Neo1 - TDR12857
Research type
Research Study
Full title
An open-label, multicenter, multinational, ascending dose study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of repeated biweekly infusions of neoGAA in naïve and alglucosidase alfa treated late-onset Pompe disease patients.
IRAS ID
131372
Contact name
Volker Straub
Contact email
Sponsor organisation
Genzyme Corporation
Eudract number
2012-004167-42
ISRCTN Number
N/A
Clinicaltrials.gov Identifier
N/A
Research summary
This is an open-label, multicentre, multinational, ascending dose study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of repeated bi-weekly infusions of NeoGAA in naïve and algucosidase alfa (AA) treated late-onset Pompe disease patients.
The objectives of the study are to determine the safety and tolerability, pharmacokinetic & pharmacodynamics effects of NeoGAA on skeletal muscle and other exploratory biomarkers & efficacy endpoints.
Pompe is a rare, genetic disease cause by deficiency of acid alpha-glucosidase (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.
Currently, AA 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 than the existing standard of care. NeoGAA differs from GAA by having additional IMP residues on the surface which is intended to increase the uptake of GAA into the cells, thus helping in glycogen clearance.
The information gathered from this study is intended to be used in the further development of NeoGAA and improved treatments.
To be eligible for the study, patients would need to be aged 18 or over, with confirmed Pompe disease and their disease should not be in the later stages of progression.
The study will recruit a total of 21 subjects, with participating (hospital) sites in Europe and the USA. Patients will have 17 study visits over a 6 month period, at approximately 2 weekly intervals when they will be given NeoGAA treatment by infusion.REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
13/NE/0169
Date of REC Opinion
22 Jul 2013
REC opinion
Further Information Favourable Opinion