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: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU)
Product Code: SBC-103
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: not available
CAS Number: 37288-40-7
Curre
Sponsors
Alexion Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Definitive diagnosis of MPS IIIB, 2. Greater than or equal to 2 years old at the time of written informed consent/assent, and has an age equivalent of = 1 year on the Vineland II. 3. Subject weight is = 30 kg. 4. Subject or subject’s parent or legal guardian (if applicable) consents 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. Are the trial subjects under 18? yes Number of subjects for this age range: 3 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 1 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Has any other medical condition or circumstance in which a PET/CT scan is contraindicated according to local institutional policy. 2. Prior exposure to radiation 3. Received treatment with gene therapy at any time, or any investigational drug, 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. 4. Has any internal or non-removable external metal items 5. Previous hematopoietic stem cell or bone marrow transplant. 8. A bleeding disorder, or any other medical condition or circumstance in which a lumbar puncture (for collection of CSF) is contraindicated 9. Known hypersensitivity to eggs. 10. Other medical conditions or co-morbidities
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS IIIB, Sanfilippo B.;Secondary Objective: PK, biodistribution and activity ;Primary end point(s): •Assessment of anti-drug antibodies (ADAs) (positive/negative) ;Timepoint(s) of evaluation of this end point: Trial duration | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): *PK parameters (serum, plasma, tissue) *Quantitative assessment of 89Zr-SBC-103 in the central nervous system (CNS) *Quantitative assessment of 89Zr-SBC-103 values in images of the major organs of the body (including liver, heart, and spleen) *Direct radiolabel counting and assessment of NAGLU enzyme activity *Relationship between the serum drug concentrations, unlabeled SBC-103, and drug concentrations of radiolabeled SBC-103 *Total HS in CSF, serum, and urine. *Neurocognitive and developmental function *Brain structure;Timepoint(s) of evaluation of this end point: Trial duration | — |
Countries
Netherlands
Contacts
Public ContactLoralie Brennen, Assoc Dir Clin Ops
Alexion Pharmaceuticals, Inc.
Outcome results
None listed