sepofarsen: RNA antisense oligonucleotide for intravitreal injection
Sham: Sham-Procedure (no experimental drug administered)
Study summary
The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment
Eligibility
Sex
ALL
Min age
8 Years
Max age
—
Healthy volunteers
No
Main Inclusion Criteria Relating to Study Initiation:
* Male or female, ≥ 8 years of age at Screening with a clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A\>G mutation, based on genotyping analysis at Screening. A historic genotyping report from a certified laboratory is acceptable with Sponsor approval.
* BCVA better or equal to Logarithm of the Minimum Angle of Resolution (LogMAR) +3.0 (Hand Motion), and equal to or worse than LogMAR +0.4 in the treatment eye.
* Detectable outer nuclear layer (ONL) in the area of the macula.
* An electroretinogram (ERG) result consistent with LCA. A historic ERG result may be acceptable for eligibility.
Main Exclusion Criteria Relating to Study Initiation:
* Presence of any significant ocular or non-ocular disease/disorder (including medication and laboratory test abnormalities).
* Prior receipt of intraocular surgery, periocular surgery, or IVT injection within 1 month prior to study start or planned intraocular surgery or procedure during the course of the study.Subjects who received an intraocular or periocular surgery between 1 to 3 months prior Screening, may only be considered for inclusion if there are no clinically significant complications of surgery present, and following approval by the Medical Monitor.
* History or presence of ocular herpetic diseases.
* Presence of any active ocular infection in the either eye.
* Presence of lens opacities/cataracts in the treatment eye.
* Current treatment or treatment within the past 12 months with therapies known to influence the immune system.
* History of glaucoma, or an IOP greater than 24 mmHg, at is not controlled with medication.
* History of amblyopia
* Use of any investigational drug or device within 90 days or 5 half-lives of Day 1, whichever is longer, or plans to participate in another study of a drug or device during the PQ-110-003 study period.
* Any prior receipt of genetic or stem-cell therapy.
* Known hypersensitivity to antisense oligonucleotides or any constituents of the injection.
* Pregnant and breastfeeding subjects.
Main Inclusion Criteria Relating to Treatment Initiation Contralateral Eye:
* BCVA equal to or better than LP (logMAR +4), using the best BCVA reading at Month 12 and based on ETDRS or BRVT.
* Detectable outer nuclear layer (ONL) in the area of the macula.
* Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging.
Main Exclusion Criteria Relating to Treatment Initiation Contralateral Eye:
* Presence of any significant ocular or non-ocular disease/disorder (including medication and laboratory test abnormalities).
* History or presence of ocular herpetic diseases.
* Presence of any active ocular infection in either eye.
* Presence of any lens opacities which are clinically significant, would adequately prevent clinical and photographic evaluation of the retina.
* A planned IVT injection or intraocular or periocular surgery/procedure (including refractive surgery) during the course of the study.
* A history of glaucoma or an IOP greater than 24 mmHg that is not controlled with medication.
* History of amblyopia.
* Plans to participate in another study of a drug or device during the study period.
* Pregnant and breastfeeding subjects.
Primary outcome measure(s)
Change in BCVA — 12 months Change in Best-corrected visual acuity (BCVA) relative to baseline after 12 months of treatment versus sham-procedure
Trial sites (14)
Facility
City
Region
Status
University of Iowa
Iowa City
Iowa
Universitair Ziekenhuis Gent (UZ)
Ghent
Belgium
INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte
Belo Horizonte
Minas Gerais
Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)
São Paulo
São Paulo
The Hospital for Sick Children - SickKids
Toronto
Ontario
McGill University Health Centre - Centre for Innovative Medicine
Montreal
Quebec
Centre de maladies rares CHNO des Quinze Vingt
Paris
France
Hospital Civil de Strasbourg
Strasbourg
France
Justus-Liebig Universität - Department of Ophthalmology
Giessen
Germany
University of Tuebingen - Inst. for Ophthalmic Research
Tübingen
Germany
Eye Clinic University of Campania Luigi Vanvitelli
Naples
Italy
Amsterdam University Medica Center - Locatie AMC
Amsterdam
Netherlands
Het Oogziekenhuis Rotterdam
Rotterdam
Netherlands
Moorfields Eye Hospital - NHS Foundation Trust
London
United Kingdom
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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