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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
Sponsor:
Information provided by (Responsible Party):
Regenxbio Inc.

Tracking Information
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
 (submitted: July 17, 2020)
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
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: July 17, 2020)
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.
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
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.
 
Recruitment Information
Recruitment Status Not yet recruiting
Estimated Enrollment
 (submitted: July 17, 2020)
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:

  1. The participant's legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
  2. The participant has a documented diagnosis of CLN2 disease due to TPP1 deficiency, or has a relative clinically diagnosed with CLN2 disease who has the same CLN2 mutations as the participant
  3. The participant has had one or more eye examinations by an eye care specialist at any time since birth.

Exclusion Criteria:

No exclusion criteria apply to this study.

Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: REGENXBIO Patient Advocacy 866-860-0117 patientadvocacy@regenxbio.com
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
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Regenxbio Inc.
Study Sponsor Regenxbio Inc.
Collaborators Not Provided
Investigators Not Provided
PRS Account Regenxbio Inc.
Verification Date February 2021