TerminatedPhase 1/2

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

Stopped: The Sponsor's decision to terminate the SBC-103 program was reached after review of the data from all interventional clinical studies of SBC-103.

United Kingdom3 participantsStarted 2015-10-15

Plain-language 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.

Who can participate

Age range5 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

Safety and Tolerability of SBC-103

Timeframe: Planned duration was baseline to 164 weeks but due to early termination of the study, actual is 96 weeks.

Trial details

NCT IDNCT02618512

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-08-18

Results submitted2018-02-16

View on ClinicalTrials.gov More Mucopolysaccharidosis III, Type B (MPS IIIB) trials