comply with protocol requirements, including follow-up visits.
Minimum age
55 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Liver enzymes > 2 X upper limit of normal;
* Any prior treatment for AMD in the study / control eye, excluding anti-VEGF injections;
* Extensive sub-foveal scarring, extensive geographic atrophy, or thick subretinal blood in the study eye as determined by the investigator;
* Significant retinal disease other than sub-foveal CNV AMD;

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 assessing the outcomes
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/08/2017
Sample size
Target
Accrual to date
Final
40
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
Lions Eye Institute - Nedlands
Recruitment postcode(s) [1] 0 0
6009 - Nedlands

Funding & Sponsors
Primary sponsor type
Other
Name
Lions Eye Institute, Perth, Western Australia
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Adverum Biotechnologies, Inc.
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The study will involve approximately 40 subjects aged 55 or above who have exudative age-related macular degeneration (wet AMD). Patients will be randomized to receive one of two doses of rAAV.sFlt-1 or assigned to the control group.
Trial website
https://clinicaltrials.gov/study/NCT01494805
Trial related presentations / publications
Constable IJ, Pierce CM, Lai CM, Magno AL, Degli-Esposti MA, French MA, McAllister IL, Butler S, Barone SB, Schwartz SD, Blumenkranz MS, Rakoczy EP. Phase 2a Randomized Clinical Trial: Safety and Post Hoc Analysis of Subretinal rAAV.sFLT-1 for Wet Age-related Macular Degeneration. EBioMedicine. 2016 Dec;14:168-175. doi: 10.1016/j.ebiom.2016.11.016. Epub 2016 Nov 10.
Rakoczy EP, Lai CM, Magno AL, Wikstrom ME, French MA, Pierce CM, Schwartz SD, Blumenkranz MS, Chalberg TW, Degli-Esposti MA, Constable IJ. Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial. Lancet. 2015 Dec 12;386(10011):2395-403. doi: 10.1016/S0140-6736(15)00345-1. Epub 2015 Sep 30.
Public notes

Contacts
Principal investigator
Name 0 0
Ian Constable, Professor
Address 0 0
Lions Eye Institute
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01494805