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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.

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.

Condition or disease
Neuronal Ceroid Lipofuscinosis Type 2 (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 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.

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Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Prospective, Observational Study to Evaluate Ocular Disease Progression in Children With CLN2 Batten Disease
Estimated Study Start Date : March 31, 2021
Estimated Primary Completion Date : October 2023
Estimated Study Completion Date : October 2023





Primary Outcome Measures :
  1. Change in retinal structure in children with CLN2 Batten disease [ Time Frame: 96 weeks ]
    As assessed by SD-OCT measures over time.


Secondary Outcome Measures :
  1. Change in visual function [ Time Frame: 96 weeks ]
    As assessed by visual acuity over time.

  2. Change in visual function [ Time Frame: 96 weeks ]
    As assessed by pupillary light reflex over time.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Children with CLN2 Batten disease undergoing current standard of care for their condition.
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04462692


Contacts
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Contact: REGENXBIO Patient Advocacy 866-860-0117 patientadvocacy@regenxbio.com

Sponsors and Collaborators
Regenxbio Inc.
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Responsible Party: Regenxbio Inc.
ClinicalTrials.gov Identifier: NCT04462692    
Other Study ID Numbers: RGX-381-9101
First Posted: July 8, 2020    Key Record Dates
Last Update Posted: February 5, 2021
Last Verified: February 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Regenxbio Inc.:
CLN2, Batten Disease
Additional relevant MeSH terms:
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Neuronal Ceroid-Lipofuscinoses
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Lipid Metabolism Disorders
Metabolic Diseases