Extension Study for BMN 250 in Patients MPS IIIB

• Research type

  Research Study

• Full title

  A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

• IRAS ID

  237536

• Sponsor organisation

  BioMarin Pharmaceutical Inc.

• Eudract number

  2017-003083-13

• Duration of Study in the UK

  4 years, 5 months, 0 days

• Research summary

  Mucopolysaccharidosis type IIIB (MPS IIIB) is a rare, severe neurodegenerative disorder that primarily affects the brain and spinal cord. People with MPS IIIB are unable to produce the enzyme required to break down large sugar molecules called glycosaminoglycans, specially one called heparin sulphate. This leads to symptoms such as progressive dementia, aggressive behaviour, hyperactivity, seizures, deafness, loss of vision, and an inability to sleep for more than a few hours at a time.

  The previous phase 1/2 study (250-201) evaluated the effects of the study drug, BMN 250, on the body at doses up to 300 mg/week and it was confirmed that 300 mg/week was the maximum tolerated dose.

  The purpose of this extension study is to determine the long-term safety effects of the study drug and further understand how it affects the participant's cognitive function. Only subjects who were on the 250-201 study will be screened for this follow-on study. All participants will be administered with 300 mg of the drug via the drug delivery system already implanted in the brain. Sleep quality and behaviour will be tracked while 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.

  Participants are expected to be involved in the study for around 4 and a half years.

  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

  North West - Liverpool Central Research Ethics Committee

• REC reference

  17/NW/0711

• Date of REC Opinion

  2 Mar 2018

• REC opinion

  Further Information Favourable Opinion