NCT01505062

Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

A Phase I/IIA Dose Escalation Safety Study of Subretinally Injected SAR421869, Administered to Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the safety and tolerability of ascending doses of subretinal injections of SAR421869 in participants with Usher syndrome type 1B. To evaluate for possible biological activity of SAR421869.

Detailed description

Following screening procedures, the gene transfer agent were injected once only under the retina by an opthalmic surgeon under anesthesia. Participants then had regular follow-up visits where general health examinations, blood tests and ophthalmic examinations including best corrected visual acuity, slit lamp examination, intraocular pressure, fundoscopy, autofluorescence, optical coherence tomography, perimetry and electroretinogram were undertaken. At the end of the study, the participants were invited to enter in an open-label safety study for long-term follow-up visits (at least once every six months) including ophthalmological examinations and recording of adverse events (AEs) were continued for 5 years; then the Investigator followed the participants by telephone for a subsequent 10 years at a minimum interval of once a year to monitor delayed AEs.

Conditions

• Usher Syndrome
• Retinitis Pigmentosa

Interventions

Type	Name	Description
DRUG	SAR421869	Formulation: Sterile suspension for intraocular injection, 100 microliters (μL) aliquots in 0.3 milliliter (mL) type I borosilicate glass 'V' vials with a butyl stopper and aluminum crimp seal. Route of administration: subretinal injection

Timeline

• Start date: 2012-03-26
• Primary completion: 2019-08-16
• Completion: 2019-08-16
• First posted: 2012-01-06
• Last updated: 2022-04-28
• Results posted: 2020-03-18

Locations

2 sites across 2 countries: United States, France