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

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ClinicalTrials.gov Identifier: NCT02485899
Recruitment Status : Completed
First Posted : June 30, 2015
Last Update Posted : February 11, 2021
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Study Description
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Brief Summary:
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 or disease Intervention/treatment Phase
Jansky-Bielschowsky Disease Batten Disease Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 CLN2 Disease CLN2 Disorder Biological: BMN 190 Device: Intraventricular Access Device Phase 1 Phase 2

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 BMN 190 treatment. The 190-202 study is an open label extension protocol to assess long-term safety and efficacy.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
Allocation: N/A
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
Actual Study Start Date : February 2015
Actual Primary Completion Date : December 10, 2020
Actual Study Completion Date : December 10, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Safety

Arms and Interventions
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Arm Intervention/treatment
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

Device: Intraventricular 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.


Outcome Measures
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Primary Outcome Measures :
  1. 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.

  2. Motor and Language Changes [ Time Frame: up to 240 weeks ]
    Change in motor and language (ML) 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 :
  1. Quantitative Assessment of Magnetic Resonance Imaging [ Time Frame: up to 240 weeks ]
    Changes in quantitative assessment of MRI.

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

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


Eligibility Criteria
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Information from the National Library of Medicine

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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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02485899


Locations
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United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Germany
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, Germany, 20246
Italy
Children's Hospital Bambino Gesù,IRCCS
Rome, Piazza, Italy, 00165
United Kingdom
Great Ormond Street Childrens Hospital
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
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Study Director: Medical Monitor, MD BioMarin Pharmaceutical
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT02485899    
Other Study ID Numbers: 190-202
First Posted: June 30, 2015    Key Record Dates
Last Update Posted: February 11, 2021
Last Verified: February 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: Yes
Keywords provided by BioMarin Pharmaceutical:
Late infantile Neuronal Ceroid Lipofuscinosis Type 2
LINCL
NCL2
CLN2
Jansky-Bielschowsky disease
Additional relevant MeSH terms:
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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