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

• Register: ClinicalTrials.gov
• Last refreshed on: 20 March 2023
• Main ID: NCT02678689
• Date of registration: 15/01/2016
• Prospective Registration: Yes
• Primary sponsor: BioMarin Pharmaceutical
• Public title: A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
• Scientific title: 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
• Date of first enrolment: February 2016
• Target sample size: 14
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02678689
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Germany, Italy, United Kingdom, United States

Contacts

• Name: Medical Director, MD
• Affiliation: BioMarin Pharmaceutical

Key inclusion & exclusion criteria

Enrollment over the age of 2 years is complete.

Inclusion Criteria:

- 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, 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 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

- 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



Age minimum: 0 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

CLN2 Disease

CLN2 Disorder

Batten Disease

Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2

Jansky-Bielschowsky Disease

Intervention(s)

Device: Intraventricular access device

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

Primary Outcome(s)

Change in the 0-6-point Motor/Language (ML) score on the Hamburg CLN2 rating scale [Time Frame: Up to 144 weeks + 6 month follow up post last study treatment]

Immunogenicity of BMN 190 in CSF and serum [Time Frame: Up to 144 weeks]

Incidence and severity of adverse events as assessed by CTCAE v 4.0 [Time Frame: Up to 144 weeks + 6 month follow up post last study treatment]

Secondary Outcome(s)

Assess time to disease manifestation for asymptomatic patients [Time Frame: Up to 144 weeks]

Vital signs [Time Frame: Up to 144 weeks + 6 month follow up post last study treatment]

Change in Brain Volumes as Assessed by Cranial Magnetic Resonance Imaging (MRI) [Time Frame: Up to 144 weeks]

Change in the total Hamburg CLN2 rating scale [Time Frame: Up to 144 weeks + 6 month follow up post last study treatment]

Change in CSF and Plasma laboratory parameters [Time Frame: Up to 144 weeks]

Electrocardiogram (ECG), 3 or 5-lead, 12-lead [Time Frame: Up to 144 weeks + 6 month follow up post last study treatment]

Physical examination [Time Frame: Up to 144 weeks + 6 month follow up post last study treatment]

Neurological examinations [Time Frame: Up to 144 weeks]

Change in clinical laboratory tests [Time Frame: Up to 144 weeks]

Secondary ID(s)

190-203

Source(s) of Monetary Support

Please refer to primary and secondary sponsors