MSC12790 (PAPAYA)
Research type
Research Study
Full title
A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients with Pompe Disease
IRAS ID
228074
Contact name
Carlo Incerti
Contact email
Sponsor organisation
Genzyme, A Sanofi Company
Eudract number
2010-022231-11
Clinicaltrials.gov Identifier
2010-022231-11, EudraCT
Duration of Study in the UK
0 years, 7 months, 16 days
Research summary
This is a prospective, open-label, multicenter study of patients with Pompe disease who are naïve to treatment with alglucosidase alfa or who have been previously treated with alglucosidase alfa for at least 6 months. The
patient or the patient’s legal guardian(s) must provide informed consent prior to performing any protocol-related procedure. Eligible patients will receive one intravenous (IV) infusion of alglucosidase alfa of 20 mg/kg of body weight.Pharmacokinetic and safety assessments will be performed at the scheduled treatment visit. Adverse events (AE) and concomitant medications/therapies will be monitored continuously throughout the study. Patients who are withdrawn from the study will not be replaced (ie, a patient’s study number will not be reused); however, additional patients may be enrolled to offset patient dropouts.
An independent Data and Safety Monitoring Board (DSMB) will perform ad hoc reviews of safety data as outlined in the DSMB Charter, which is maintained separately from the study protocol. An immunologist will be consulted, when necessary, to review information and provide treatment recommendations for infusion-associated reactions (IARs).
REC name
London - Central Research Ethics Committee
REC reference
17/LO/1421
Date of REC Opinion
6 Nov 2017
REC opinion
Further Information Favourable Opinion