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Cerliponase alfa

Current Clinical Trials: CLN2 Disease

 

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

Study Type: Phase 2

Goal: The purpose of this study is to 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: Enrolling participants by invitation only

Additional information: View on Clinicaltrials.gov

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

Study Type: Expanded Access

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: BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1)

Status: Enrolling by Invitation

Additional information: View on Clinicaltrials.gov

Completed Clinical Studies: CLN2 Disease

Study: 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: BMN 190

Study Type: Phase 1/2

Goal: 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.

Status: This study has been completed

Additional information: View on Clinicaltrials.gov

Cerliponase Alfa for CLN2 Disease

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BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS

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Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

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