Efficacy and Safety of BIIB111 for the Treatment of Choroideremia

NCTID	NCT03496012 (View at clinicaltrials.gov)
Description	The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of BIIB111 in participants with choroideremia (CHM). (Show More)
Development Status	Inactive
Indication	Choroideremia
Disease Ontology Term	DOID:9821
Compound Name	BIIB111
Compound Alias	Timrepigene emparvovec
Sponsor	Biogen
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	169
Results Posted	View Results

Target Gene/Variant	CHM
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type	none
Dose 1	1.0E10 gp
Dose 2	1.0E11 gp
Dose 3
Dose 4
Dose 5

No.of Trial Sites	18
Locations	Canada,Netherlands,United States,Finland,Denmark,United Kingdom,France,Germany

Has US IND	True
FDA Designations	Regenerative Medicine Advanced Therapy
Recent Updates	Phase III study did not meet its primary endpoint or demonstrate efficacy on key secondary endpoints