Phase 3 N=60 Randomized Treatment

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

Fabry Disease

Bottom Line

View on ClinicalTrials.gov: NCT01218659 ↗

Enrolled (actual)

Serious AEs

27.8%

Results posted

Nov 2018

Primary outcome: Primary: Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate — -4.35; -3.24 mL/min/1.73 m^2/year

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: migalastat hydrochloride (Drug); agalsidase (Biological)
Age: Pediatric, Adult, Older Adult · 16+ yrs
Sex: All
Sponsor: Amicus Therapeutics
Primary completion: May 2014

Outcome Measures

Outcome	Result	p-value
PRIMARY Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate	-4.35; -3.24	—
PRIMARY Annualized Rate Of Change From Baseline To Month 18 In eGFR	-0.40; -1.03	—
SECONDARY Annualized Rate Of Change From Baseline To Month 18 In eGFR By The Modification Of Diet In Renal Disease Equation	-1.51; -1.53	—

Summary

Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A) mutation that is amenable to migalastat, based on the clinical trial human embryonic kidney cell (HEK) assay.

Eligibility Criteria

Inclusion Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01218659). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.