Clinical Trial: Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in MPS IIIB

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

Brief Summary: To evaluate the safety and tolerability of intravenous (IV) administration of SBC 103 in subjects with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) with evaluable signs or symptoms of developmental delay.

Detailed Summary:
Sponsor: Alexion Pharmaceuticals

Current Primary Outcome: Safety and Tolerability of IV SBC-103 as measured by Physical examination, vital signs, ECG, clinical laboratory tests, concomitant medication and development of anti-drug antibodies. [ Time Frame: Baseline to Week 156 ]

Original Primary Outcome:

  • Incidence of adverse events, serious adverse events, and infusion-associated reactions (IAR)s from baseline [ Time Frame: Part A: 24 Weeks; Part B: 128 Weeks and/or 30 days after last study drug infusion in Part B ]
  • Changes from baseline in clinical laboratory tests in serum, urine and cerebrospinal fluid [ Time Frame: Part A: 24 Weeks; Part B: 128 Weeks and/or 30 days after last study drug infusion in Part B ]
  • Changes from baseline in 12-lead electrocardiograms [ Time Frame: Part A: 24 Weeks; Part B: 128 Weeks and/or 30 days after last study drug infusion in Part B ]
  • Changes in vital signs during and post-infusion, relative to pre-infusion values [ Time Frame: Part A: 24 Weeks; Part B: 128 Weeks and/or 30 days after last study drug infusion in Part B ]
  • Changes from baseline in physical exam findings [ Time Frame: Part A: 24 Weeks; Part B: 128 Weeks and/or 30 days after last study drug infusion in Part B ]
  • Changes from baseline in the use of concomitant medications/therapies [ Time Frame: Part A: 24 Weeks; Part B: 128 Weeks and/or 30 days after last study drug infusion in Part B ]
  • Development of anti-drug antibodies since baseline [ Time Frame: Part A: 24 Weeks; Part B: 128 Weeks and/or 30 days after last study drug infusion in Part B ]


Current Secondary Outcome: PK/PD Effect of SBC-103 after single and multiple doses, neurocognitive and developmental function, brain structure volumetric assessment, and indices of microstructural integrity. [ Time Frame: Baseline to Week 156 ]

Original Secondary Outcome:

  • Change from baseline on heparan sulfate in cerebrospinal fluid, blood serum, and urine. [ Time Frame: Baseline to Week 24, Week 28, Week 40, Week 52, Week 66, Week 78, Week 90, Week 104, Week 118, Week 130, Week 142, Week 156 ]
  • The effect of SBC-103 on PK parameters [ Time Frame: Week 0, Week 12, Week 24, Week 52, Week 78, Week 104, Week 130, Week 156 ]
    • Serum maximum concentration (Cmax)
    • Time to maximum concentration (Tmax)
    • Area-under-the-concentration-time curve extrapolated to infinity (AUC∞)
    • Half-life (T1/2)
    • Clearance (Cl)
    • Apparent volume of distribution at steady state (Vss)
  • Change from baseline in neurocognitive and developmental function as determined by the scores on select developmental and behavioral assessment tools. [ Time Frame: Baseline to Week 24, Annually up to 3 years ]
  • Changes from baseline in brain structure as assessed by magnetic resonance imaging (MRI). [ Time Frame: Baseline to Week 24, Bi-annually up to 3 years ]


Information By: Alexion Pharmaceuticals

Dates:
Date Received: December 15, 2014
Date Started: December 2014
Date Completion:
Last Updated: August 5, 2016
Last Verified: August 2016