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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

  • Indication

    CLN2 disease

  • Investigational Therapeutic

    Brineura® (cerliponase alfa or 190)

  • Study Type

    Phase 2

  • 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.

  • Status

    Active, not recruiting

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Study 190-501

Recruiting

Cerliponase alfa Observational Study (US)

  • Indication

    CLN2 Disease

  • Investigational Therapeutic

    N/A

  • Study Type

    Observational

  • Goal

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

  • Status

    This study is currently recruiting participants

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Study 190-504

Recruiting

Cerliponase alfa Observational Study (EMA member states)

  • Indication

    CLN2 Disease

  • Investigational Therapeutic

    N/A

  • Study Type

    Observational

  • Goal

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

  • Status

    This study is currently recruiting participants

 

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

  • Investigational Therapeutic

    N/A

  • Study Type

    Observational

  • Goal

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

  • 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

  • Indication

    CLN2 Disease

  • Investigational Therapeutic

    Brineura® (cerliponase alfa or 190)

  • Study Type

    Expanded Access

  • 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

  • Status

    This study has been completed

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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

  • Indication

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

  • Investigational Therapeutic

    Brineura® (cerliponase alfa or 190)

  • Study Type

    Phase 1/2

  • Goal

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

  • Status

    This study has been completed

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