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A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease | Clinical Trials | Clareo Health

COMPLETEDPHASE2

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

A Phase 2, Open-Label, Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

NCT02678689•BioMarin Pharmaceutical

View on ClinicalTrials.gov

Summary

This Phase 2 open-label, multicenter study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration every other week (qow) for a period of 144 weeks, in patients with CLN2. The study is designed to assess disease progression in CLN2 patients treated with BMN 190 compared to natural history data from untreated historical controls.

Detailed Description

BMN 190 is a recombinant form of human tripeptidyl peptidase 1 (TPP1), the enzyme deficient in patients with CLN2 diseases (also known as classical late-infantile CLN2, cLINCL, or Jansky-Bielschowsky disease), a form of Batten Disease. As an enzyme replacement therapy (ERT), BMN 190 is designed to help restore TPP1 enzyme activity. BMN 190 is designed to reduce the progressive, pathologic accumulation of lysosomal storage material. 190-203 is a Phase 2 open-label, multicenter study that will evaluate the safety, tolerability, and efficacy of BMN 190 in pediatric patients \< 18 years of age with CLN2 disease. Study drug dosing will be determined by the patient's age and administered via intracerebroventricular (ICV) infusion every other week (qow), for a duration of 144 weeks.

Eligibility

Age

0 - 17 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Enrollment is complete.
•Diagnosis of CLN2 disease as determined by TPP1 enzyme activity (dried blood spot) in the fibroblasts and leukocytes available at Screening
•Quantitative clinical assessment of the Hamburg motor-language aggregate score 3-6 at Screening on CLN2 disease motor-language scale, as defined in the Ratings Assessment Guideline
•\< 18 years of age at the time of informed consent
•Written informed consent from parent or legal guardian and assent form subject, if appropriate
•Males and females who are of reproductive age should practice true abstinence, defined as no sexual activity, during the study and for 6 months after the study has been completed (or withdrawal from the study). If sexually active and not practicing true abstinence, males and females of reproductive age must use a highly effective method of contraception while participating in the study.
•Ability to comply with protocol required assessments (ICV implantation, drug administration, laboratory sample collection, electroencephalogram (EEG), electrocardiogram (ECG),magnetic resonance imaging (MRI), etc.)

Exclusion Criteria

•Presence of another inherited neurological disease, e.g., other forms of CLN or seizures unrelated to CLN2 disease (patients with febrile seizures may be eligible)
•Presence of another neurological illness that may have caused cognitive decline (e.g., trauma, meningitis, hemorrhage) or interference with disease rating (autism) before Screening
•Presence of percutaneous feeding tube placement prior to enrollment
•Has received stem cell, gene therapy, or ERT
•Presence of contraindications for neurosurgery (e.g., congenital heart disease, severe respiratory impairment, or clotting abnormalities)
•Presence of contraindications for MRI scans (e.g., cardiac pacemaker, metal fragment or chip in the eye, aneurysm clip in the brain)
•Episode of generalized motor status epilepticus within 4 weeks before the First Dose visit
•Severe infection (e.g., pneumonia, pyelonephritis, or meningitis) within 4 weeks before the First Dose visit (enrollment may be postponed)
•Presence of ventricular abnormality (hydrocephalus, malformation)
•Presence of ventricular shunt
+4 more criteria

Locations (4)

Nationwide Children's Hospital

Columbus, Ohio, United States

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, Germany

Children's Hospital Bambino Gesù,IRCCS

Rome, Piazza, Italy

Great Ormond Street Childrens Hospital

London, United Kingdom

Key Dates

Start Date

January 22, 2016

Primary Completion Date

April 20, 2022

Completion Date

April 20, 2022

Last Updated

February 17, 2025

Enrollment

ACTUAL participants

Conditions

Jansky-Bielschowsky DiseaseBatten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2CLN2 DiseaseCLN2 Disorder

Interventions

BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)

BIOLOGICAL

Intracerebroventricular access device

DEVICE

Clinical and Neuropsychological Investigations in Batten Disease

NCT01873924

Neuronal Ceroid LipofuscinosisNeuronal Ceroid Lipofuscinosis CLN1+8 more

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RECRUITINGPHASE1

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

NCT02254863

AdrenoleukodystrophyBatten Disease+10 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 17, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

Inclusion Criteria

Exclusion Criteria

Clinical and Neuropsychological Investigations in Batten Disease

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Cerliponase Alfa Observational Study in the US

AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis