CLN2 DiseaseNCT02963350
A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease
A study testing BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1) in people with CLN2 Disease, run by BioMarin Pharmaceutical at 5 sites in 4 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- CLN2 Disease
- What is being tested?
- BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1)
- How many people
- Not given
- Where
- 5 sitesin 4 countries
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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).
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- 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)
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
United States2 sites
Study site
Columbus, Ohio
Study site
Orange, California
About this study
- 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.
- Conditions
- CLN2 Disease
- Being tested
- BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1)Drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CLN2 DiseaseStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jessica Cohen Pfeffer, MD, Study director, Medical Monitor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02963350
- Sponsor's protocol ID
- 190-502
- Status last verified
- Jul 2017
- First submitted
- 9 Nov 2016
- Last updated
- 24 Jul 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease
- ClinicalTrials.gov ID
- NCT02963350
- Acronym / sponsor ID
- 190-502
- Conditions
- CLN2 Disease
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- First posted
- 15 Nov 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
- 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 design
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (15 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1) | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jessica Cohen Pfeffer, MD study director
Medical Monitor
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1) in people with CLN2 Disease, run by BioMarin Pharmaceutical at 5 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 24 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Germany, Italy, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jul 2017.