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Gene Therapy
Gene Therapy for Leber Congenital Amaurosis
Phase 1 & 2
Waitlist Available
Research Sponsored by Editas Medicine, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
Study Summary
This trial is testing a new gene therapy for people with a specific genetic mutation that causes Leber Congenital Amaurosis, a severe form of vision loss.
Who is the study for?
This trial is for males and females aged 3 or older with a specific genetic form of retinal degeneration (LCA10) due to mutations in the CEP290 gene. Participants must have severe vision loss, but not other disease-causing mutations or conditions that could affect the study's outcome like recent vaccinations, unwillingness to take oral prednisone, or prior similar treatments.Check my eligibility
What is being tested?
The trial tests EDIT-101 delivered through an injection into the retina for safety and effectiveness. It targets patients with LCA10 caused by certain CEP290 gene mutations. The study involves giving participants one dose of EDIT-101 and observing its effects on their condition.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include typical risks associated with subretinal injections such as eye inflammation, infection risk increase following corticosteroid exposure, and possible adverse reactions related to the EDIT-101 treatment.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am 3 years or older with a specific genetic eye condition.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Frequency of Adverse Events related to EDIT-101
Incidence of dose limiting toxicities
Number of participants experiencing procedural related adverse events
Secondary outcome measures
Change from baseline in LogMAR measurement of BCVA
Change from baseline in Mobility course score
Change from baseline in Patient Global Impressions of Change score
+13 moreTrial Design
5Treatment groups
Experimental Treatment
Group I: Pediatric Middle DoseExperimental Treatment1 Intervention
Single dose of EDIT-101 administered by subretinal injection surgery
Group II: Pediatric High DoseExperimental Treatment1 Intervention
Single dose of EDIT-101 administered by subretinal injection surgery
Group III: Adults Middle DoseExperimental Treatment1 Intervention
Single dose of EDIT-101 administered by subretinal injection surgery
Group IV: Adults Low DoseExperimental Treatment1 Intervention
Single dose of EDIT-101 administered by subretinal injection surgery
Group V: Adults High DoseExperimental Treatment1 Intervention
Single dose of EDIT-101 administered by subretinal injection surgery
Find a Location
Who is running the clinical trial?
Editas Medicine, Inc.Lead Sponsor
4 Previous Clinical Trials
134 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have not had any vaccinations in the last 28 days.I am 3 years or older with a specific genetic eye condition.I passed the hardest level of a mobility test.I have never received gene therapy or oligonucleotide treatment.The participant has very poor vision in the study eye, with a measurement of 20/800 or worse.I have other genetic mutations known to cause disease.I currently have an eye or systemic infection or inflammation.I cannot or do not want to take oral prednisone.My eye pressure increased above 25 mm Hg after using steroids.Participants, other than those specifically being studied, must have a visual acuity between 1.0 and 3.0 logMAR in the eye being studied.
Research Study Groups:
This trial has the following groups:- Group 1: Adults High Dose
- Group 2: Pediatric Middle Dose
- Group 3: Pediatric High Dose
- Group 4: Adults Middle Dose
- Group 5: Adults Low Dose
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is this research endeavor still open for participants to join?
"The specified clinical trial is still searching for potential subjects and was initially posted on September 26th 2019, with the latest update occurring on July 13th 2022."
Answered by AI
How many healthcare facilities are currently executing this clinical trial?
"There are currently 8 participating medical centres in this clinical trial, including the Casey Eye Institute - OSHU based in Portland, Oregon and Massachusetts Eye and Ear Infirmary situated in Boston. Additionally, W.K. Kellogg Eye Center - University of Michigan is located within Ann Arbor, Michigan along with 5 other sites throughout America."
Answered by AI
How many participants are currently included in this clinical trial?
"Editas Medicine, Inc. is running this clinical trial and needs 34 patients who meet the inclusion criteria to participate at two major locations: Casey Eye Institute - OSHU in Portland, Oregon and Massachusetts Eye and Ear Infirmary in Boston, Massachusetts."
Answered by AI
Who else is applying?
What site did they apply to?
Bascom Palmer Eye Institute
What portion of applicants met pre-screening criteria?
Met criteria
Why did patients apply to this trial?
Need to know about my visual condicionando posible cure in the future.
PatientReceived 2+ prior treatments
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