A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS - ND

• Register: EUCTR
• Last refreshed on: 16 June 2014
• Main ID: EUCTR2009-013459-31-IT
• Date of registration: 15/01/2010
• Prospective Registration: Yes
• Primary sponsor: AMICUS THERAPEUTICS, INC
• Public title: A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS - ND
• Scientific title: A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS - ND
• Date of first enrolment: 02/02/2010
• Target sample size: 60
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-013459-31
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no

Countries of recruitment

Belgium, Denmark, France, Germany, Italy, Netherlands, Spain, United Kingdom

Key inclusion & exclusion criteria

Inclusion criteria:
Male or female between the ages of 16 and 74 inclusive, diagnosed with Fabry disease Note: subjects under the age of 18 will be enrolled only at sites with all required regulatory and ethics approvals to do so. 2. Confirmed GLA mutation that has been shown to be responsive to AT1001 in vitro (as listed on pharmacogenetic reference table [Appendix A]) 3. Na?ve to ERT or has not received ERT for 6 consecutive months or longer before the screening visit for the study 4. Urine GL-3 greater than four times the upper limit of normal at Screening 5. Subjects taking angiotensin converting enzyme inhibitors (ACEIs) or angiotensin receptor blockers (ARBs) must be on a stable dose for a minimum of 4 weeks before the baseline visit 6. Male and female subjects of childbearing potential agree to use medically accepted methods of contraception during study and for 30 days after study completion 7. Subject is willing and able to provide written informed consent, and assent if applicable
Are the trial subjects under 18? yes
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
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Subject has undergone or is scheduled to undergo kidney transplantation, or is currently on dialysis 2. eGFR < 30 mL/min/1.73m2 (CKD Stage 4 or 5) based on MDRD equation 3. QTc ≥ 450 msec for males or ≥ 470 msec for females 4. Pregnant or breast-feeding 5. History of allergy or sensitivity to study medication (including excipients) or other iminosugars (e.g., miglustat, miglitol) 6. Subject is treated or has been treated with any investigational drug within 30 days of the screening visit 7. Subject is currently treated or has ever been treated with AT1001 8. Any intercurrent condition or concomitant medication use considered to be an absolute contraindication to kidney biopsy or that may preclude accurate interpretation of study data 9. Otherwise unsuitable for the study, in the opinion of the Investigator.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Fabry disease
MedDRA version: 9.1 Level: LLT Classification code 10016016

Intervention(s)

Pharmaceutical Form: Capsule, hard
INN or Proposed INN: migalastat hydrochloride
CAS Number: 75172-81-5
Current Sponsor code: AT1001
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: The primary objective of this study is to compare the effect of AT1001 versus placebo on kidney GL-3 as assessed by histological scoring of the number of inclusions in interstitial capillaries after 6 months of treatment.

Primary end point(s): Kidney GL-3 (interstitial capillary histology): The average number of GL-3 inclusions per kidney interstitial capillary is assessed by a quantitative histological method used to count GL-3 inclusions in a sample of interstitial capillaries at baseline and at Month 6.

Secondary Objective: The secondary objectives of Stage 1 of this study are:  To compare the effect of AT1001 versus placebo on urine GL-3 levels as measured by LC-MS/MS  To compare the effect of AT1001 versus placebo on renal function (iohexol GFR, eGFR, 24-hour urine protein)  To compare the safety and tolerability of AT1001 versus placebo

Secondary ID(s)

AT1001-011

2009-013459-31-GB