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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01505062
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 : January 6, 2012
Results First Posted : March 18, 2020
Last Update Posted : April 28, 2022
Sponsor:
Information provided by (Responsible Party):
Sanofi

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Usher Syndrome
Retinitis Pigmentosa
Intervention Drug: SAR421869
Enrollment 9
Recruitment Details In 2017, Sanofi suspended trials of SAR421869, while assessing its future. Until final decision was made, trial TDU13600 was not complete and in fact, further recruitment was planned. In 2019, Sanofi decided to terminate trial due to final decision on SAR421869, and shared decision with health authorities. Results have been reported expeditiously.
Pre-assignment Details A total of 11 participants were screened. Nine participants of them were enrolled in the study with 3 participants in each of Cohorts 1 to 3. Due to early termination no participant was recruited in Cohorts 4 and 5.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description Participants received subretinally a single injection of SAR421869 at target dose of 1.4*10^5 transducing units (TU) per eye. Participants received subretinally a single injection of SAR421869 at target dose of 4.7*10^5 TU per eye. Participants received subretinally a single injection of SAR421869 at target dose of 1.4*10^6 TU per eye.
Period Title: Overall Study
Started 3 3 3
Completed 3 3 3
Not Completed 0 0 0
Arm/Group Title Cohort 1 Cohort 2 Cohort 3 Total
Hide Arm/Group Description Participants received subretinally a single injection of SAR421869 at target dose of 1.4*10^5 TU per eye. Participants received subretinally a single injection of SAR421869 at target dose of 4.7*10^5 TU per eye. Participants received subretinally a single injection of SAR421869 at target dose of 1.4*10^6 TU per eye. Total of all reporting groups
Overall Number of Baseline Participants 3 3 3 9
Hide Baseline Analysis Population Description
Analysis was performed on safety population that included all participants who were enrolled in the study and received investigational medicinal product (IMP).
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 3 participants 3 participants 3 participants 9 participants
25
(22 to 28)
42
(32 to 57)
50
(32 to 56)
32
(22 to 57)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 9 participants
Female
2
  66.7%
2
  66.7%
2
  66.7%
6
  66.7%
Male
1
  33.3%
1
  33.3%
1
  33.3%
3
  33.3%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 9 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
3
 100.0%
3
 100.0%
3
 100.0%
9
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
Hide Description An adverse event (AE) was any unfavorable and unintended physical sign, symptom, or laboratory parameter that developed or worsened in severity during the course of the study, whether or not considered related to the IMP. The TEAEs were defined as any event that started or increased in severity after the participant received IMP, including abnormal laboratory results, electrocardiogram, etc.
Time Frame From Baseline to Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description:
Participants received subretinally a single injection of SAR421869 at target dose of 1.4*10^5 TU per eye.
Participants received subretinally a single injection of SAR421869 at target dose of 4.7*10^5 TU per eye.
Participants received subretinally a single injection of SAR421869 at target dose of 1.4*10^6 TU per eye.
Overall Number of Participants Analyzed 3 3 3
Measure Type: Number
Unit of Measure: percentage of participants
100 100 100
2.Primary Outcome
Title Percentage of Participants With TEAEs by Severity
Hide Description An AE was any unfavorable and unintended physical sign, symptom, or laboratory parameter that developed or worsened in severity during the course of the study, whether or not considered related to the IMP. The TEAEs were defined as any event that started or increased in severity after the participant received IMP, including abnormal laboratory results, electrocardiogram, etc. For each AE, the severity was categorized as either mild, moderate or severe where 'mild' was defined as discomfort noticed but did not interfere with the participant's daily routines (an annoyance), 'moderate' was defined as some impairment of function, not hazardous to health (uncomfortable or embarrassing), and 'severe' was defined as significant impairment of function, hazardous to health (incapacitating).
Time Frame From Baseline to Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population.
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description:
Participants received subretinally a single injection of SAR421869 at target dose of 1.4*10^5 TU per eye.
Participants received subretinally a single injection of SAR421869 at target dose of 4.7*10^5 TU per eye.
Participants received subretinally a single injection of SAR421869 at target dose of 1.4*10^6 TU per eye.
Overall Number of Participants Analyzed 3 3 3
Measure Type: Number
Unit of Measure: percentage of participants
Mild 100 100 100
Moderate 0 33 67
Severe 0 0 67
Time Frame All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Adverse Event Reporting Description Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
 
Arm/Group Title Cohort 1 Cohort 2 Cohort 3
Hide Arm/Group Description Participants received subretinally a single injection of SAR421869 at target dose of 1.4*10^5 TU per eye. Participants received subretinally a single injection of SAR421869 at target dose of 4.7*10^5 TU per eye. Participants received subretinally a single injection of SAR421869 at target dose of 1.4*10^6 TU per eye.
All-Cause Mortality
Cohort 1 Cohort 2 Cohort 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)   0/3 (0.00%)   0/3 (0.00%) 
Hide Serious Adverse Events
Cohort 1 Cohort 2 Cohort 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)   0/3 (0.00%)   2/3 (66.67%) 
Eye disorders       
Uveitis † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Visual acuity reduced † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
1
Term from vocabulary, MedDRA 22.0
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Cohort 1 Cohort 2 Cohort 3
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   3/3 (100.00%)   3/3 (100.00%)   3/3 (100.00%) 
Eye disorders       
Anterior chamber cell † 1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Cataract † 1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Conjunctival haemorrhage † 1  3/3 (100.00%)  2/3 (66.67%)  0/3 (0.00%) 
Dyschromatopsia † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Eye pain † 1  2/3 (66.67%)  1/3 (33.33%)  1/3 (33.33%) 
Eye pruritus † 1  2/3 (66.67%)  1/3 (33.33%)  0/3 (0.00%) 
Macular fibrosis † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Photophobia † 1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Photopsia † 1  0/3 (0.00%)  1/3 (33.33%)  1/3 (33.33%) 
Retinal detachment † 1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Retinal haemorrhage † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Subretinal fluid † 1  2/3 (66.67%)  0/3 (0.00%)  0/3 (0.00%) 
Vision blurred † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Visual acuity reduced † 1  1/3 (33.33%)  0/3 (0.00%)  1/3 (33.33%) 
Vitreous floaters † 1  0/3 (0.00%)  1/3 (33.33%)  1/3 (33.33%) 
Gastrointestinal disorders       
Constipation † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Dental caries † 1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Dry mouth † 1  1/3 (33.33%)  0/3 (0.00%)  1/3 (33.33%) 
Gastrooesophageal reflux disease † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Nausea † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Vomiting † 1  1/3 (33.33%)  1/3 (33.33%)  0/3 (0.00%) 
General disorders       
Chest pain † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Fatigue † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Injection site extravasation † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Investigations       
Blood creatine phosphokinase increased † 1  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%) 
Intraocular pressure decreased † 1  1/3 (33.33%)  0/3 (0.00%)  1/3 (33.33%) 
Intraocular pressure increased † 1  0/3 (0.00%)  2/3 (66.67%)  2/3 (66.67%) 
Urine analysis abnormal † 1  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%) 
White blood cell count increased † 1  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%) 
Metabolism and nutrition disorders       
Decreased appetite † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Increased appetite † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Nervous system disorders       
Headache † 1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Migraine † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Occipital neuralgia † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Paraesthesia † 1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Syncope † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Psychiatric disorders       
Nervousness † 1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Renal and urinary disorders       
Glycosuria † 1  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Pollakiuria † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Urge incontinence † 1  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
1
Term from vocabulary, MedDRA 22.0
†
Indicates events were collected by systematic assessment
The planned analysis was adjusted and carried out only on the available safety and tolerability data collected before the Sponsor's decision to stop SAR421869 development prematurely.
Certain Agreements
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
Layout table for Results Point of Contact information
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):
Layout table for additonal information
Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT01505062    
Other Study ID Numbers: TDU13600
US1/001/10 ( Other Identifier: Oxford Biomedica )
First Submitted: January 4, 2012
First Posted: January 6, 2012
Results First Submitted: February 28, 2020
Results First Posted: March 18, 2020
Last Update Posted: April 28, 2022