A Phase 1/2 open label study in MPS IIIB subjects to investigate the long term safety and effect of SBC-103 given by IV infusion.

• Register: EUCTR
• Last refreshed on: 16 January 2017
• Main ID: EUCTR2015-000753-20-NL
• Date of registration: 24/08/2015
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals, Inc.
• Public title: A Phase 1/2 open label study in MPS IIIB subjects to investigate the long term safety and effect of SBC-103 given by IV infusion.
• Scientific title: A PHASE I/II OPEN LABEL STUDY IN MPS IIIB SUBJECTS TO INVESTIGATE THE SAFETY, BIODISTRIBUTION, PHARMACOKINETICS, AND PHARMACODYNAMICS/EFFICACY OF SBC-103 ADMINISTERED INTRAVENOUSLY
• Date of first enrolment: 16/08/2016
• Target sample size: 4
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-000753-20
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Netherlands

Contacts

• Name: Loralie Brennen, Assoc Dir Clin Ops
• Affiliation: Alexion Pharmaceuticals, Inc.

• Name: Loralie Brennen, Assoc Dir Clin Ops
• Affiliation: Alexion Pharmaceuticals, Inc.

Key inclusion & exclusion 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:
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



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

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

Intervention(s)

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
Current Sponsor code: SBC-103
Other descriptive name: rhNAGLU
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2-

Product Name: 89 Zirconium Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) (radiolabeled)
Product Code: 89Zr-SBC-103
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: not available
CAS Number: 37288-40-7
Current Sponsor code: SBC-103 radiolabelled
Other descriptive name: rhNAGLU radiolabelled
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2-

Primary Outcome(s)

Main Objective: To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS IIIB, Sanfilippo B.

Primary end point(s): •Assessment of anti-drug antibodies (ADAs) (positive/negative)

Secondary Objective: PK, biodistribution and activity

Timepoint(s) of evaluation of this end point: Trial duration

Secondary Outcome(s)

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

Secondary ID(s)

NGLU-CL06

Source(s) of Monetary Support

Alexion Pharmaceuticals, Inc.