Mini-COMET
Research type
Research Study
Full title
An open-label ascending dose cohort study to assess the safety, pharmacokinetics, and preliminary efficacy of neoGAA (GZ402666) in patients with infantile-onset Pompe disease treated with alglucosidase alfa who demonstrate clinical decline or sub-optimal clinical response.
IRAS ID
213231
Contact name
Carlo Incerti
Contact email
Sponsor organisation
Sanofi Genzyme
Eudract number
2016-003475-21
Clinicaltrials.gov Identifier
U1111-1179-4616, WHO universal trial number
Duration of Study in the UK
5 years, 0 months, 30 days
Research summary
Pompe is a rare, genetic disease caused by a deficiency of the enzyme acid alphaglucosidase (GAA) which breaks down glycogen. The glycogen that accumulates in body tissues, especially cardiac and respiratory muscles as well as other major skeletal muscles, leads to muscle weakness, loss of respiratory function and often premature death.
Alglucosidase Alfa (AA), a longterm enzyme replacement therapy (ERT), is a licensed product used worldwide in patients with Pompe disease. NeoGAA is a second generation ERT for Pompe disease, that in preclinical studies, is more potent than the existing standard of care. NeoGAA differs from GAA by having additional bisM6P residues on the
surface which is intended to increase the uptake of GAA into the cells, particularly skeletal muscle cells, thus helping in the clearance of glycogen.This is an open-label ascending dose cohort study to assess the safety, pharmacokinetics, and preliminary efficacy of neoGAA (GZ402666) in patients with infantile-onset Pompe disease treated with alglucosidase alfa who demonstrate clinical decline or sub-optimal clinical response.
Primary objective is evaluate the safety profile of neoGAA in patients with infantile-onset Pompe disease (IOPD) previously treated with alglucosidase alfa.
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
17/NW/0209
Date of REC Opinion
17 May 2017
REC opinion
Further Information Favourable Opinion