A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients With Usher Syndrome Type 1B

An Open-Label Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients with Usher Syndrome type 1B

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-000597-29-FR

Enrollment

Registered

2016-04-12

Start date

2015-09-30

Completion date

Unknown

Last updated

2024-09-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Usher syndrome type 1B MedDRA version: 18.1 Level: PT Classification code 10063396 Term: Usher's syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: UshStat Product Code: SAR421869 Pharmaceutical Form: Suspension for injection

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Provide signed and dated written informed consent (and if appropriate assent) and any locally required authorization eg, Health Insurance Portability and Accountability Act (HIPAA). Must have been enrolled in protocol TDU13600. Must have received a subretinal injection of UshStat®. Are the trial subjects under 18? yes Number of subjects for this age range: 2 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 16 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Did not receive UshStat® as part of the TDU13600 protocol.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the long-term safety and tolerability of UshStat® in patients with Usher syndrome type 1B;Secondary Objective: To assess long-term safety and biological activity of UshStat®;Primary end point(s): The incidence of adverse events The number and percentage of patients with treatment emergent adverse events ;Timepoint(s) of evaluation of this end point: During the first 5 years of follow up, every six months, then on an annual basis for a subsequent 10 years	—

Measure

Time frame

Main Objective: To evaluate the long-term safety and tolerability of UshStat® in patients with Usher syndrome type 1B;Secondary Objective: To assess long-term safety and biological activity of UshStat®;Primary end point(s): The incidence of adverse events The number and percentage of patients with treatment emergent adverse events ;Timepoint(s) of evaluation of this end point: During the first 5 years of follow up, every six months, then on an annual basis for a subsequent 10 years

—

Secondary

Measure	Time frame
Secondary end point(s): 1- Change from baseline in ocular safety assessments Best Corrected Visual Acuity (BCVA), Slit-lamp, Fundusoscopy, Intraocular Pressure, Lab parameters, concomitant meds 2- Delay in retinal degeneration Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, kinetic, static perimetry, microperimetry, autofluorescence, ERG, OCT ;Timepoint(s) of evaluation of this end point: 1-2 : During the first 5 years of follow up, every six months, then on an annual basis for a subsequent 10 years	—

Measure

Time frame

Secondary end point(s): 1- Change from baseline in ocular safety assessments Best Corrected Visual Acuity (BCVA), Slit-lamp, Fundusoscopy, Intraocular Pressure, Lab parameters, concomitant meds 2- Delay in retinal degeneration Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, kinetic, static perimetry, microperimetry, autofluorescence, ERG, OCT ;Timepoint(s) of evaluation of this end point: 1-2 : During the first 5 years of follow up, every six months, then on an annual basis for a subsequent 10 years

—

Countries

France, United States

Contacts

Public ContactDirection des Opérations Cliniques

sanofi-aventis France

Public-registry-MA@sanofi.com0800 222 555

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026