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Trial record 2 of 2 for:    cerliponase alfa

A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT02485899
First received: April 24, 2015
Last updated: October 28, 2016
Last verified: October 2016
  Purpose
The Phase 1/2 study (190-201) evaluated the efficacy and safety of doses up to 300 mg/every other week (qow) BMN 190 in patients with CLN2. The dose and regimen for this study (190-202) are based on the results of the 190-201 study. The rationale for this phase 2 extension study is to provide patients who complete the 190-201 study with the option to continue to receive continued BMN 190 treatment. The 190-202 study is an open label extension protocol to assess long-term safety and efficacy.

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

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

Resource links provided by NLM:


Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • Long Term Safety as assessed by analysis of adverse events. [ Time Frame: up to 240 weeks ]
    Long term safety of BMN 190 administered to subjects with CLN2 disease via an implanted intracerebroventricular (ICV) reservoir and cannula as assessed by analysis of adverse events.

  • Motor and Language Changes [ Time Frame: up to 240 weeks ]
    Change in motor and language subscales of the CLN2 disease rating scale in patients with CLN2 following administration of 300 mg every other week of BMN 190.


Secondary Outcome Measures:
  • Quantitative Assessment of Magnetic Resonance Imaging [ Time Frame: up to 240 weeks ]
    Changes in quantitative assessment of MRI.

  • CLN2 Disease Scale Score [ Time Frame: up to 240 weeks ]
    Changes in the CLN2 disease scale total score

  • Quality of Life Changes [ Time Frame: up to 240 weeks ]
    Changes in the quality of life with long-term use of BMN 190


Other Outcome Measures:
  • Age-Appropriateness of Developmental Milestone [ Time Frame: up to 240 weeks ]
    Evaluation of age-appropriateness of developmental Milestones

  • Impact of Treatment on BioMarkers [ Time Frame: up to 240 weeks ]
    Evaluation of impact of treatment on disease-related CSF and/or plasma biomarkers


Enrollment: 23
Study Start Date: February 2015
Estimated Primary Completion Date: December 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1) Biological: BMN 190
300 mg ICV infusion administered every other week for up to 240 weeks
Other Names:
  • recombinant human tripeptidyl peptidase-1 (rhTPP1)
  • cerliponase alfa

Detailed Description:
BMN 190 is a recombinant form of human tripeptidyl peptidase 1 (TPP1), the enzyme deficient in patients with CLN2 disease (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 restore TPP1 enzyme activity. BMN 190 is designed to reduce the progressive, pathologic accumulation of lysosomal storage material, and improve the symptoms of disease. The Phase 1/2 study (190-201) evaluated the efficacy and safety of doses up to 300 mg/every other week (qow) BMN 190 in patients with CLN2. The dose and regimen for this study (190-202) are based on the results of the 190-201 study. The rationale for this phase 2 extension study is to provide patients who complete the 190-201 study with the option to continue to receive continued BMN 190 treatment. The 190-202 study is an open label extension protocol to assess long-term safety and efficacy.
  Eligibility

Ages Eligible for Study:   3 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Must have completed 48 weeks in Study 190-201.
  • Is willing and able to provide written, signed informed consent. Or, in the case of patients under the age of 18 (or other age as defined by regional law or regulation), provide written assent (if required) and have written informed consent, signed by a legally authorized representative, after the nature of the study has been explained, and prior to performance of research-related procedures.
  • 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.
  • If female, of childbearing potential, must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests done during the study.

Exclusion Criteria:

  • Has had a loss of 3 or more points in the combined motor and language components of the Hamburg CLN2 rating scale between Baseline of Study 190-201 and the Study Completion visit in Study 190-201 and would not benefit from enrolling in the study in the Investigator's discretion.
  • Has a score of 0 points on the combined motor and language components of the Hamburg CLN2 rating scale.
  • Is pregnant or breastfeeding, at Baseline, or planning to become pregnant (self or partner) at any time during the study.
  • Has used any investigational product (other than BMN 190 in 190-201), or investigational medical device, within 30 days prior to Baseline; or is required to use any investigational agent prior to completion of all scheduled study assessments.
  • Has a concurrent disease or condition that would interfere with study participation, or pose a safety risk, as determined by the Investigator.
  • Has any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02485899

Locations
United States, Ohio
Columbus, Ohio, United States
Germany
Hamburg, Germany
Italy
Rome, Italy
United Kingdom
London, United Kingdom
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
Study Director: Temitayo Ajayi, MD BioMarin Pharmaceutical
  More Information

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT02485899     History of Changes
Other Study ID Numbers: 190-202
Study First Received: April 24, 2015
Last Updated: October 28, 2016

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 September 21, 2017