A Phase 2/3 interventional study of sepofarsen and Sham in Leber Congenital Amaurosis 10, Blindness and Leber Congenital Amaurosis, sponsored by ProQR Therapeutics. Active, not recruiting at 14 sites in 9 countries. Open to participants aged 8 Years and older. Per ClinicalTrials.gov, last updated 2022-03-17.
Sponsored by ProQR Therapeutics · Phase 2/3, Interventional, and Treatment
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
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.
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.
144 studies on the registry are indexed under Blindness; 33 are open to participants now.
This study's enrollment of 36 is close to the median of 37 across 101 interventional studies indexed under Blindness.
Browse Blindness studies →ProQR Therapeutics is the lead sponsor of 7 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Main Inclusion Criteria Relating to Study Initiation:
Main Exclusion Criteria Relating to Study Initiation:
Main Inclusion Criteria Relating to Treatment Initiation Contralateral Eye:
Main Exclusion Criteria Relating to Treatment Initiation Contralateral Eye:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Drug: sepofarsen
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
Drug: sepofarsen
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
Other: Sham
RNA antisense oligonucleotide for intravitreal injection
Also known as: QR-110
Sham-Procedure (no experimental drug administered)
Change in BCVA
Change in Best-corrected visual acuity (BCVA) relative to baseline after 12 months of treatment versus sham-procedure
Time frame: 12 months
Change from baseline in BCVA ≤ -0.3 LogMAR
Change from baseline in BCVA in subjects with BCVA better than 1.7 Logarithm of the minimum angle of resolution (LogMAR) at baseline
Time frame: 12 and 24 months
Clinical meaningful improvement in subjects with BCVA ≤ 1.7 LogMAR
Change from baseline in BCVA by a clinically meaningful improvement in subjects with BCVA equal to or worse than 1.7 LogMAR at baseline.
Time frame: 12 and 24 months
Change in BCVA based on FrACT
Change from baseline in BCVA based on Freiburg visual acuity and contrast test (FrACT)
Time frame: 12 and 24 months
Change in mobility course score
Change from baseline in mobility course score
Time frame: 12 and 24 months
Change in ellipsoid zone (EZ) width/area assessed by SD-OCT
Change from baseline in ellipsoid zone (EZ) width/area assessed by SD-OCT
Time frame: 12 and 24 months
Change in oculomotor instability (OCI)
Change in oculomotor instability from baseline
Time frame: 12 and 24 months
Change in FST light sensitivity
Change from baseline in light sensitivity Full-field light sensitivity threshold (FST) testing (white, red, blue)
Time frame: 12 and 24 months
Change in LLVA
Change from baseline in low luminance visual acuity (LLVA)
Time frame: 12 and 24 months
Change in patient reported visual function via VFQ-25 (adults)
Change in patient reported visual function, as measured by the Visual Function Questionnaire-25 (VFQ-25) score for adult subjects relative to baseline
Time frame: 12 and 24 months
Change in patient reported visual function via CVAQC (pediatrics)
Change in patient reported visual function, as measured by the Cardiff Visual Ability Questionnaire for Children (CVAQC) for pediatric subjects relative to baseline
Time frame: 12 and 24 months
Change in the Patient Global Impressions of Severity (PGI-S)
Change in the patient-reported outcome (PRO) Patient Global Impressions of Severity (PGI-S)
Time frame: 12 and 24 months
Change in the Patient Global Impressions of Change (PGI-C)
Change in the PRO Patient Global Impressions of Change (PGI-C)
Time frame: 12 and 24 months
Change in FAF
Change from baseline as determined by fundus autofluorescence (FAF) imaging
Time frame: 12 and 24 months
Changes in microperimetry
Change from baseline as determined by microperimetry
Time frame: 12 and 24 months
Systemic exposure to QR-110
Systemic exposure to QR-110
Time frame: 12 and 24 months
Ocular and non-ocular AEs
Frequency and severity of ocular and non-ocular AEs
Time frame: 12 and 24 months
Plan to share: No
Ran from Apr 2019 to primary completion in Jan 2022, 33 months.
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This study is active, not recruiting, as verified in Feb 2022. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Sepofarsen
ProQR Therapeutics