A Treatment Study of Mucopolysaccharidosis Type IIIB



Status:Recruiting
Conditions:Other Indications, Metabolic
Therapuetic Areas:Pharmacology / Toxicology, Other
Healthy:No
Age Range:1 - 10
Updated:8/31/2018
Start Date:April 2016
End Date:March 2019
Contact:BioMarin Trial Inquiries
Email:medinfo@bmrn.com

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A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Intracerebroventricular BMN 250 in Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

The study's primary objectives are to evaluate the safety and tolerability of BMN 250
administered to subjects with MPS IIIB via an ICV reservoir and catheter and to evaluate the
impact of BMN 250 on cognitive function in patients with MPS IIIB as assessed by the
Development Quotient.


Inclusion Criteria:

Individuals eligible to participate in Part 1 of this study must meet all of the following
criteria:

- Has deficient NAGLU enzyme activity at Screening. Blood for NAGLU enzyme activity will
be collected and analyzed centrally.

- Is ≥ 1 and < 11 years of age (at least 1 of the 3 subjects in Part 1 must be ≥ 1 and <
6 years of age)

- Has presented with signs/symptoms consistent with MPS IIIB; for individuals who have
not presented with signs/symptoms of disease (eg, siblings of known patients), the
determination of eligibility will be at the discretion of the BioMarin medical monitor
in conjunction with the site investigator.

- Written informed consent from parent or legal guardian and assent from subject, if
required

- Has the ability to comply with protocol requirements, in the opinion of the
investigator

Individuals eligible to participate in Part 2 of this study must meet all of the following
criteria:

- Participated in and met protocol requirements for transitioning from Study 250-901 or
participated in Part 1 of Study 250-201

- Written informed consent from parent or legal guardian and assent from subject, if
required

Exclusion Criteria:

Individuals who meet any of the following exclusion criteria are ineligible to participate
in Part 1 of the study:

- Has received stem cell, gene therapy or ERT for MPS IIIB

- Has contraindications for neurosurgery (eg, congenital heart disease, severe
respiratory impairment, or clotting abnormalities)

- Has contraindications for MRI scans (eg, cardiac pacemaker, metal fragment or chip in
the eye, or aneurysm clip in the brain)

- Has a history of poorly controlled seizure disorder

- Is prone to complications from intraventricular drug administration, including
patients with hydrocephalus or ventricular shunts

- Has received any investigational medication within 30 days prior to the Baseline visit
or is scheduled to receive any investigational drug during the course of the study

- Has a medical condition or extenuating circumstance that, in the opinion of the
investigator, might compromise the subject's ability to comply with protocol
requirements, the subject's well-being or safety, or the interpretability of the
subject's clinical data.

- Is pregnant at any time during the study

Individuals who meet any of the following exclusion criteria are ineligible to participate
in Part 2 of this study:

- Has received stem cell, gene therapy or ERT for MPS IIIB

- Has contraindications for neurosurgery (eg, congenital heart disease, severe
respiratory impairment, or clotting abnormalities)

- Has contraindications for MRI scans (eg, cardiac pacemaker, metal fragment or chip in
the eye, or aneurysm clip in the brain)

- Is prone to complications from intraventricular drug administration, including
patients with hydrocephalus or ventricular shunts

- Has received any investigational medication within 30 days prior to the Baseline visit
or is scheduled to receive any investigational drug during the course of the study

- Has a medical condition or extenuating circumstance that, in the opinion of the
investigator, might compromise the subject's ability to comply with protocol
requirements, the subject's well-being or safety, or the interpretability of the
subject's clinical data.

- Is pregnant at any time during the study
We found this trial at
2
sites
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Hamburg,
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Oakland, California 94609
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Oakland, CA
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