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CLN2 Disease clinical trials

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NCT ID: NCT04613089 Recruiting - Batten Disease Clinical Trials

Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database

Start date: April 8, 2020
Phase:
Study type: Observational [Patient Registry]

This is an observational study that aims at assessing the natural history of NCL diseases as part of the international DEM-CHILD Database. 1. Patient data are collected from medical records, patient questionnaires and routine follow up clinical examinations with focus on assessing progression in key areas of disease such as motor, language, cognition, seizures, vision, and behavior. 2. A local biorepository of samples from genetically defined NCL patients will be established as well as a virtual biorepository within the DEM-CHILD DB to be able to easily localize international availability of patient samples.

NCT ID: NCT02963350 Approved for marketing - CLN2 Disease Clinical Trials

A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease

Start date: n/a
Phase: N/A
Study type: Expanded Access

- To provide access to BMN 190 to patients with CLN2 disease who cannot participate in a clinical trial. - To collect additional information on the safety and tolerability of BMN 190 administration in patients with CLN2 disease.

NCT ID: NCT02678689 Completed - Batten Disease Clinical Trials

A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

Start date: February 2016
Phase: Phase 2
Study type: Interventional

This Phase 2 open-label, multicenter study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration every other week (qow) for a period of 144 weeks, in patients with CLN2. The study is designed to assess disease progression in CLN2 patients treated with BMN 190 compared to natural history data from untreated historical controls.

NCT ID: NCT02485899 Completed - Batten Disease Clinical Trials

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

Start date: February 2015
Phase: Phase 1/Phase 2
Study type: Interventional

The Phase 1/2 study (190-201) evaluated the efficacy and safety of a 300 mg dose of BMN 190 administered every other week (qow) to patients with CLN2. The dose and regimen for this study (190-202) are based on the results of the 190-201 study. The rationale for this phase 2 extension study is to provide patients who complete the 190-201 study with the option to continue BMN 190 treatment. The 190-202 study is an open label extension protocol to assess long-term safety and efficacy.

NCT ID: NCT01907087 Completed - Batten Disease Clinical Trials

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

Start date: September 2013
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to determine whether BMN 190 is safe and effective in the treatment of patients with Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease.