Phase
Condition
Retinitis Pigmentosa
Retina
Eye Disorders/infections
Treatment
No intervention, will not receive any active study intervention
Intravitreal Injection of Ultevursen
Clinical Study ID
Ages > 8 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
An adult (≥18 years) willing and able to provide informed consent for participationprior to performing any study related procedures
OR A minor (8 to <18 years) able to provide age-appropriate assent for studyparticipation with a parent or legal guardian willing and able to provide writtenpermission for the subject's participation prior to performing any study relatedprocedures. An adult willing to comply with the protocol, follow study instructions,attend study visits as required and willing and able to complete all studyassessments, in the opinion of the Investigator. OR A minor able to complete all study assessments and comply with the protocol andhas a parent or caregiver willing and able to follow study instructions and attendstudy visits with the subject as required, in the opinion of the Investigator.
Both eyes exhibit clinical presentation consistent with RP involving Usher syndrometype 2 or NSRP based on ophthalmic, audiologic, or vestibular examinations. Atscreening, the Investigator will make the clinical diagnosis of "Usher syndrome type 2a," defined as RP with congenital hearing loss, or "non-syndromic RP," defined asRP without congenital hearing loss.
A molecular diagnosis of biallelic disease causing variants (pathogenic or likelypathogenic) in the USH2A gene where at least one of the variants is located on exon
A historic genotyping report from a certified laboratory is acceptable withSponsor approval.
Clearly visible and measurable SD-OCT horizontal EZ width of ≥2.2 mm in both eyesbased on the assessment of the CRC.
BCVA ≥55 letters based on ETDRS (equivalent to 20/80 based on Snellen notation, orlogarithm of the minimum angle of resolution [logMAR] +0.6) in both eyes.
Impairment of VF as assessed by SP with a mean sensitivity greater than 4 decibels (dB) and less than 25 dB measured by a V target size in the TE at screening.
Mean sensitivity greater than 2 dB as determined by MP in the TE at screening.
Symmetry of baseline disease in both eyes, defined as the mean BCVA (based on ETDRS)of one eye within ≤10 letters of the mean BCVA of the other eye at screening.
Exclusion
Exclusion Criteria:
Presence of additional non-exon 13 USH2A pathogenic or likely pathogenic variant onthe USH2A allele carrying the exon 13 mutation in subjects who have one exon 13disease causing variant and one non-exon 13 disease causing variant.
Presence of additional non-exon 13 USH2A pathogenic mutation(s) on both USH2Aalleles in subjects who have biallelic exon 13 mutations.
Presence of pathogenic or likely pathogenic variants in genes (other than the USH2Agene) which are known to be associated with other inherited retinal degenerativediseases or syndromes. Specifically, the presence of homozygous or compoundheterozygous known disease-causing mutations in other genes involved in recessiveretinal dystrophies, or the confirmed presence of a known single disease-causingvariant in genes involved in dominant, X-linked, or mitochondrial retinal dystrophygenes is exclusionary.
At screening, the EZ horizontal or vertical width are outside the field of theSD-OCT scan based on the assessment of the CRC.
Presence of any significant ocular or non-ocular disease/disorder (includingmedication and laboratory test abnormalities) which, in the opinion of theInvestigator may either put the subject at risk because of participation in thestudy, may impact the subject's ability to participate in the study, or mayinterfere with assessment of efficacy and safety in the study.
Presence of unstable concurrent cystoid macular edema (CME), or subject started on (or changed dose of) any medication for CME in the 3 months prior to enrollment. CMEis allowed if stable for 3 months (with or without treatment). However, stable CMEthat disrupts the EZ width measurement, as determined by CRC, is an exclusion.
Any intraocular surgery within 3 months of study entry or any planned intraocular orperi-ocular surgery during the study. Subjects may be eligible after 3 monthspost-surgery as long as they have fully recovered, in the opinion of theInvestigator.
Receipt of any IVT injection prior to study entry.
Study Design
Study Description
Connect with a study center
Ghent University Hospital
Ghent 2797656, B-9000
BelgiumActive - Recruiting
Hospital for Sick Children
Toronto 6167865, Ontario 6093943 M5G1E8
CanadaActive - Recruiting
McGill University Health Centre for Innovative Medicine
Montreal 6077243, Quebec 6115047 H4A3J1
CanadaActive - Recruiting
Rigshospitalet and University of Copenhagen
Glostrup Municipality 2621356, 2600
DenmarkActive - Recruiting
Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique
Montpellier 2992166, 34295
FranceActive - Recruiting
Centre de maladies rares CHNO des Quinze Vingt
Paris 2988507, 75012
FranceActive - Recruiting
Universitätsklinikum Tübingen
Tübingen 2820860, 72076
GermanyActive - Recruiting
ASST Santi Paolo e Carlo Hospital, University of Milan
Milan 3173435, 20142
ItalyActive - Recruiting
AOU Università degli Studi della Campania Luigi Vanvitelli
Napoli 9031661, 80131
ItalySite Not Available
Amsterdam University Medical Center - Locatie AMC
Amsterdam 2759794, 1105 AZ
NetherlandsActive - Recruiting
Radboud Universitair Medisch Centrum
Nijmegen 2750053, 6525 GA
NetherlandsActive - Recruiting
Het Oogziekenhuis Rotterdam
Rotterdam 2747891, 3011 BH
NetherlandsActive - Recruiting
Oxford Eye Hospital
Headington, Oxford OX3 9DU
United KingdomSite Not Available
Oxford Eye Hospital
Headington 2647239, Oxford OX3 9DU
United KingdomActive - Recruiting
University of Edinburgh / NHS Lothian
Edinburgh 2650225, EH39HA
United KingdomActive - Recruiting
Moorfields Eye Hosptial
London, EC1V 2PD
United KingdomSite Not Available
Moorfields Eye Hosptial
London 2643743, EC1V 2PD
United KingdomActive - Recruiting
The University of California, San Francisco
San Francisco 5391959, California 5332921 94143
United StatesActive - Recruiting
Bascom Palmer Eye Institute/University of Miami
Miami 4164138, Florida 4155751 33136
United StatesActive - Recruiting
Emory University
Atlanta 4180439, Georgia 4197000 30322
United StatesActive - Recruiting
Massachusetts Eye and Ear
Boston, Massachusetts 02114
United StatesSite Not Available
Massachusetts Eye and Ear
Boston 4930956, Massachusetts 6254926 02114
United StatesActive - Recruiting
University of Michigan- Kellogg Eye Center
Ann Arbor, Michigan 48105
United StatesSite Not Available
University of Michigan- Kellogg Eye Center
Ann Arbor 4984247, Michigan 5001836 48105
United StatesActive - Recruiting
Casey Eye Institute, Oregon Health & Science University
Portland, Oregon 97239
United StatesSite Not Available
Casey Eye Institute, Oregon Health & Science University
Portland 5746545, Oregon 5744337 97239
United StatesActive - Recruiting
University of Pennsylvania, Scheie Eye Institute
Philadelphia 4560349, Pennsylvania 6254927 19104
United StatesActive - Recruiting
Retina Foundation of the Southwest
Dallas, Texas 75231
United StatesSite Not Available
Retina Foundation of the Southwest
Dallas 4684888, Texas 4736286 75231
United StatesActive - Recruiting
Baylor College of Medicine
Houston 4699066, Texas 4736286 77030
United StatesActive - Recruiting
University of Wisconsin- Madison
Madison, Wisconsin 53705
United StatesSite Not Available
University of Wisconsin- Madison
Madison 5261457, Wisconsin 5279468 53705
United StatesActive - Recruiting

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