Drug-drug interaction study between AT2220 and ERT

Conditions: Pompe Disease
MedDRA version: 14.1Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Registration Number: EUCTR2011-002154-32-GB

Lead Sponsor: Amicus Therapeutics, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 22

Inclusion Criteria

1. Male or female, diagnosed with Pompe disease and between 18 and 65 years of age, inclusive
2. Subject has been on a stable regimen and dose of alglucosidase alfa for at least 3 months before screening (stable regimen defined as currently receiving alglucosidase alfa every 2 weeks and stable dose defined as not varying by more than ± 10%)
3. Subject has an estimated glomerular filtration rate (eGFR) = 50 mL/min at Screening; eGFR to be estimated using the 4-parameter Modification of Diet in Renal Disease (MDRD) equation:
eGFR (mL/min/1.73 m^2) = 175 x (Scr)^(-1.154) x (Age)^(-0.203) x (0.742 if female) x (1.212 if African-American)
4. Male and female subjects of childbearing potential agree to use medically accepted methods of contraception during the study and for 30 days after study drug administration
5. Subject is willing and able to provide written informed consent and is able to comply with all study procedures
Are the trial subjects under 18? no
Number of subjects for this age range:
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

1. Subject has had a documented transient ischemic attack, stroke, unstable angina or myocardial infarction within the 3 months before Screening
2. Subject has clinically significant unstable cardiac disease (e.g., cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina or NYHA class III or IV congestive heart failure)
3. Subject requiring invasive mechanical ventilation or is confined to a wheelchair
4. Subject has a history of allergy or sensitivity to study drug (including excipients) or other iminosugars (e.g., miglustat, miglitol)
5. Subject is pregnant or breastfeeding
6. Subject tests positive for hepatitis B surface antigen or hepatitis C antibody
7. Subject has received any investigational/experimental drug or device within 30 days of Screening
8. Subject has any concurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study