Pompe disease MedDRA version: 20.0 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 100000004850
Conditions
Interventions
Product Name: duvoglustat hydrochloride
Product Code: AT2220
Pharmaceutical Form: Solution for injection
INN or Proposed INN: duvoglustat HCl
Sponsors
Amicus Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Male or female, diagnosed with Pompe disease and between 16 and 65 years of age, inclusive (where required by local authorities, the minimum age will be 18 years of age) 2.Subject has never been treated with rhGAA (rhGAA-naïve) OR Subject has been treated with Lumizyme (rhGAA-experienced) for at least 12 months, AND for at least the 12 weeks before the Screening Visit, has been consistently treated with Lumizyme as per its local commercial labeling (or as per local regulations, with prior approval of the sponsor), including being on a stable dose, with a stable infusion duration (between 3 and 5 hours) and biweekly schedule (stable defined as not varying by more than ± 10%) For subjects who fall outside of these criteria, entry into the clinical trial is at the sponsor’s discretion 3.Subject, whether male or female, agrees to use medically accepted methods of contraception during the study and for 30 days after their last administration of duvoglustat (unless subject is not of childbearing potential) 4.Subject is willing and able to provide written informed consent and, as applicable, assent. Subjects under 18 years of age will provide written informed assent, and written informed consent will be provided by their parent or legal guardian 5.Subject is able to comply with all study procedures Are the trial subjects under 18? yes Number of subjects for this age range: 2 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 21 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: 1.Subject has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 12 weeks before the Screening Visit 2.Subject has clinically significant unstable cardiac disease (ie, any cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or NYHA class III or IV congestive heart failure) 3.Subject requires any invasive mechanical ventilation 4.Subject is restricted to a wheelchair or is otherwise not fully ambulatory; subjects who require wheelchair assistance or walkers under special circumstances may be eligible at the discretion of the sponsor 5.Subject has a history of allergy or sensitivity to study drug (including excipients) or other iminosugars (eg, miglitol, miglustat) 6.Subject is pregnant or breastfeeding 7.Subject has a positive medical history for hepatitis A, B, or C, or HIV (human immunodeficiency virus) 8.Subject has received any investigational/experimental device within the 30 days before the Screening Visit 9.Subject is taking, or has taken at any time in the 12 weeks before the Screening Visit, a prohibited medication [miglitol (eg, Glyset); acarbose (eg, Precose, Glucobay); voglibose (eg, Volix, Vocarb, Volibo); oseltamivir (eg, Tamiflu); dietary supplements containing mulberry extract; chloroquine; any investigational/experimental drug] 10.Subject is taking, or has taken at any time in the 26 weeks before the Screening Visit, the prohibited medication miglustat (eg, Zavesca) 11.Subject is taking, or has taken at any time in the 12 months before the Screening Visit, any immunosuppressant drug (eg, cyclosporins, azathioprines, methotrexate, monoclonal antibodies that block the immune system, and long-term corticosteroids). Subjects who require corticosteroids for acute management of infusion reactions may be allowed with sponsor approval 12.Subject has any concurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the subject may have an unacceptable risk by participating in this study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of intravenous (IV) duvoglustat administered immediately before and at point(s) during and after IV infusion of recombinant human acid a glucosidase (rhGAA); To determine the optimal dose and administration regimen of IV duvoglustat based on plasma, urine, and muscle pharmacokinetics (PK) of IV duvoglustat ;Secondary Objective: To evaluate the effects of IV duvoglustat on plasma rhGAA activity ; Primary end point(s): For the primary study objective of evaluating the safety of IV duvoglustat the parameters are as follows: adverse events (AEs), clinical laboratory evaluations (serum chemistry, hematology, and urinalysis), concomitant medications, ECGs, physical examination, weight, vital signs, 6MWT, and FVC For the primary study objective of determining the optimal dose and administration regimen of IV duvoglustat, it will be based on plasma, urine, and muscle PK of IV duvoglustat. ;Timepoint(s) of evaluation of this end point: Throughout the study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): For the secondary study objective of evaluating the effects of IV duvoglustat on plasma rhGAA activity, the parameters will be as specified in the SAP.;Timepoint(s) of evaluation of this end point: Throughout the study. | — |
Countries
Netherlands, United Kingdom, United States
Contacts
Public ContactPatient Advocacy
Amicus Therapeutics, Inc.
Outcome results
None listed