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Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

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ClinicalTrials.gov Identifier: NCT00999609
Recruitment Status : Active, not recruiting
First Posted : October 22, 2009
Results First Posted : March 26, 2018
Last Update Posted : March 26, 2018
Sponsor:
Collaborators:
Children's Hospital of Philadelphia
University of Iowa
Information provided by (Responsible Party):
Spark Therapeutics

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Inherited Retinal Dystrophy Due to RPE65 Mutations
Leber Congenital Amaurosis
Intervention: Biological: AAV2-hRPE65v2,voretigene neparvovec-rzyl

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Intervention Bilateral subretinal injection of voretigene neparvovec-rzyl (AAV2-hRPE65v2), 1.5x 10e11 vg in a total volume of 0.3ml per eye, administered no fewer than 6 days apart
Control No intervention, no sham; uninjected control group

Participant Flow:   Overall Study
    Intervention   Control
STARTED   21 [1]   10 
COMPLETED   20 [2]   9 [2] 
NOT COMPLETED   1   1 
[1] Before intervention
[2] 1 year post intervention



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Intervention Bilateral subretinal injection of voretigene neparvovec-rzyl (AAV2-hRPE65v2), 1.5x 10e11 vg in a total volume of 0.3ml per eye, administered no fewer than 6 days apart
Control Control group did not receive voretigene neparvovec-rzyl. The control group became eligible to receive voretigene neparvovec-rzyl 1 year after their baseline evaluations, provided they still met all eligibility criteria.
Total Total of all reporting groups

Baseline Measures
   Intervention   Control   Total 
Overall Participants Analyzed 
[Units: Participants]
 21   10   31 
Age 
[Units: Years]
Mean (Standard Deviation)
 14.7  (11.8)   15.9  (9.5)   15.1  (10.9) 
Age, Customized 
[Units: Participants]
Count of Participants
     
Age Group       
<10 years old      9  42.9%      4  40.0%      13  41.9% 
≥10 years old      12  57.1%      6  60.0%      18  58.1% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      12  57.1%      6  60.0%      18  58.1% 
Male      9  42.9%      4  40.0%      13  41.9% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Hispanic or Latino      5  23.8%      1  10.0%      6  19.4% 
Not Hispanic or Latino      16  76.2%      9  90.0%      25  80.6% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
American Indian or Alaska Native      2   9.5%      1  10.0%      3   9.7% 
Asian      3  14.3%      2  20.0%      5  16.1% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      2   9.5%      0   0.0%      2   6.5% 
White      14  66.7%      7  70.0%      21  67.7% 
More than one race      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
Multi-luminance Mobility Test (MLMT), bilateral, passing at a lux level of < 125 or ≥ 125 lux [1] 
[Units: Participants]
Count of Participants
     
Lowest passing light level of < 125 lux      12  57.1%      4  40.0%      16  51.6% 
Lowest passing light level of ≥ 125 lux      9  42.9%      6  60.0%      15  48.4% 
[1] The multi-luminance mobility test (MLMT) measures changes in functional vision, based on the ability to navigate a course accurately and at a reasonable pace at different levels of environmental illumination. The MLMT was assessed at 1 or more of 7 light levels ranging from 400 lux (a brightly lit office) to 1 lux (a moonless summer night). At baseline, stratification was based on the lowest passing light level (less than or equal to 125 lux or greater than 125 lux).


  Outcome Measures

1.  Primary:   Multi-luminance Mobility Testing (MLMT), Bilateral   [ Time Frame: One year (change from baseline) ]

2.  Secondary:   Full-field Light Sensitivity Threshold (FST) Testing: White Light   [ Time Frame: One year (change from baseline) ]

3.  Secondary:   Multi-luminance Mobility Testing (Monocular)   [ Time Frame: One year (change from baseline) ]

4.  Secondary:   Visual Acuity   [ Time Frame: One year (change from baseline) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information

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.


Results Point of Contact:  
Name/Title: Clinical Director
Organization: Spark Therapeutics
e-mail: clinicaltrials@sparktx.com


Publications of Results:

Other Publications:

Responsible Party: Spark Therapeutics
ClinicalTrials.gov Identifier: NCT00999609     History of Changes
Other Study ID Numbers: AAV2-hRPE65v2-301
First Submitted: October 21, 2009
First Posted: October 22, 2009
Results First Submitted: January 12, 2018
Results First Posted: March 26, 2018
Last Update Posted: March 26, 2018