Back to Results

Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

Interested in this Trial?

Contact Us

Trial Status Unknown

Trial Identifier

NCT03862274

Condition

CLN2, Batten Disease, Neuronal Ceroid-Lipofuscinoses

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

The investigators propose a study to assess cognitive and developmental outcomes of patients with CLN2 that are untreated and receiving cerliponase alfa. This study aims to validate standardized assessment measures to establish a standard of care. The secondary aim is to compare cognitive and developmental outcomes of patients with CLN2 that are receiving celiponase alfa to a natural history cohort. To accomplish specific aims of the study, the investigators will use a multi-method approach to collect retrospective data collected as standard of care and prospective developmental data in children with CLN2 disease. The investigators will use a combination of standardized measures that include direct assessment and parent report of child development. The investigators focus will also include multiple measures of development including language, motor, social-emotional, and adaptive functioning.

Eligibility Criteria

Birth Sex

All

Age

Any

Healthy Volunteers

No

Drug/Treatment:

CLN2 Treatment

Study Type:

Observational

Number of Participants:

30

Study Started:

2018-12-01

Study Updated:

2023-02-13

Trial Locations

  • Nationwide Children's Hospital

    Columbus, Ohio, United States

Inclusion Criteria

  • Patients that have a TPP1 enzyme deficiency
  • Patients have confirmed molecular diagnosis of pathogenic variants in the TPP1 gene
  • Patients that are enrolled in post-marketing studies will be allowed to enroll into the current study

Exclusion Criteria

  • Patients without a diagnosis of CLN2 and deficiency of TPP1
  • Patients that are currently enrolled as part of a larger multi-center clinical tria

Contact BioMarin Clinical Trials

Please complete and submit the following form to express interest in participating and request more information about one of our active clinical trials

*required fields

"*" indicates required fields

Please select the trial that you are interested in from the dropdown below. Note that this list only includes currently active trials. If you cannot find the trial you are seeking, please enter the study identifier and title in the comments box below, along with any other details you would like us to know to help address your inquiry.
Consent*
Consent*
For more Information about BioMarin's privacy obligations or to exercise your privacy rights, please visit BioMarin's Data Privacy Center.
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form

What’s Next?

Visit the following links to learn more about clinical trials.

Discuss this trial with your care team.

You may wish to print this page to share the details with your healthcare providers and discuss whether you may be eligible.

Print

Get in touch.

Contact Us