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NCT Number: NCT01736592

Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration

Primary Objective:

To evaluate the long-term safety and tolerability of SAR422459 in patients with Stargardt's Macular Degeneration.

Secondary Objective:

To assess:

* Safety * Biological activity

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Stargardt's Disease Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Macular Degeneration Retinal Degeneration Retinal Diseases Stargardt Disease

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Investigational Site Number : 250001, Paris, France

Loading trial locations.

About this study

Patients will be followed for 15 years after completion/early termination from the previous TDU13583 study (NCT01367444).

As the initial TDU13583 study is terminated - recruitment to this LTS13588 study is over with 27 patients enrolled.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Patients must meet ALL of the following criteria:

  • Provide signed and dated written informed consent and any locally required authorization (e.g., Health Insurance Portability and Accountability Act [HIPAA])
  • Must have been enrolled in protocol TDU13583 (SG1/001/10)
  • Must have received a subretinal injection of SAR422459
  • Must have completed protocol TDU13583 to Week 48 or undergone an early discontinuation visit.

Exclusion criteria

The following would exclude Patients from participation in the study:

  • Did not receive SAR422459 as part of the TDU13583 protocol.

Treatment and study plan

Long term follow up in all patients who received SAR422459 in previous study TDU13583

Drug

Blood draw for the laboratory assessment

Primary outcomes

  1. The incidence of Adverse Events

    Time frame: 15 years

    The number and percentage of patients with treatment emergent adverse events

Secondary outcomes

  1. Clinically important changes in ocular safety assessments

    Time frame: baseline to 15 years

    From baseline in (TDU13583) study and from the last visit best-corrected visual acuity (BCVA), slit-lamp examination, Fundososcopy, intraocular pressure, laboratory parameters, concomitant medications

  2. Delay in retinal degeneration

    Time frame: baseline to 15 years

    Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, static perimetry, microperimetry, autofluorescence, optical coherence tomography (OCT)

Sponsors and collaborators

Lead sponsor

Sanofi

Industry

Registry information

Official study title

An Open Label Study to Determine the Long Term Safety, Tolerability and Biological Activity of SAR422459 in Patients With Stargardt's Macular Degeneration

Important dates

Study start
2012
Primary completion
2033
Study completion
2033
First posted
Nov 29, 2012
Registry last updated
Jun 12, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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