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.
A Phase 2 Open-Label Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Patients With CLN2 Disease
CLN2 disease
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.
Cerliponase alfa or 190
Active, not recruiting
Phase 2
Cerliponase alfa Observational Study (US)
CLN2 Disease
Evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease)
N/A
This study is currently recruiting participants
Observational
Cerliponase alfa Observational Study (EMA member states)
CLN2 Disease
Evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease)
N/A
This study is currently recruiting participants
Observational
Cerliponase alfa observational survey to evaluate long-term safety of cerliponase alfa in patients in Japan with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease)
CLN2 Disease
Evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease)
N/A
This study is currently recruiting participants
Observational
A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease
CLN2 Disease
This open-label expanded access trial will collect additional information on the safety and tolerability of BMN 190 administration in patients with CLN2 disease
Cerliponase alfa or 190
This study has been completed
Expanded Access
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
CLN2 Disease (Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2)
Determine whether BMN 190 is safe and effective in the treatment of patients with CLN2 disease
Cerliponase alfa or 190
This study has been completed
Phase 1/2
A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
CLN2 Disease
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.
BMN 190 Intracerebroventricular (ICV) access device
Completed
Phase 1/2