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An Observational Study in Children With CLN2 Batten Disease

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ClinicalTrials.gov Identifier: NCT04462692
Recruitment Status : Not yet recruiting
First Posted : July 8, 2020
Last Update Posted : February 5, 2021
Sponsor:
Information provided by (Responsible Party):
Regenxbio Inc.

Tracking Information
First Submitted Date July 2, 2020
First Posted Date July 8, 2020
Last Update Posted Date February 5, 2021
Estimated Study Start Date March 31, 2021
Estimated Primary Completion Date October 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: July 2, 2020)
Change in retinal structure in children with CLN2 Batten disease [ Time Frame: 96 weeks ]
As assessed by SD-OCT measures over time.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: July 2, 2020)
  • Change in visual function [ Time Frame: 96 weeks ]
    As assessed by visual acuity over time.
  • Change in visual function [ Time Frame: 96 weeks ]
    As assessed by pupillary light reflex over time.
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 An Observational Study in Children With CLN2 Batten Disease
Official Title A Prospective, Observational Study to Evaluate Ocular Disease Progression in Children With CLN2 Batten Disease
Brief Summary This is a prospective, longitudinal natural history study to document the progression of ocular manifestations of CLN2 disease among a community-dwelling population of pediatric participants affected by this disease.
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 prospective data collection, ocular disease progression in children with a clinical presentation consistent with CLN2 Batten disease undergoing current standard of care for their condition. No investigational product is administered in this observational study.
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Children with CLN2 Batten disease undergoing current standard of care for their condition.
Condition 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 2, 2020)
50
Original Estimated Enrollment Same as current
Estimated Study Completion Date October 2023
Estimated Primary Completion Date October 2023   (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. His or her legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
  2. Has documented diagnosis of CLN2 disease due to TPP1 deficiency, or has a relative clinically diagnosed with CLN2 with the same mutation as the participant
  3. Is currently receiving biweekly ERT treatment with cerliponase alfa

Exclusion Criteria:

A participant is excluded from the study if any of the following criteria apply:

  1. Has had prior treatment with an adeno-associated virus-based AAV gene therapy
  2. Is currently participating in a clinical trial of investigational product for the treatment of CLN2 disease
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 NCT04462692
Other Study ID Numbers RGX-381-9101
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