A Retrospective, Natural History Study in Children With CLN2 | NCT04480476

Studies
A Retrospective, Natural History Study in Children With CLN2

NCT04480476

Sponsor

REGENXBIO Inc.

Status

Withdrawn

Last Update Posted

Nov 1, 2021Actual

Enrollment

0Actual

Phase

Not provided

Conditions

Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)

Interventions

Not provided

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Oct 2021

Overall Recruitment Status or Expanded Access Status

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

study stopped due to company decision

Expanded Access Info

Start Date

Mar 31, 2021Estimated

Primary Completion Date

Apr 2022Estimated

Completion Date

Apr 2022Estimated

First Submitted Date

Jul 10, 2020

First Submission Date that Met QC Criteria

Jul 17, 2020

First Posted Date

Jul 21, 2020Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Oct 25, 2021

Last Update Posted Date

Nov 1, 2021Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

0Actual

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Characterize retinal structural changes in children with CLN2	As assessed in by SD-OCT measures in ophthalmic records of children with CLN2	From first available medical chart through informed consent, an average of 10 years

Secondary Outcomes

Measure	Description	Time Frame
Characterize changes in visual function.	As measured by changes in visual acuity over time in ophthalmic records of children with CLN2.	From first available medical chart through informed consent, an average of 10 years

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

A participant is eligible to be included in the study only if all of the following criteria apply:

The participant's legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
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
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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Patients with CLN2 Batten Disease

Sampling Method

Non-Probability Sample

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D009472	Neuronal Ceroid-Lipofuscinoses

Ancestor Terms

ID	Term
D020271	Heredodegenerative Disorders, Nervous System
D019636	Neurodegenerative Diseases
D009422	Nervous System Diseases
D030342	Genetic Diseases, Inborn

Browse Leaves

Not provided

Browse Branches

Not provided