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Basic Information Clinical Results Relationship Graph

Basic Information

Gene symbol	KDR	Synonyms	CD309, FLK1, VEGFR, VEGFR2	Type of gene	protein-coding
Chromosome	4	Map location	4q12	dbXrefs
Gene perturbation-related omics dataset	PertOrg
Description	kinase insert domain receptor

GTO ID	GTC0131
Trial ID	NCT01494805
Disease	Age-Related Macular Degeneration
Altered gene	VEGFR
Therapeutic/Target gene	Therapeutic gene
Therapy	Gene transfer
Treatment	rAAV.sFlt-1
Phase	Phase1\|Phase2
Recruitment status	Completed
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)
Year	2011
Country	Australia
Company sponsor	Lions Eye Institute, Perth, Western Australia
Other ID(s)	2008-135

Vector information

Vector	adeno-associated virus
Construct	rAAV.sFlt-1
Vector type	recombinant adeno-associated virus (rAAV) vector
Transgene/Inserted gene	sFLT-1, is the soluble form of the VEGFR1 receptor, which binds and inactivates VEGF-A, VEGF-B, and PlGF through its domain 2

Clinical Result

Cohort1: dose level 1

Administration route	subretinal injection
Dosage	1E10 vg
Pts	3
Age	Adult, Older_Adult
Outcome	In the low-dose group, BCVA improved significantly, with patient 1 improving from 33 to 40 letters, patient 2 from 28 to 41 letters, and patient 4 from 46 to 58 letters after cataract removal. The median baseline BCVA was 40 ETDRS letters and the median CPT decreased from 549 μm to 311 μm at week 52.
Adverse reactions	No serious clinical adverse events
References	PMID: 26431823

Cohort2: dose level 2

Administration route	subretinal injection
Dosage	1E11 vg
Pts	3
Age	Adult, Older_Adult
Outcome	In the high-dose group, patient 5's BCVA improved to baseline levels despite initial fibrovascular scarring, patient 6 improved from 54 to 64 letters, and patient 8 from 34 to 49 letters. The median baseline BCVA was 40 ETDRS letters, improving to 50 letters at week 52, with median CPT decreasing from 549 μm to 311 μm.
Adverse reactions	No serious clinical adverse events
References	PMID: 26431823

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