Trial record 11 of 16 for:    Batten Disease

Safety and Efficacy Study of BMN190 for the Treatment of Patients With CLN2 Disease

This study is currently recruiting participants.
Verified March 2014 by BioMarin Pharmaceutical
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01907087
First received: July 19, 2013
Last updated: March 21, 2014
Last verified: March 2014
  Purpose

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.


Condition Intervention Phase
Jansky-Bielschowsky Disease
Batten Disease
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
CLN2 Disease
Biological: BMN 190
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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 Type 2 (CLN2) Disease

Resource links provided by NLM:


Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • To evaluate the safety of every other week infusions of BMN 190 based on: vital signs, physical examination, electrocardiogram tests, clinical laboratory tests, adverse events, concomitant medications, immunogenicity tests [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]

    Vital signs, adverse events, concomitant medications: Screening, Baseline, Weeks 1 to 49.

    Physical examination: Screening, Baseline, Weeks 1 to 49.

    Electrocardiogram tests: Baseline, Weeks 1, 24 and 49

    Clinical laboratory tests: Baseline, Weeks 1, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49

    Immunogenicity tests: Baseline, Weeks 1, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49


  • To evaluate the efficacy of every other week infusions of BMN 190 by monitoring changes in clinical measures as measured by the CLN2 disease rating scale [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
    Screening, baseline, Weeks 1, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49


Secondary Outcome Measures:
  • To evaluate the efficacy of every other week infusions of BMN 190 by monitoring changes in clinical measures as measured by magnetic resonance imaging (MRI) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    Screening, Baseline, Weeks 1, 9, 17, 33, 49

  • To determine the pharmacokinetic (PK) parameters of infused BMN 190 in subjects with CLN2 [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
    Weeks 1, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49


Estimated Enrollment: 22
Study Start Date: September 2013
Estimated Study Completion Date: March 2016
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: BMN190
recombinant human tripeptidyl peptidase-1 (rhTPP1)
Biological: BMN 190
30-300 mg ICV infusion administered every other week for at least 48 weeks.
Other Name: recombinant human tripeptidyl peptidase-1 (rhTPP1)

Detailed Description:

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. This is an open label Phase 1/2 study conducted in patients with CLN2 disease. Efficacy measures (disease rating scale and MRI) will be compared to a natural history control.

The study will be conducted under cGCP and patients will be closely monitored.

  Eligibility

Ages Eligible for Study:   3 Years to 16 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Has a diagnosis of CLN2 determined by TPP1 enzyme activity (dried blood spot) available at study entry. If no genotype information is available, blood will be collected for CLN2 gene analysis at baseline. In addition, blood for TPP1 enzyme activity (dried blood spot) will be collected at baseline to be analyzed centrally
  • Has mild to moderate disease documented by a two-domain score of 3- 6 on motor and language domains of the Hamburg Scale, with a score of at least 1 in each of these two domains
  • Written informed consent from parent or legal guardian and assent from subject, if appropriate
  • Has the ability to comply with protocol requirements, in the opinion of the investigator

Exclusion Criteria:

  • Is less than 3 years old at enrollment
  • Has another inherited neurologic disease, e.g. other forms of CLN or seizures unrelated to CLN2 (patients with febrile seizures may be eligible)
  • Requires ventilation support, except for noninvasive support at night
  • Has received stem cell, gene therapy, or ERT for CLN2
  • Has contraindications for neurosurgery (e.g., congenital heart disease, severe respiratory impairment, or clotting abnormalities)
  • Has contraindications for MRI scans (e.g., cardiac pacemaker, metal fragment or chip in the eye, aneurysm clip in the brain) - Has generalized motor status epilepticus within 4 weeks before the First Dose visit, taking care that status epilepticus is on clinical examination and not only electroencephalogram (EEG) (enrollment may be postponed)
  • Has severe infection (e.g., pneumonia, pyelonephritis, or meningitis) within 4 weeks before the First Dose visit (enrollment may be postponed)
  • 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 requirements or compromise the subject's well being, safety, or clinical interpretability
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01907087

Contacts
Contact: Janet Nuttall, MPH jnuttall@bmrn.com
Contact: David Jacoby, MD djacoby@bmrn.com

Locations
Germany
University Hamburg-Eppendorf Recruiting
Hamburg, Germany, 20246
Contact: Angela Schulz, MD       an.schulz@uke.de   
Principal Investigator: Angela Schulz, MD         
United Kingdom
Evelina Children's Hospital Recruiting
London, United Kingdom, WC2R 2LS
Contact: Ruth Williams, MD       Ruth.Williams@gstt.nhs.uk   
Contact: Rachael Pennington, RN       Rachael.Pennington@gstt.nhs.uk   
Principal Investigator: Ruth Williams, MD         
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
Study Director: David Jacoby BioMarin Pharmaceutical
  More Information

No publications provided

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01907087     History of Changes
Other Study ID Numbers: 190-201
Study First Received: July 19, 2013
Last Updated: March 21, 2014
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
Germany: Federal Institute for Drugs and Medical Devices

Keywords provided by BioMarin Pharmaceutical:
Late infantile Neuronal Ceroid Lipofuscinosis Type 2
LINCL
NCL2
CLN2
Jansky-Bielschowsky disease

Additional relevant MeSH terms:
Neuronal Ceroid-Lipofuscinoses
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Lipid Metabolism Disorders
Metabolic Diseases

ClinicalTrials.gov processed this record on April 21, 2014