Stargardt's DiseasePhase 2NCT01736592
Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration
A Phase 2 study testing Long term follow up in all patients who received SAR422459 in previous study TDU13583 in people with Stargardt's Disease, run by Sanofi at 2 sites in 2 countries.
Trial summary
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 Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Stargardt's Disease
- What is being tested?
- Long term follow up in all patients who received SAR422459 in previous study TDU13583
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 14 Dec 2012to 29 Aug 2033
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- The following would exclude Patients from participation in the study:
- 1. Did not receive SAR422459 as part of the TDU13583 protocol.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
Oregon Health and Science University Site Number : 840001
Portland, Oregon, 97239-3098
About this study
Official title: An Open Label Study to Determine the Long Term Safety, Tolerability and Biological Activity 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
Read the detailed description
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.
- Conditions
- Stargardt's Disease
- Being tested
- Long term follow up in all patients who received SAR422459 in previous study TDU13583
- Medical subject headings
- Stargardt Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Long term follow up in all patients who received SAR422459 in previous study TDU13583Drug
Blood draw for the laboratory assessment
| Study group | Role | What they receive |
|---|---|---|
| Long Term Follow up | Other | Long term follow up in all patients who received SAR422459 in previous study TDU13583 Receives: Drug: Long term follow up in all patients who received SAR422459 in previous study TDU13583 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence of Adverse Events
The number and percentage of patients with treatment emergent adverse events
Measured: 15 years
Secondary outcomes (2)
Clinically important changes in ocular safety assessments
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
Measured: baseline to 15 years
Delay in retinal degeneration
Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, static perimetry, microperimetry, autofluorescence, optical coherence tomography (OCT)
Measured: baseline to 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2012 (estimated)
- Study start14 Dec 2012
- Primary completion29 Aug 2033 (estimated)
- Study completion29 Aug 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stargardt's DiseaseStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01736592
- Sponsor's protocol ID
- LTS13588
- Other IDs
- SG1/002/11, 2024-513501-31, 2012-001990-95
- Status last verified
- Aug 2026
- First submitted
- 26 Nov 2012
- Last updated
- 26 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Study to Determine the Long Term Safety, Tolerability and Biological Activity of SAR422459 in Patients With Stargardt's Macular Degeneration
- ClinicalTrials.gov ID
- NCT01736592
- Acronym / sponsor ID
- LTS13588
- Conditions
- Stargardt's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 27 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 14 Dec 2012 (actual)
- Primary completion
- 29 Aug 2033 (estimated)
- Study completion
- 29 Aug 2033 (estimated)
- First posted
- 29 Nov 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
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.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Long Term Follow up | Other | Drug: Long term follow up in all patients who received SAR422459 in previous study TDU13583Long term follow up in all patients who received SAR422459 in previous study TDU13583 |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (2 listed)
- 1. Did not receive SAR422459 as part of the TDU13583 protocol.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Long term follow up in all patients who received SAR422459 in previous study TDU13583Blood draw for the laboratory assessment | Drug | Long Term Follow up |
Primary outcome
The incidence of Adverse Events
Time frame: 15 years
The number and percentage of patients with treatment emergent adverse events
2 secondary outcomes
Clinically important changes in ocular safety assessments
Time frame: baseline to 15 years
Delay in retinal degeneration
Time frame: baseline to 15 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2026.
- LTS13588 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Long term follow up in all patients who received SAR422459 in previous study TDU13583 in people with Stargardt's Disease, run by Sanofi at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence of Adverse Events (time frame: 15 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2026.