CLN2 Clinical Trials

Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease is an ultra-rare and rapidly progressing pediatric brain disorder and one of the most common forms of neuronal ceroid lipofuscinosis, a group of inherited disorders collectively known as Batten disease.

Learn more about our CLN2 disease clinical trials below.

Cerliponase alfa

Study 190-203 Active, Not Recruiting

A Phase 2 Open-Label Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Patients With CLN2 Disease

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Indication

CLN2 disease

Goal

Evaluate the safety, tolerability, and efficacy of BMN 190 in patients with CLN2. The study is designed to assess disease progression in siblings of children enrolled in the 190-201 study.

Investigational therapeutic

Cerliponase alfa or 190

Status

Active, not recruiting

Study Type

Phase 2

Study 190-501 Recruiting

Cerliponase alfa Observational Study (US)

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Indication

CLN2 Disease

Goal

Evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease)

Investigational therapeutic

N/A

Status

This study is currently recruiting participants

Study Type

Observational

Study 190-504 Recruiting

Cerliponase alfa Observational Study (EMA member states)

Indication

CLN2 Disease

Goal

Evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease)

Investigational therapeutic

N/A

Status

This study is currently recruiting participants

Study Type

Observational

Study 190-506 Recruiting

Cerliponase alfa observational survey to evaluate long-term safety of cerliponase alfa in patients in Japan with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease)

Indication

CLN2 Disease

Goal

Evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease)

Investigational therapeutic

N/A

Status

This study is currently recruiting participants

Study Type

Observational

Study 190-502 Complete

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

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Indication

CLN2 Disease

Goal

This open-label expanded access trial will collect additional information on the safety and tolerability of BMN 190 administration in patients with CLN2 disease

Investigational therapeutic

Cerliponase alfa or 190

Status

This study has been completed

Study Type

Expanded Access

Study 190-201 Complete

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

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Indication

CLN2 Disease (Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2)

Goal

Determine whether BMN 190 is safe and effective in the treatment of patients with CLN2 disease

Investigational therapeutic

Cerliponase alfa or 190

Status

This study has been completed

Study Type

Phase 1/2

Study 190-202 Recruiting

A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

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Indication

CLN2 Disease

Goal

The purpose of this 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.

Investigational therapeutic

BMN 190 Intracerebroventricular (ICV) access device

Status

Completed

Study Type

Phase 1/2