Current Studies
Study 190-203
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
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Investigational Therapeutic
Brineura® (cerliponase alfa or 190)
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Study Type
Phase 2
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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.
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Status
Active, not recruiting
Study 190-501
RecruitingCerliponase alfa Observational Study (US)
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Indication
CLN2 Disease
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Investigational Therapeutic
N/A
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Study Type
Observational
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Goal
Evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease)
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Status
This study is currently recruiting participants
Study 190-504
RecruitingCerliponase alfa Observational Study (EMA member states)
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Indication
CLN2 Disease
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Investigational Therapeutic
N/A
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Study Type
Observational
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Goal
Evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease)
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Status
This study is currently recruiting participants
Study 190-506
RecruitingCerliponase alfa observational survey to evaluate long-term safety of cerliponase alfa in patients in Japan with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease)
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Indication
CLN2 Disease
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Investigational Therapeutic
N/A
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Study Type
Observational
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Goal
Evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease)
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Status
This study is currently recruiting participants
Completed Studies
Study 190-502
A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease
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Indication
CLN2 Disease
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Investigational Therapeutic
Brineura® (cerliponase alfa or 190)
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Study Type
Expanded Access
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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
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Status
This study has been completed
Study 190-201
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)
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Investigational Therapeutic
Brineura® (cerliponase alfa or 190)
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Study Type
Phase 1/2
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Goal
Determine whether BMN 190 is safe and effective in the treatment of patients with CLN2 disease
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Status
This study has been completed
Study 190-202
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
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Investigational Therapeutic
BMN 190 Intracerebroventricular (ICV) access device
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Study Type
Phase 1/2
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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.
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Status
Completed