An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease

• Register: EUCTR
• Last refreshed on: 30 April 2019
• Main ID: EUCTR2014-003480-37-GB
• Date of registration: 20/10/2014
• Prospective Registration: Yes
• Primary sponsor: BioMarin Pharmaceutical Inc.
• Public title: An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease
• Scientific title: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease
• Date of first enrolment: 10/03/2015
• Target sample size: 23
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-003480-37
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: yes; Other specify the comparator: External Control (Historical Control); Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Germany, Italy, United Kingdom, United States

Contacts

• Name: Clinical Trials Information
• Affiliation: BioMarin Pharmaceutical Inc.

• Name: Clinical Trials Information
• Affiliation: BioMarin Pharmaceutical Inc.

Key inclusion & exclusion 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
•If sexually active, must be willing to use an acceptable method of contraception while participating in the study
•If female, of childbearing potential, must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests done during the study

Are the trial subjects under 18? yes
Number of subjects for this age range: 23
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Late-Infantile Neuronal Ceroid Lipofuscinosis type 2 (CLN2).
MedDRA version: 20.1 Level: LLT Classification code 10052074 Term: Neuronal ceroid lipofuscinosis NOS System Organ Class: 100000004850

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Trade Name: Brineura
Product Code: BMN 190
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: cerliponase alfa
CAS Number: 151662-36-1
Current Sponsor code: BMN 190
Other descriptive name: RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 30-

Primary Outcome(s)

Primary end point(s): The primary points of the study include the following:
•to evaluate the long-term safety of BMN 190 administration at 300 mg qow in patients with CLN2
•to assess change in motor and language subscales of the CLN2 disease rating scale in patients with CLN2 receiving BMN 190 at 300 mg qow

Timepoint(s) of evaluation of this end point: Patients will complete safety assessments including CSF surveillance labs and a brief physical examination every 2 weeks. Clinical laboratory tests and immunogenicity tests will be performed every 12 weeks. Efficacy per the disease scales will be assessed every 8 weeks.

Secondary Objective: The secondary objectives of the study include the following:
•to assess changes in quantitative assessment of MRI
•to assess change in CLN2 disease scale total score
•to evaluate quality of life (QOL) with long-term BMN 190 administration

Main Objective: The primary objectives of this study include the following:
•to evaluate the long-term safety of BMN 190 administration at 300 mg qow in patients with CLN2
•to assess change in motor and language subscales of the CLN2 disease rating scale in patients with CLN2 receiving BMN 190 at 300 mg qow

Secondary Outcome(s)

Secondary end point(s): Secondary endpoints of the study include the following:
•to assess changes in quantitative assessment of MRI
•to assess change in CLN2 disease scale total score
•to evaluate quality of life (QOL) with long-term BMN 190 administration

Timepoint(s) of evaluation of this end point: MRI and quality of life measures will be collected every 24 weeks. Total score on the disease scales will be assessed every 8 weeks.

Secondary ID(s)

190-202

Source(s) of Monetary Support

BioMarin Pharmaceutical Inc.

Ethics review

Status: Approved
Approval date:
Contact: