- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02963350
A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease
July 21, 2017 updated by: BioMarin Pharmaceutical
- To provide access to BMN 190 to patients with CLN2 disease who cannot participate in a clinical trial.
- To collect additional information on the safety and tolerability of BMN 190 administration in patients with CLN2 disease.
Study Overview
Status
Approved for marketing
Conditions
Intervention / Treatment
Study Type
Expanded Access
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Hamburg, Germany
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Rome, Italy
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London, United Kingdom
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California
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Orange, California, United States
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Ohio
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Columbus, Ohio, United States
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
2 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
N/A
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Diagnosed with CLN2 disease as confirmed by deficient TPP1 enzyme activity in leukocytes or molecular analysis by identifying 2 known pathogenic mutations. If enzyme analysis is performed by dried blood spot, diagnosis must be confirmed with molecular testing
- Age ≥2 old at the time of informed consent
- 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 program has been explained, and prior to any program assessments.
- If sexually active, must be willing to use 2 forms of acceptable methods of contraception while participating in the program.
- If female of childbearing potential, must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests during the program.
- Willing and able to comply with all program procedures.
Exclusion Criteria:
- Another inherited neurologic disease, e.g., other forms of CLN or seizures unrelated to TPPI deficiency/CLN2 disease (patients with febrile seizures may be eligible).
- Has received stem cell, gene therapy, or ERT for CLN2 disease.
- Contraindications for neurosurgery (e.g., congenital heart disease, severe respiratory impairment, or clotting abnormalities).
- 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 infusion.
- Presence of ventricular abnormality (hydrocephalus, malformation).
- Presence of ventricular shunt.
- Has known hypersensitivity to any of the components of BMN 190.
- Currently enrolled or previously enrolled in a clinical study with BMN 190.
- Use of any investigational product or investigational medical device within 30 days prior to Baseline, or requirement for any investigational agent prior to completion of all scheduled program assessments.
- Have travel plans that may interfere with dosing regimen, scheduled program visits and safety monitoring.
- Has a medical condition or extenuating circumstance that, in the opinion of the physician, might compromise the patient's ability to comply with the protocol required testing or procedures or compromise the patient's wellbeing, safety, or clinical interpretability.
- Pregnancy any time during the program; a female patient judged by the physician to be of childbearing potential will be tested for pregnancy.
- A CLN2 combined motor/language score of less than 1 (apply to US only)
- Asymptomatic (symptomatic is defined as having any evidence of neurological involvement attributed to CLN2 disease irrespective of the CLN2 score, including clinical signs and symptoms of disease such as seizures, ataxia, language delay or other developmental delays) (apply to US only)
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Jessica Cohen Pfeffer, MD, Medical Monitor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Registration Dates
First Submitted
November 9, 2016
First Submitted That Met QC Criteria
November 10, 2016
First Posted (Estimate)
November 15, 2016
Study Record Updates
Last Update Posted (Actual)
July 24, 2017
Last Update Submitted That Met QC Criteria
July 21, 2017
Last Verified
July 1, 2017
More Information
Terms related to this study
Other Study ID Numbers
- 190-502
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease | CLN2 DisorderUnited States, Germany, Italy, United Kingdom
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Nadir Hastalıkları Araştırma DerneğiRecruitingNeuronal Ceroid Lipofuxinosis Type2 (CLN2)Turkey
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Jessica ScherrBioMarin PharmaceuticalEnrolling by invitationBatten Disease | Neuronal Ceroid-Lipofuscinoses | CLN2United States
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky DiseaseUnited States, Italy, Germany, United Kingdom
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Universitätsklinikum Hamburg-EppendorfRecruitingBatten Disease | Neuronal Ceroid Lipofuscinosis | CLN1 Disease | CLN2 Disease | CLN3 Disease | CLN4 Disease | CLN5 Disease | CLN6 Disease | CLN7 Disease | CLN8 Disease | CLN10 Disease | CLN11 Disease | CLN12 Disease | CLN13 Disease | CLN14 DiseaseGermany
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REGENXBIO Inc.WithdrawnNeuronal Ceroid Lipofuscinosis Type 2 (CLN2)
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University of RochesterRecruitingBatten Disease | Neuronal Ceroid Lipofuscinosis | Neuronal Ceroid Lipofuscinosis CLN6 | Neuronal Ceroid Lipofuscinosis CLN3 | Neuronal Ceroid Lipofuscinosis CLN5 | Neuronal Ceroid Lipofuscinosis CLN1 | Neuronal Ceroid Lipofuscinosis CLN2 | Neuronal Ceroid Lipofuscinosis CLN7 | Neuronal Ceroid Lipofuscinosis... and other conditionsUnited States
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REGENXBIO Inc.WithdrawnLate-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
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Children's Hospital of Orange CountyActive, not recruitingNeuronal Ceroid-Lipofuscinoses | Neuronal Ceroid Lipofuscinosis CLN2 | Spinocerebellar Ataxia, Autosomal Recessive 7United States
Clinical Trials on BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1)
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease | CLN2 DisorderUnited States, Germany, Italy, United Kingdom
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky DiseaseUnited States, Italy, Germany, United Kingdom
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease | CLN2 DisorderUnited States, Germany, Italy, United Kingdom
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Digna Biotech S.L.Unknown
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Zensun Sci. & Tech. Co., Ltd.Recruiting
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EMD SeronoTerminatedArthritis, PsoriaticUnited States, Canada
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)CompletedAcute Myeloid Leukemia | Myelodysplastic Syndrome | Myeloproliferative Neoplasm | Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Cord Blood Transplant RecipientUnited States
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Columbia UniversityTercicaTerminatedGrowth Hormone DeficiencyUnited States
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CSPC ZhongQi Pharmaceutical Technology Co., Ltd.RecruitingUterine Cervical CancerChina
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CSPC ZhongQi Pharmaceutical Technology Co., Ltd.RecruitingLymphoma | Cervical Cancer | Malignant Mesothelioma | Advanced Solid Tumours | Non-Small-Cell Lung CancerChina