SBC-103 Intravenously in SBC-103 Treatment Naïve MPS II (NGLU-CL01-T)
Research type
Research Study
Full title
A Phase I/II Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously
IRAS ID
183135
Contact name
Suresh Vijay
Contact email
Sponsor organisation
Alexion Pharmaceuticals Inc
Eudract number
2015-001983-20
Clinicaltrials.gov Identifier
IND 118402, IND Number:
Duration of Study in the UK
3 years, 5 months, 1 days
Research summary
Mucopolysaccharidosis type IIIB (MPS IIIB), also known as Sanfilippo B syndrome, is a very rare lysosomal storage disorder. The disease is caused by mutations in the alpha-N-acetylglucosaminidase (NAGLU) gene. This leads to the accumulation of heparan sulfate in the brain and other tissues which results in the devastating complications of this disease. Typically patients have a near-normal development during the first 2 years of life, followed by a slowing and full stagnation of development at around 3 to 4 years, and finally regression of cognitive capabilities. Patients usually become fully dependent on care early in their teenage years. There are currently no safe or effective therapies for the treatment of MPS IIIB. Supportive therapies are used to attempt to mitigate some of the effects of the disease. Options for management of clinical symptoms are limited, and include the use of central nervous system medications to control seizures, behavioural problems, and sleep problems.
SBC103 (recombinant human NAGLU) is being developed as an enzyme replacement therapy (ERT) for MPS IIIB. This study will evaluate the safety and tolerability of intravenous (IV) administration of SBC-103 in previously studied, SBC-103 treatment naïve participants with MPS IIIB who participated in the NGLU-CL01 study.
Following completion of screening assessments to confirm study eligibility, up to 5 participants will receive SBC-103 by IV infusion once every other week for a total duration of up to 3 years (156 weeks). Each participant will be treated with 1 mg/kg Intravenous (IV) for a minimum of 12 weeks. If appropriate, following a review of the 12 week safety data for each patient (and other available safety data) the dose may be escalated to 3 mg/kg. Safety assessments will be performed in all participants.
This study will include procedures- physical examination, blood samples, ECG’s, questionnaires and general anaesthesia and lumbar punctures.
REC name
North West - Haydock Research Ethics Committee
REC reference
15/NW/0535
Date of REC Opinion
20 Jul 2015
REC opinion
Favourable Opinion