A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
NCT ID: NCT03913143
Last Updated: 2022-03-17
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2/PHASE3
36 participants
INTERVENTIONAL
2019-04-04
2023-03-31
Brief Summary
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Detailed Description
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At study start subjects will be randomized to one of 3 treatment groups with either active study drug or sham treatment.
Sepofarsen (QR-110) will be administered via intravitreal (IVT) injection into the subject's treatment eye (the subject's worse eye).
Subjects in the sham-procedure group will undergo a procedure that will closely mimic the active injection.
After each dosing subjects will be assessed for safety and tolerability at follow up visits.
After the first eye has been treated for at least 12 months, treatment of the contralateral eye and cross-over of subjects assigned to sham procedure may be initiated in eligible eyes (in a masked manner) based on assessment of benefit/risk (including review of data from all clinical trials), and with concurrence of the Medical Monitor.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Group 1: Dose 1 sepofarsen (QR-110)
Initial loading dose, followed by maintenance doses at month 3 and every 6 months there after, administered by intravitreal injection (24 months duration of treatment). After 12 months treatment of the contralateral eye may be initiated
sepofarsen
RNA antisense oligonucleotide for intravitreal injection
Group 2: Dose 2 sepofarsen (QR-110)
Initial loading dose, followed by maintenance doses at month 3 and every 6 months there after, administered by intravitreal injection (24 months duration of treatment). After 12 months treatment of the contralateral eye may be initiated
sepofarsen
RNA antisense oligonucleotide for intravitreal injection
Group 3: Sham
Sham procedure (no experimental drug administered), Day 1, month 3 and every six months there after. After 12 months cross over to active study drug may be initiated
Sham
Sham-Procedure (no experimental drug administered)
Interventions
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sepofarsen
RNA antisense oligonucleotide for intravitreal injection
Sham
Sham-Procedure (no experimental drug administered)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
* 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.
Exclusion Criteria
* 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.
* 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.
8 Years
ALL
No
Sponsors
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ProQR Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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ProQR Medical Monitor
Role: STUDY_DIRECTOR
ProQR Therapeutics
Locations
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University of Iowa
Iowa City, Iowa, United States
Universitair Ziekenhuis Gent (UZ)
Ghent, , Belgium
INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte
Belo Horizonte, Minas Gerais, Brazil
Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)
São Paulo, São Paulo, Brazil
The Hospital for Sick Children - SickKids
Toronto, Ontario, Canada
McGill University Health Centre - Centre for Innovative Medicine
Montreal, Quebec, Canada
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
Countries
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Related Links
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Sponsor website
Other Identifiers
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2018-003501-25
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
PQ-110-003
Identifier Type: -
Identifier Source: org_study_id
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