Phase 1/2 study of BMN 250 in MPS IIIB patients
Research type
Research Study
Full title
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)
IRAS ID
190416
Contact name
Maureen Cleary
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
2015-001985-25
Duration of Study in the UK
2 years, 0 months, 1 days
Research summary
Mucopolysaccharidosis type IIIB (MPS IIIB) is a severe neurodegenerative disorder. The purpose of this study is to learn what effects, good or bad, the study drug has on the disease and how it works in the body. Safety will be carefully watched and controlled.
People with MPS IIIB do not have enough NAGLU, an enzyme needed to break down a molecule normally found in the body, leading to the symptoms seen with MPS IIIB. BMN 250 is intended as an enzyme replacement therapy.
Up to 33 children aged 1-10 will participate in this study for at least a year. There will be at least 1 study visit per week, each lasting from 1 to 3 days.
Surgery will be required at the start of the study to implant a drug delivery system in the brain. A number tests and procedures will be done at the study visits, including: blood and urine samples, physical examinations, ECG (electrical test of the heart rhythm), EEG (electrical test of the brain wave pattern), questionnaires, MRI scan and collection of cerebrospinal fluid. Some of these assessments will be done under general anaesthetic or strong sedation.
The study has 2 parts. In Part 1, 3 participants will each receive at least 4 weekly doses of BMN 250 at up to 3 different dose levels until a dose level is determined for Part 2 of the study. In Part 2, up to 30 participants previously enrolled in Study 250-901 will receive BMN 250 weekly for 48 weeks. The 3 participants from Part 1 will also move into Part 2 and receive weekly BMN 250 weekly for an additional 48 weeks.
The study is sponsored by BioMarin and is planned to be run at Great Ormond Street Hospital in the UK and other centres worldwide.REC name
London - Central Research Ethics Committee
REC reference
15/LO/1796
Date of REC Opinion
2 Nov 2015
REC opinion
Favourable Opinion