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Main
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Note: This record shows only the 20 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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29 October 2012 |
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Main ID: |
NCT01380743 |
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Date of registration:
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23/06/2011 |
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Primary sponsor: |
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Public title:
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Drug-drug Interaction Study
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Scientific title:
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An Open-Label, Multi-Center, Study to Investigate Drug-Drug Interactions Between AT2220 (Duvoglustat Hydrochloride) and Alglucosidase Alfa in Patients With Pompe Disease |
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Date of first enrolment:
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October 2011 |
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Target sample size:
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22 |
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Recruitment status: |
Active, not recruiting |
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URL:
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http://clinicaltrials.gov/show/NCT01380743 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Countries of recruitment
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Canada
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France
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United Kingdom
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United States
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Contacts
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Name:
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Medical Monitor |
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Address:
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Telephone:
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Email:
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Affiliation:
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Study Sponsor |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Male or female, diagnosed with Pompe disease and between 18 and 65 years of age,
inclusive
- Subject has been on a stable regimen and dose of alglucosidase alfa or at least 3
month before screening (stable regimen defined as currently receiving alglucosidase
alfa every 2 weeks and stable dose defined as not varying by more than ± 10%)
- Subject has an estimated creatinine clearance = 50 mL/min at Screening; eGFR to be
estimated using the 4-parameter MDRD equation:
eGFR (mL/min/1.73 m2) = 175 x (Scr)-1.154 x (Age)-0.203 x (0.742 if female) x (1.212 if
African-American)
- Male and female subjects of childbearing potential agree to use medically accepted
methods of contraception during the study and for 30 days after study completion
- Subject is willing and able to provide written informed consent and is able to comply
with all study procedures
Exclusion Criteria:
- Subject has had a documented transient ischemic attack, ischemic stroke, unstable
angina or myocardial infarction within the 3 months before Screening
- 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)
- Subject requiring mechanical ventilation or is confined to a wheelchair
- Subject has a history of allergy or sensitivity to study drug (including excipients)
or other iminosugars (e.g., miglustat, miglitol)
- Subject is pregnant or breastfeeding
- Subject tests positive for hepatitis B surface antigen or hepatitis C antibody
- Subject has received any investigational/experimental drug or device within 30 days
of Screening
- Subject has any intercurrent 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
Age minimum:
18 Years
Age maximum:
65 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Pompe Disease
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Intervention(s)
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Drug: duvoglustat hydrochloride
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Primary Outcome(s)
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Pharmacokinetics
[Time Frame: 1 month]
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Safety
[Time Frame: 3 months]
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Secondary Outcome(s)
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AT2220 concentration in muscle
[Time Frame: Day 3 or Day 7]
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GAA activity in muscle
[Time Frame: Day 3 or Day 7]
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Secondary ID(s)
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AT2220-010
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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