Single Ascending Dose Study in Participants With LCA10
| Status: | Not yet recruiting |
|---|---|
| Conditions: | Ocular |
| Therapuetic Areas: | Ophthalmology |
| Healthy: | No |
| Age Range: | 3 - Any |
| Updated: | 3/15/2019 |
| Start Date: | June 3, 2019 |
| End Date: | March 22, 2024 |
| Contact: | Clinical Trials Registry Team |
| Email: | IR-CTRegistration@allergan.com |
| Phone: | 1-877-277-8566 |
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 Degeneration Caused by a Compound Heterozygous or Homozygous Mutation Involving c.2991+1655A>G in Intron 26 (IVS26) of the CEP290 Gene ("LCA10-IVS26")
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").
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").
This is an open-label, single ascending dose study of AGN-151587 (EDIT-101) in adult and
pediatric (ie, ages 3 to 17) participants with LCA10-IVS26. Approximately 18 participants
will be enrolled in up to 5 cohorts to evaluate up to 3 dose levels of AGN-151587 in this
study. 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.
pediatric (ie, ages 3 to 17) participants with LCA10-IVS26. Approximately 18 participants
will be enrolled in up to 5 cohorts to evaluate up to 3 dose levels of AGN-151587 in this
study. 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.
Inclusion Criteria:
- Male or female
- At least 3 years of age at screening with CEP290-related retinal degeneration caused
by a homozygous or compound heterozygous mutation involving c.2991+1655A>G in IVS26 of
the CEP290 gene.
- Best corrected visual acuity of light perception to 0.4 logMAR (20/50 Snellen
equivalent).
Exclusion Criteria:
- Other known disease-causing mutations
- Achieves a passing score for the mobility course at the most difficult level
- In either eye, active systemic or ocular/intraocular infection or inflammation
- In either eye, history of steroid-responsive intraocular pressure with increases > 25
mm Hg following corticosteroid exposure
- Any vaccination/immunization in the last 28 days before screening
- Inability or unwillingness to take oral prednisone
- Prior gene therapy or oligonucleotide treatment
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