Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

Plain-language summary

This study examines the developmental outcomes of children diagnosed with CLN2 disease (a form of Batten disease). It includes patients with a TPP1 enzyme deficiency and a confirmed molecular diagnosis of pathogenic variants in the TPP1 gene. It is a non-phase study that enrolls by invitation.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Enrolling by invitation

Sponsor

Jessica Scherr

Intervention

CLN2 Treatment

Start date

12/1/2018

Estimated completion

12/1/2025

Countries

United States

Related genes

Locations

• Columbus, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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 trial