Mucopolysaccharidosis III, type B (MPS IIIB), Sanfilippo B MedDRA version: 19.0 Level: LLT Classification code 10056918 Term: Sanfilippo's syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 19.0 Level: PT Classification code 10056890 Term: Mucopolysaccharidosis III System Organ Class: 100
Conditions
Sponsors
Alexion Pharmaceuticals, Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Previous participation in the NGLU-CL01 study. 2. Subject consents or subject’s parent or legal guardian (if applicable) grants consent for the subject to participate in the study and provides informed consent prior to any study procedures being performed. If the subject is of minor age; he/she is willing to provide assent where required per local regulations, and if deemed able to do so. 3. Female subjects who are of childbearing potential at the time of consent or who become of childbearing potential during participation on study (a) must have a negative urine pregnancy test at Screening, (b) cannot be breast feeding, and (c) must use a highly reliable method of birth control (expected failure rate less than 5% per year) for the duration of the study and for 30 days after last dose of SBC-103. Women may be considered of non-childbearing potential if they have not started their menses or are surgically sterile (i.e., total hysterectomy or bilateral salpingo-oophorectomy). 4. Male subjects must use a highly reliable method of birth control (expected failure rate less than 5% per year) during any sexual contact with females of childbearing potential while participating in the study and for 30 days following discontinuation from this study even if he has undergone a successful vasectomy. 5. Willingness and ability to comply with protocol requirements to the extent that may be expected of a subject with cognitive impairment. Are the trial subjects under 18? yes Number of subjects for this age range: 3 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Received treatment with gene therapy at any time, or any investigational drug (including high dose genistein > 150 mg/kg/day) or device intended as a treatment for MPS IIIB within 30 days prior to Screening, or is currently being treated in another study that involves an investigational drug or device. 2. Has any internal or non-removable external metal items 3. Previous hematopoietic stem cell or bone marrow transplant. 4. Known or suspected hypersensitivity to anaesthesia or the use of a sedative is contraindicated for any other reason. 5. History of poorly controlled seizure disorder. 6. A bleeding disorder, or any other medical condition or circumstance in which a lumbar puncture (for collection of CSF) is contraindicated according to local institutional policy. 7. Known hypersensitivity to eggs. Subjects at high risk for food allergy that may include eggs should be tested according to local guidelines. 8. Other medical conditions or comorbidities
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of intravenous administration of SBC-103 in previously studied, SBC-103 treatment naïve subjects with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) who participated in the NGLU-CL01 study. ;Secondary Objective: PK, activity, PD/efficacy of treatment with SBC-103 administered IV ;Primary end point(s): Safety and tolerability of SBC-103 in subjects with MPS IIIB. ;Timepoint(s) of evaluation of this end point: Trial duration | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Trial duration; Secondary end point(s): • PK profile of SBC-103 • Onset and magnitude of changes in levels of total HS in CSF, serum, and urine following dosing with SBC-103 administered IV. • Changes from the NGLU-CL01 study visit to baseline of the NGLU-CL01-T study (observational period) in the following: clinical laboratory tests (haematology, serum chemistry, and urinalysis)and CSF findings (cell counts, glucose, and protein); 12-lead ECGs; vital signs; physical examination and use of concomitant medications/therapies. • Change in neurocognitive and developmental function during the NGLU-CL01-T study (on treatment) • Change in neurocognitive and developmental function from the NGLU-CL01 study visit to baseline of the NGLU-CL01-T study (observational period) • Changes in brain structure • Changes in BBB integrity during the NGLU-CL01-T study (on treatment) • Changes in BBB integrity from the NGLU-CL01 study visit to baseline of the NGLU-CL01-T study (observational period) | — |
Countries
United Kingdom
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed