Clinical Trial

Disease: Blindness, Leber Congenital Amaurosis, (NCT03872479)

Disease info:

Leber congenital amaurosis is an eye disorder that primarily affects the retina, which is the specialized tissue at the back of the eye that detects light and color. People with this disorder typically have severe visual impairment beginning in infancy.

Frequency:
Leber congenital amaurosis occurs in 2 to 3 per 100,000 newborns. It is one of the most common causes of blindness in children.
Official title:
Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of AGN-151587 (EDIT-101) in Adult and Pediatric Participants With Leber Congenital Amaurosis Type 10 (LCA10), With Centrosomal Protein 290 (CEP290)-Related Retinal
Who:

Study Director: Francisco Lopez, MD, PhD Allergan

Sponsor:

Editas Medicine, Inc.

Partners:
Locations:

United States, Florida

United States, Massachusetts

United States, Michigan

United States, Oregon

 

Study start:
Sep. 26, 2019
Enrollment:
18 participants
Gene editing method:
CRISPR-cas9
Type of edit:
Gene correction
Gene:
Centrosomal Protein 290 (CEP290)
Delivery method:
Adeno-associated virus (AAV5) - In-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/record/NCT03872479
Safety updates:

29-09-2021 Editas Medicine Announces Positive Initial Clinical Data From Ongoing Phase 1/2 BRILLIANCE Clinical Trial Of EDIT-101 For LCA10

23-06-2021 Editas Medicine Announces Enrollment Of The First Pediatric Cohort In The BRILLIANCE Clinical Trial Of EDIT-101 For The Treatment Of LCA10 Following IDMC Endorsement

04-03-2020 Allergan and Editas Medicine Announce Dosing of First Patient in Landmark Phase 1/2 Clinical Trial of CRISPR Medicine AGN-151587 (EDIT-101) for the Treatment of LCA10

Other links:

LinkLeber congenital amaurosis disease informationLinkNews story: CRISPR treatment inserted directly into the body for first timeLinkDevelopment of AGN 151587 (EDIT101), a gene editing approach to restore vision in Leber Congenital Amaurosis Type 10LinkPreclinical Assessment of In Vivo Gene Editing Efficiency, Specificity, and Tolerability of EDIT-101, an Investigational CRISPR Treatment for Leber Congenital Amaurosis 10 (LCA10)LinkDisease Roundup: Leber Congenital Amaurosis

Note:
EDIT-101 uses an AAV5 vector to deliver the Staphylococcus aureus Cas9 and CEP290-specific guide RNAs (gRNAs) to photoreceptor cells by subretinal injection
IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active not recruiting

Description

The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of AGN-151587 (EDIT-101) administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A>G in intron 26 of the CEP290 gene ("LCA10-IVS26"). AGN-151587 is a novel gene editing product designed to eliminate the mutation on the CEP290 gene that results in the retinal degeneration that defines LCA10-IVS26.

Last updated: Jan. 1, 2023
Source: US National Institutes of Health (NIH)
clinicaltrials.gov
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