Status and phase
Conditions
Treatments
Study type
Funder types
Identifiers
About
Primary Objective:
To evaluate the safety and tolerability of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.
Secondary Objective:
To evaluate the pharmacokinetics, pharmacodynamics of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.
To evaluate the effect of neoGAA on exploratory efficacy endpoints in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.
Full description
Screening: within 90 days Period of treatment: 24 weeks (including 13 bi-weekly infusions) Post treatment evaluation visit: 2 weeks after last neoGAA infusion (at Week 27) End of study visit: 4 weeks after last neoGAA infusion (at Week 29) Total duration: approximately 41 weeks
Enrollment
Sex
Ages
Volunteers
Inclusion and exclusion criteria
Inclusion criteria :
For both Group 1 and Group 2:
Group 2 patients only:
Exclusion criteria:
For both Group 1 and Group 2:
Group 1 only:
Group 2 only:
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Primary purpose
Allocation
Interventional model
Masking
24 participants in 6 patient groups
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Data sourced from clinicaltrials.gov
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