AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE

Amicus Therapeutics, Inc.0 个研究点目标入组 24 人开始时间: 2011年8月10日最近更新: 2017年8月21日

适应症

试验速览

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•1. Male, diagnosed with Fabry disease and between 18 and 65 years of age, inclusive :
•2. Body Mass Index (BMI) between 18-35
•3. Subject initiated treatment with agalsidase at least 1 month, prior to screening and has received at least two infusions, before Screening Visit
•4. Subject’s dose level, dosing regimen and form (i.e., alfa or beta) of agalsidase have been stable (stable dose defined as not varying by more than ± 20%) for at least 1 month before Screening Visit
•5. Subject has a estimated creatinine clearance = 50 mL/min at Screening; creatinine clearance to be estimated using the 4-parameter MDRD equation:
•eGFR (mL/min/1.73 m2) = 186 x (Scr)-1.154 x (Age)-0.203 x (0.742 if female) x (1.212 if African-American)
•6. Subject agrees to use medically accepted methods of contraception during the study and for 30 days after study completion
•7. Subject is willing and able to provide written informed consent
•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 24
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. Subject has had a documented transient ischemic attack, ischemic 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 has a history of allergy or sensitivity to study drug (including excipients) or other iminosugars (e.g., miglustat, miglitol)
•4. Subject requires a concomitant medication prohibited by the protocol: Glyset® (miglitol), or Zavesca® (miglustat)
•5. Any investigational/experimental drug or device within 30 days of Screening, except for the use of investigational Enzyme Replacement Therapy for Fabry Disease.
•6. Protocol Amendment 4.0, version date November 2, 2011, eliminates Exclusion Criterion 6. The numerical reference of the other Exclusion Criteria will remain as before in prior versions of the protocol.
•7. 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

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