Phase I/IIA Study of SAR422459 in Participants With Stargardt's Macular Degeneration
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| ClinicalTrials.gov Identifier: NCT01367444 |
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Recruitment Status :
Terminated
(Study stopped not for safety reasons. Due to review of clinical development plans and priorities, Sponsor decided to stop development of the product.)
First Posted : June 7, 2011
Results First Posted : June 5, 2020
Last Update Posted : April 14, 2022
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Sponsor:
Sanofi
Information provided by (Responsible Party):
Sanofi
- Study Details
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| Study Type | Interventional |
|---|---|
| Study Design | Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
| Condition |
Stargardt's Disease |
| Intervention |
Drug: SAR422459 |
| Enrollment | 27 |
Participant Flow
| Recruitment Details | The Study was conducted in France and the United States. The trial was ended prematurely and the end of trial date declaration to health authorities was 16-August-2019. |
| Pre-assignment Details | A total of 35 participants who had Stargardt's Macular Degeneration (SMD) were screened, out of which 27 participants were enrolled in 7 cohorts (Cohorts 1 to 7). |
| Arm/Group Title | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 | Cohort 6 | Cohort 7 |
|---|---|---|---|---|---|---|---|
 Arm/Group Description |
Participants (aged greater than or equal to [>=] 18 years) with advanced SMD, and visual acuity (VA) less than or equal to (<=) 20/200 in the worst eye and severe cone-rod dysfunction with no detectable or severely abnormal full-field electroretinography (ERG) responses, received SAR422459 at lowest target dose level 1.8*10^5 transducing units (TU)/study eye. | Participants (aged >=18 years) with SMD, and VA <=20/200 in the worst eye with abnormal full-field ERG responses, received SAR422459 at lowest target dose level 1.8*10^5 TU/study eye. | Participants (aged >=18 years) with SMD, and VA <=20/200 in the worst eye with abnormal full-field ERG responses, received SAR422459 at escalated target dose level 6*10^5 TU/study eye. | Participants (aged >=18 years) with SMD, and VA <=20/200 in the worst eye with abnormal full-field ERG responses, received SAR422459 at target dose level 1.8*10^6 TU/study eye. | Participants (aged >=18 years) with SMD, and VA <=20/100 in the worst eye with abnormal full-field ERG responses, received SAR422459 at highest target dose level 1.8*10^6 TU/study eye. | Participants (aged 6 to 26 years) with symptomatic early or childhood-onset SMD, and VA >=20/200 in both eyes at the time of the screening visit and anticipated to experience rapid deterioration in visual function and/or retinal structure, received SAR422459 at highest target dose level 1.8*10^6 TU/study eye. | Pediatric participants (aged 6 to 17 years) with symptomatic SMD, and VA >=20/100 in both eyes at the time of the screening visit and anticipated to experience rapid deterioration in visual function and/or retinal structure, received SAR422459 at highest target dose level 1.8*10^6 TU/study eye. |
| Period Title: Overall Study | |||||||
| Started | 4 | 4 | 4 | 4 | 6 | 4 | 1 |
| Completed | 4 | 4 | 4 | 4 | 6 | 4 | 1 |
| Not Completed | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
Baseline Characteristics
| Arm/Group Title | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Cohort 5 | Cohort 6 | Cohort 7 | Total | |
|---|---|---|---|---|---|---|---|---|---|
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Arm/Group Description |
Participants (aged >=18 years) with advanced SMD, and VA <=20/200 in the worst eye and severe cone-rod dysfunction with no detectable or severely abnormal full-field ERG responses, received SAR422459 at lowest target dose level 1.8*10^5 TU/study eye. | Participants (aged >=18 years) with SMD, and VA <=20/200 in the worst eye with abnormal full-field ERG responses, received SAR422459 at lowest target dose level 1.8*10^5 TU/study eye. | Participants (aged >=18 years) with SMD, and VA <=20/200 in the worst eye with abnormal full-field ERG responses, received SAR422459 at escalated target dose level 6*10^5 TU/study eye. | Participants (aged >=18 years) with SMD, and VA <=20/200 in the worst eye with abnormal full-field ERG responses, received SAR422459 at target dose level 1.8*10^6 TU/study eye. | Participants (aged >=18 years) with SMD, and VA <=20/100 in the worst eye with abnormal full-field ERG responses, received SAR422459 at highest target dose level 1.8*10^6 TU/study eye. | Participants (aged 6 to 26 years) with symptomatic early or childhood-onset SMD, and VA >=20/200 in both eyes at the time of the screening visit and anticipated to experience rapid deterioration in visual function and/or retinal structure, received SAR422459 at highest target dose level 1.8*10^6 TU/study eye. | Pediatric participants (aged 6 to 17 years) with symptomatic SMD, and VA >=20/100 in both eyes at the time of the screening visit and anticipated to experience rapid deterioration in visual function and/or retinal structure, received SAR422459 at highest target dose level 1.8*10^6 TU/study eye. | Total of all reporting groups | |
| Overall Number of Baseline Participants | 4 | 4 | 4 | 4 | 6 | 4 | 1 | 27 | |
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Baseline Analysis Population Description |
[Not Specified]
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Age, Continuous
Median (Full Range) Unit of measure: Years |
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| Number Analyzed | 4 participants | 4 participants | 4 participants | 4 participants | 6 participants | 4 participants | 1 participants | 27 participants | |
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55.5
(32 to 66)
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40
(30 to 63)
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44.5
(35 to 60)
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40
(28 to 56)
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28
(24 to 41)
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23
(21 to 37)
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16
(16 to 16)
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36
(16 to 66)
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 4 participants | 4 participants | 4 participants | 4 participants | 6 participants | 4 participants | 1 participants | 27 participants | |
| Female | 1 | 3 | 4 | 0 | 2 | 2 | 1 | 13 | |
| Male | 3 | 1 | 0 | 4 | 4 | 2 | 0 | 14 | |
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Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 4 participants | 4 participants | 4 participants | 4 participants | 6 participants | 4 participants | 1 participants | 27 participants | |
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | |
| Asian | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 1 | |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | |
| Black or African American | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 1 | |
| White | 4 | 4 | 3 | 3 | 6 | 4 | 1 | 25 | |
| More than one race | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | |
Outcome Measures
Adverse Events
Limitations and Caveats
The planned analysis was adjusted and carried out on the available safety data collected before the Sponsor's decision to stop SAR422459 development prematurely.
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
Results Point of Contact
| Name/Title: | Trial Transparency Team |
| Organization: | Sanofi |
| Phone: | 800-633-1610 ext 1# |
| EMail: | Contact-US@sanofi.com |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Sanofi |
| ClinicalTrials.gov Identifier: | NCT01367444 |
| Other Study ID Numbers: |
TDU13583 SG1/001/10 |
| First Submitted: | June 3, 2011 |
| First Posted: | June 7, 2011 |
| Results First Submitted: | May 19, 2020 |
| Results First Posted: | June 5, 2020 |
| Last Update Posted: | April 14, 2022 |