A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Patients With CLN2 Disease

• Status: Enrolling by invitation
• Trial end date: 0000-00-00
• Target enrollment: 5
• Participant gender: Both

Summary

This Phase 2 open-label study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration at 300mg every other week (qow) for a period of 96 weeks, in patients with CLN2. The study is designed to assess disease progression in siblings of children enrolled in the 190-201 study.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: BioMarin Pharmaceutical
• Last Updated: 2016-06-29

Criteria

Inclusion Criteria:

• Diagnosis of CLN2 disease determined by TPP1 enzyme activity available at Screening

• At least 1 sibling with confirmed CLN2 disease who was enrolled in Study 190-201

• 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

• Age ≥ 1 year at the time of informed consent

• Written informed consent from parent or legal guardian and assent form subject, if appropriate

• Ability to comply with protocol required assessments (laboratory sample collection, EEG, ECG, 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

• Has received stem cell, gene therapy, or ERT for CLN2 disease

• 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

• Has known hypersensitivity to any of the components of BMN 190

• Has received any investigational mediation within 30 days before the first infusion of study drug or is scheduled to receive any investigational drug other than BMN 190 during the course of the study

• Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with the protocol required testing or procedures or compromise the subject's well being, safety, or clinical interpretability

• Pregnancy any time during the study; a female subject judged by the investigator to be of childbearing potential will be tested for pregnancy