Clinical Trial: NeoGAA Extension Study

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of NeoGAA In Patients With Pompe Disease

Brief Summary:

Primary Objective:

Long-term safety and pharmacokinetics (PK) of neoGAA

Secondary Objective:

Long-term effect of neo-GAA on pharmacodynamic and exploratory efficacy variables


Detailed Summary: The duration of the study will be 6 years from the date the first patient enters the study. Each patient will continue with the study until the patient withdraws, the Investigator withdraws the patient, or the Sponsor terminates the study.
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome:

  • Assessment of adverse events (AEs) and treatment-emergent adverse events (TEAEs), including infusion-associated reactions (IARs) and deaths [ Time Frame: screening/baseline until year 6 ]
  • Laboratory assessments including hematology, biochemistry and urinalysis [ Time Frame: Monthly, from baseline until year 6 ]
  • Vital signs [ Time Frame: screening/baseline until year 6 ]


Original Primary Outcome:

  • Assessment of adverse events (AEs) and treatment-emergent adverse events (TEAEs) [ Time Frame: screening/baseline until year 6 ]
  • Laboratory assessments including hematology, biochemistry and urinalysis [ Time Frame: Monthly, from baseline until year 6 ]
  • Vital signs [ Time Frame: screening/baseline until year 6 ]


Current Secondary Outcome:

  • Electrocardiogram [ Time Frame: every 6 months, from baseline until year 6 ]
  • anti-neoGAA immunoglobulin G (IgG) antibodies, and neutralizing antibody formation in IgG seropositive patients; anti-alglucosidase alfa IgG antibodies [ Time Frame: monthly, from baseline up to 6 months, then every 3 months until year 6; every 6 months from baseline until year 6 ]
  • Cmax [ Time Frame: at 6 months, then yearly until year 6 ]
  • AUC [ Time Frame: at 6 months, then yearly until year 6 ]
  • t1/2 [ Time Frame: at 6 months, then yearly until year 6 ]
  • Skeletal muscle glycogen content [ Time Frame: every 2 years, from baseline until year 6 ]
  • Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments [ Time Frame: every 2 years, from baseline until year 6 ]
  • Urinary Hex4 [ Time Frame: every 6 months, from baseline until year 6 ]
  • plasma analyses of circulating mRNA and micro RNA [ Time Frame: every 6 months, from baseline until year 6 ]
  • serum analyses of skeletal muscle RNA expression [ Time Frame: every 2 years, from baseline until year 6 ]


Original Secondary Outcome:

  • Electrocardiogram [ Time Frame: every 6 months, from baseline until year 6 ]
  • Immunogenicity assessments (anti-neoGAA immunoglobulin G (IgG) antibodies, neutralizing antibody formation in IgG seropositive patients, and anti-alglucosidase alfa IgG antibodies) [ Time Frame: every 6 months, from baseline until year 6 ]
  • Cmax [ Time Frame: at 6 months, then yearly until year 6 ]
  • AUC [ Time Frame: at 6 months, then yearly until year 6 ]
  • t1/2 [ Time Frame: at 6 months, then yearly until year 6 ]
  • Skeletal muscle glycogen content [ Time Frame: every 2 years, from baseline until year 6 ]
  • Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments [ Time Frame: every 2 years, from baseline until year 6 ]
  • Urinary Hex4 [ Time Frame: every 6 months, from baseline until year 6 ]
  • plasma analyses of circulating mRNA and micro RNA [ Time Frame: every 6 months, from baseline until year 6 ]
  • serum analyses of skeletal muscle RNA expression [ Time Frame: every 2 years, from baseline until year 6 ]


Information By: Sanofi

Dates:
Date Received: December 4, 2013
Date Started: February 2014
Date Completion: May 2020
Last Updated: March 31, 2017
Last Verified: March 2017