NCT04480476

Brief Summary

This is a multi center, retrospective, chart review study to document the evolution of ocular disease progression in pediatric patients with CLN2.

Trial Health

15
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Timeline
Completed

Started Mar 2021

Shorter than P25 for all trials

Status
withdrawn

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 10, 2020

Completed
11 days until next milestone

First Posted

Study publicly available on registry

July 21, 2020

Completed
8 months until next milestone

Study Start

First participant enrolled

March 31, 2021

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2022

Completed
Last Updated

November 1, 2021

Status Verified

October 1, 2021

Enrollment Period

1 year

First QC Date

July 10, 2020

Last Update Submit

October 25, 2021

Conditions

Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)

Keywords

CLN2, Batten Disease

Outcome Measures

Primary Outcomes (1)

  • 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 (1)

  • Characterize changes in visual function.

    From first available medical chart through informed consent, an average of 10 years

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients with CLN2 Batten Disease

You may qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Neuronal Ceroid-Lipofuscinoses

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases
0

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
RETROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 10, 2020

First Posted

July 21, 2020

Study Start

March 31, 2021

Primary Completion

April 1, 2022

Study Completion

April 1, 2022

Last Updated

November 1, 2021

Record last verified: 2021-10

Data Sharing

IPD Sharing
Will not share