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Clinical Trials/NL-OMON54872
NL-OMON54872WithdrawnPhase 2

Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of 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*) - EDIT-101 study in patients with LCA10

Editas Medicine, Inc.0 sites2 target enrollmentStarted: TBDLast updated:
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Conditions

Trial Snapshot

Phase
Phase 2
Status
Withdrawn
Enrollment
2
Study OverviewEligibility CriteriaInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

No summary available.

Study Design

Study Type
Interventional

Eligibility Criteria

Ages
2 to 64 (—)

Inclusion Criteria

  • •-Adult participants enrolling in Cohorts 1, 2, or 3 must be at least 18 years
  • •of age at the time of informed consent. Pediatric participants enrolling in
  • •Cohorts 4 or 5 must be 3 to 17 years of age, inclusive, at the time of informed
  • •-CEP290-related retinal degeneration caused by a homozygous or compound
  • •heterozygous mutation involving c.2991+1655A>G in IVS26 of the CEP290 gene
  • •confirmed by DNA sequencing (ie, 1 or 2 intron 26 c.2991+1655A>G mutations) and
  • •100% match for both gRNA and PAM sequences.
  • •-Male or Female.
  • •-A sexually mature male participant must agree to use contraception as detailed
  • •in Section 10.7 of this protocol from the time of informed consent through at
  • •least 12 months after study intervention, and to refrain from donating sperm
  • •during this period.
  • •-A female participant is eligible to participate if she is not pregnant (has a
  • •negative urine pregnancy result prior to study intervention), not
  • •breastfeeding, and at least one of the following conditions applies: Not a
  • •WOCBP as defined in Protocol Section 10.7, OR A WOCBP, or who reaches
  • •childbearing potential during the study, who agrees to follow
  • •the contraceptive guidance in Protocol Section 10.7 from the time of informed
  • •consent through at least 12 months after study intervention.
  • •-The participant (or guardian, in the case of a minor) must provide written
  • •informed consent prior to any study related procedures. Minors must provide
  • •assent in accordance with country and local regulations, as applicable.
  • •-Both eyes must be at least LP. The study eye will be the worse seeing eye and
  • •must meet the following BCVA criteria:
  • •Cohort 1: BWD, WFP, or LP
  • •Cohorts 2 - 5: LP to 0.4 logMAR (20/50 Snellen equivalent). ). Note: The
  • •sentinel participant in each of these cohorts will have severe vision loss with
  • •a logMAR BCVA of >=1.6 to 3.9 (20/800 or worse to LP) in the study eye.
  • •Subsequent participants in each cohort will have LP to 0.4 logMAR (20/50
  • •Snellen equivalent) best-corrected visual acuity in the study eye. If both eyes
  • •have the same BCVA but the worse seeing eye (as determined by the participant
  • •and the examiner) does not meet the above criteria, the better seeing eye may
  • •be designated as the study eye as long as the participant agrees and the better
  • •seeing eye meets all eligibility criteria.
  • •-Photoreceptor ONL identifiable in fovea by spectral domain OCT in the study
  • •-Able, as assessed by the investigator, and willing to complete study
  • •assessments and follow study instructions for the duration of the study.
  • •-If currently enrolled in Study EDIT-NHS01 (the Natural History Study of
  • •CEP290-Related Retinal Degeneration), the participant must agree to complete
  • •termination visit for that study, and be withdrawn from that study, before
  • •enrolling in this study.

Exclusion Criteria

  • •-Other known disease-causing mutations documented in the participant's medical
  • •history or identified through the retinal dystrophy gene panel evaluation
  • •performed at screening (including but not limited to bi-allelic mutations in
  • •other genes known to cause LCA) that, in the opinion of the investigator, would
  • •interfere with the potential therapeutic effect of the investigational product
  • •or the quality of the assessments.
  • •-Achieves a passing score for the Visual Function Navigation course at the
  • •maximum level of difficulty (ie, passes the most challenging Visual Function
  • •Navigation course under the
  • •dimmest lighting conditions) with either eye independently or both eyes
  • •-In either eye, cataract surgery in the last 3 months before the screening
  • •-In either eye, any active ocular/intraocular infection or inflammation (such
  • •as blepharitis, infectious conjunctivitis, keratitis, scleritis,
  • •endophthalmitis, idiopathic or autoimmune-associated uveitis, or herpetic
  • •lesions), assessed at screening.
  • •-In either eye, history of steroid-responsive intraocular pressure increases
  • •such that the affected eye had a pressure > 25 mm Hg following corticosteroid
  • •exposure despite topical
  • •intraocular-pressure-lowering pharmacologic therapy.
  • •-In either eye, Argus retinal implant.
  • •-In the study eye, absence of clear ocular media and adequate pupil dilation,
  • •assessed at screening, to permit good quality OCT images.
  • •-In the study eye, presence of vitreous hemorrhage.
  • •-In the study eye, any history of rhegmatogenous retinal detachment.
  • •-In the study eye, spherical equivalent of the refractive error demonstrating
  • •more than -8 diopters of myopia and more than +6 diopters of hyperopia (prior
  • •to cataract or refractive surgery), assessed at screening.
  • •-Uncontrolled diabetes mellitus (hemoglobin A1c >=10%) in the last 3 months
  • •before the screening visit or at screening.
  • •-Active gastric ulcer at screening.
  • •-Use of systemic immunosuppressive medications for any chronic disease in the
  • •last 3 months before the screening visit, or during the screening period.
  • •-Any vaccination/immunization in the last 28 days before screening, or during
  • •the screening period.
  • •-An inability or unwillingness to take the course of oral prednisone that is
  • •required in this study.
  • •-Current enrollment in an investigational interventional drug or device study
  • •(ocular or non-ocular) or participation in such a study within 6 months before
  • •the screening visit.
  • •(This does not include observational studies. Prior or current participation in
  • •Study EDIT-NHS01 is not an exclusion.)
  • •-Received prior gene therapy or oligonucleotide treatment of any kind.
  • •-The participant has a condition or is in a situation which, in the
  • •investigator's or operating surgeon's opinion, may put the participant at
  • •significant risk, may confound the study results, or may interfere
  • •significantly with the participant's participation in the study.

Investigators

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