A Retrospective, Natural History Study in Children With CLN2
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ClinicalTrials.gov Identifier: NCT04480476 |
Recruitment Status :
Not yet recruiting
First Posted : July 21, 2020
Last Update Posted : February 3, 2021
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Sponsor:
Regenxbio Inc.
Information provided by (Responsible Party):
Regenxbio Inc.
Tracking Information | |||||
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First Submitted Date | July 10, 2020 | ||||
First Posted Date | July 21, 2020 | ||||
Last Update Posted Date | February 3, 2021 | ||||
Estimated Study Start Date | March 31, 2021 | ||||
Estimated Primary Completion Date | April 2022 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures |
Characterize retinal structural changes in children with CLN2 [ Time Frame: From first available medical chart through informed consent, an average of 10 years ] As assessed in by SD-OCT measures in ophthalmic records of children with CLN2
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Original Primary Outcome Measures | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures |
Characterize changes in visual function. [ Time Frame: From first available medical chart through informed consent, an average of 10 years ] As measured by changes in visual acuity over time in ophthalmic records of children with CLN2.
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Original Secondary Outcome Measures | Same as current | ||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title | A Retrospective, Natural History Study in Children With CLN2 | ||||
Official Title | A Retrospective, Chart Review Study to Evaluate Ocular Disease Progression in Children With Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) | ||||
Brief Summary | This is a multi center, retrospective, chart review study to document the evolution of ocular disease progression in pediatric patients with CLN2. | ||||
Detailed Description | CLN2 is a rare disease with limited available ocular natural history data. While current standard of care slows motor degeneration, it is not known to treat the ocular manifestations of disease. This study is planned to document, through retrospective data collection, ocular disease progression in children with a clinical presentation consistent with CLN2. No investigational product is administered in this retrospective, chart review study. | ||||
Study Type | Observational | ||||
Study Design | Observational Model: Case-Only Time Perspective: Retrospective |
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Target Follow-Up Duration | Not Provided | ||||
Biospecimen | Not Provided | ||||
Sampling Method | Non-Probability Sample | ||||
Study Population | Patients with CLN2 Batten Disease | ||||
Condition | Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) | ||||
Intervention | Not Provided | ||||
Study Groups/Cohorts | Not Provided | ||||
Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status | Not yet recruiting | ||||
Estimated Enrollment |
50 | ||||
Original Estimated Enrollment | Same as current | ||||
Estimated Study Completion Date | April 2022 | ||||
Estimated Primary Completion Date | April 2022 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria | Inclusion Criteria: A participant is eligible to be included in the study only if all of the following criteria apply:
Exclusion Criteria: No exclusion criteria apply to this study. |
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Sex/Gender |
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Ages | Child, Adult, Older Adult | ||||
Accepts Healthy Volunteers | No | ||||
Contacts |
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Listed Location Countries | Not Provided | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number | NCT04480476 | ||||
Other Study ID Numbers | RGX-381-9102 | ||||
Has Data Monitoring Committee | No | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement |
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Responsible Party | Regenxbio Inc. | ||||
Study Sponsor | Regenxbio Inc. | ||||
Collaborators | Not Provided | ||||
Investigators | Not Provided | ||||
PRS Account | Regenxbio Inc. | ||||
Verification Date | February 2021 |