NCT04462692

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.

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

Typical duration 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withdrawn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 2, 2020

Completed
6 days until next milestone

First Posted

Study publicly available on registry

July 8, 2020

Completed
9 months until next milestone

Study Start

First participant enrolled

March 31, 2021

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2023

Completed
Last Updated

November 1, 2021

Status Verified

October 1, 2021

Enrollment Period

2.5 years

First QC Date

July 2, 2020

Last Update Submit

October 25, 2021

Conditions

Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)

Keywords

CLN2, Batten Disease

Outcome Measures

Primary Outcomes (1)

  • Change in retinal structure in children with CLN2 Batten disease

    As assessed by SD-OCT measures over time.

    96 weeks

Secondary Outcomes (2)

  • Change in visual function

    96 weeks

  • Change in visual function

    96 weeks

Eligibility Criteria

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

Children with CLN2 Batten disease undergoing current standard of care for their condition.

You may qualify if:

  • A participant is eligible to be included in the study only if all of the following criteria apply:
  • His or her legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
  • 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
  • Is currently receiving biweekly ERT treatment with cerliponase alfa

You may not qualify if:

  • A participant is excluded from the study if any of the following criteria apply:
  • Has had prior treatment with an adeno-associated virus-based AAV gene therapy
  • Is currently participating in a clinical trial of investigational product for the treatment of CLN2 disease

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
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 2, 2020

First Posted

July 8, 2020

Study Start

March 31, 2021

Primary Completion

October 1, 2023

Study Completion

October 1, 2023

Last Updated

November 1, 2021

Record last verified: 2021-10

Data Sharing

IPD Sharing
Will not share