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 (CL01-T)
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|ClinicalTrials.gov Identifier: NCT02618512|
Recruitment Status : Terminated (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.)
First Posted : December 1, 2015
Results First Posted : April 18, 2018
Last Update Posted : April 18, 2018
|Condition or disease||Intervention/treatment||Phase|
|Mucopolysaccharidosis III, Type B (MPS IIIB) Sanfilippo B||Drug: SBC-103||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||October 15, 2015|
|Actual Primary Completion Date :||August 18, 2017|
|Actual Study Completion Date :||August 18, 2017|
Patients were administered 1 mg/kg by IV infusion once every other week (qow) for at least 12 weeks. After evaluation of 12-week safety, tolerability, and pharmacodynamic data in individual patients, the dose was increased to 3 mg/kg qow. Infusions were to be at least 10 days apart and were administered every 14 days ±5 days.
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.
- Safety and Tolerability of SBC-103 [ Time Frame: Planned duration was baseline to 164 weeks but due to early termination of the study, actual is 96 weeks. ]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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02618512
|Birmingham, United Kingdom, B46NH|