NCT02678689

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 17 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Jansky-Bielschowsky Disease
• Batten Disease
• Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
• CLN2 Disease
• CLN2 Disorder

Interventions

Type	Name	Description
BIOLOGICAL	BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)
DEVICE	Intracerebroventricular access device	Surgical implantation of an MRI compatible ICV access device in the lateral ventricle of the right hemisphere is required for administration of study drug.

Timeline

• Start date: 2016-01-22
• Primary completion: 2022-04-20
• Completion: 2022-04-20
• First posted: 2016-02-10
• Last updated: 2025-02-17
• Results posted: 2025-02-17

Locations

4 sites across 4 countries: United States, Germany, Italy, United Kingdom

Regulatory

• FDA-regulated drug study
• FDA-regulated device study