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NCT02618512 Clinical Trial

A 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

Mucopolysaccharidosis III, Type B (MPS IIIB) Clinical Trial

— CL01-T  

Official 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

Clinical Trial Details — Status: Terminated

Administrative data
NCT number NCT02618512
Other study ID # NGLU-CL01-T
Secondary ID 2015-001983-20
Status Terminated
Phase Phase 1/Phase 2
First received October 21, 2015
Last updated March 19, 2018
Start date October 15, 2015
Est. completion date August 18, 2017

Study information
Verified date March 2018
Source Alexion Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This study evaluated the safety and tolerability of intravenous (IV) administration of SBC-103 in previously studied, SBC-103 treatment naïve patients with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) who participated in the NGLU-CL01 study. The NGLU-CL01 study was a non-interventional study that evaluated structural brain abnormalities and blood brain barrier (BBB) integrity by magnetic resonance imaging (MRI) and cerebrospinal fluid/serum albumin index.

Recruitment information / eligibility
Status Terminated
Enrollment 3
Est. completion date August 18, 2017
Est. primary completion date August 18, 2017
Accepts healthy volunteers No
Gender All
Age group 5 Years and older
Eligibility Participants from NGU-CL01 study were enrolled into the NGLU-CL01-T study. Subjects who met all of the inclusion criteria and none of the exclusion criteria were eligible to participate in this study.

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• SBC-103
SBC-103 is a recombinant human alpha-N-acetylglucosaminidase (rhNAGLU). Patients were started with low dose of SBC-103 for 12 weeks and then will escalate to a higher dose.

Locations
Country Name City State
n/a

Sponsors (1)
Lead Sponsor Collaborator
Alexion Pharmaceuticals

Country where clinical trial is conducted

United Kingdom, 

Outcome
Type Measure Description Time frame Safety issue
Primary Safety and Tolerability of SBC-103 The planned primary endpoint of this study was safety and tolerability of SBC-103 in patients with MPS IIIB, as measured by Number of participants with treatment-emergent adverse events, including serious adverse events; infusion-associated reactions; incidence of antidrug antibodies, clinical laboratory tests, cerebrospinal fluid findings, vital signs, and prior and concomitant medications Planned duration was baseline to 164 weeks but due to early termination of the study, actual is 96 weeks.