Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01218659
• Date of registration: 06/10/2010
• Prospective Registration: Yes
• Primary sponsor: Amicus Therapeutics
• Public title: Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
• Scientific title: A Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT
• Date of first enrolment: September 8, 2011
• Target sample size: 68
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01218659
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Austria, Belgium, Brazil, Denmark, France, Germany, Greece, Italy, Japan, Poland, Switzerland, Taiwan, Turkey, United Kingdom, United States

Contacts

• Name: Medical Monitor, Clinical Research
• Affiliation: Amicus Therapeutics

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female between the ages of 16 and 74 diagnosed with Fabry disease

- Confirmed a Gal-A mutation that is amenable to migalastat, based on the clinical trial
HEK assay

- Participant has been on ERT for at least 12 months before screening/baseline

- Dose level and regimen of ERT have been stable for 3 months before screening/baseline
and is at least 80% of the currently labeled dose and regimen for this time period

- Glomerular filtration rate (GFR) = 30 milliliter (mL)/minute (min) /1.73 m^2

- Participants taking angiotensin converting enzyme inhibitors or angiotensin receptor
blockers must be on a stable dose for at least 4 weeks before screening/baseline

- Women who can become pregnant and all men agree to be sexually abstinent or use
medically accepted methods of birth control throughout the duration of the study and
for up to 30 days after last dose of study medication

- Participant is willing and able to provide written informed consent and assent if
applicable

Exclusion Criteria:

- Participant has undergone, or is scheduled to undergo, kidney transplantation or any
other solid organ transplantation

- Participant is on regular dialysis that is specifically for the treatment of chronic
kidney disease

- Participant has had a documented transient ischemic attack, stroke, unstable angina,
or myocardial infarction within the 3 months before screening/baseline

- Participant has clinically significant unstable cardiac disease in the opinion of the
investigator (for example, cardiac disease requiring active management, such as
symptomatic arrhythmia, unstable angina, or New York Heart Association (NYHA) class
III or IV congestive heart failure)

- Pregnant or breast-feeding

- History of allergy or sensitivity to study medication (including excipients) or other
iminosugars (for example, miglustat, miglitol)

- Participant has absolute contraindication to iohexol and/or inability to undergo
iohexol GFR testing

- Participant requires treatment with Glyset® (miglitol), or Zavesca® (miglustat)

- Participant received any investigational/experimental drug, biologic or device within
30 days of screening/baseline

- Any intercurrent illness or condition that may preclude the participant from
fulfilling the study requirements or suggests to the investigator that the participant
may have an unacceptable risk by participating in this study



Age minimum: 16 Years
Age maximum: 74 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Biological: agalsidase

Drug: migalastat hydrochloride

Primary Outcome(s)

Annualized Rate Of Change From Baseline To Month 18 In eGFR [Time Frame: Baseline to Month 18]

Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate [Time Frame: Baseline to Month 18]

Secondary Outcome(s)

Annualized Rate Of Change From Baseline To Month 18 In eGFR By The Modification Of Diet In Renal Disease Equation [Time Frame: Baseline to Month 18]

Secondary ID(s)

ATTRACT

2010-022636-37

AT1001-012

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 01/11/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01218659