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Main
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Note: This record shows only 22 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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EUCTR |
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Last refreshed on:
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25 November 2019 |
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Main ID: |
EUCTR2007-001838-13-GB |
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Date of registration:
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16/05/2007 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Long-term safety study of AT1001 in people with Fabry disease
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Scientific title:
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Open Label Extension Study to Evaluate the Long-term Safety Tolerability and Pharmacodynamics of AT1001 in Patients with Fabry Disease - Extension Study of AT1001 in Fabry Disease |
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Date of first enrolment:
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05/05/2009 |
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Target sample size:
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30 |
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Recruitment status: |
Not Recruiting |
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URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-001838-13 |
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Study type:
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Interventional clinical trial of medicinal product |
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Study design:
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Controlled: no
Randomised: no
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product:
Placebo:
Other:
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): yes
Therapeutic confirmatory - (Phase III):
Therapeutic use (Phase IV):
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Countries of recruitment
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Australia
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Brazil
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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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Patient Advocacy
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Address:
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30 A Upper high Street
OX9 3EX
Thame Oxon
United Kingdom |
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Telephone:
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001 609 662-2000 |
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Email:
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clinicaltrials@amicustherapeutics.com |
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Affiliation:
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Amicus Therapeutics |
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Name:
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Patient Advocacy
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Address:
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30 A Upper high Street
OX9 3EX
Thame Oxon
United Kingdom |
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Telephone:
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001 609 662-2000 |
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Email:
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clinicaltrials@amicustherapeutics.com |
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Affiliation:
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Amicus Therapeutics |
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Key inclusion & exclusion criteria
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Inclusion criteria:
1.Subject completed the main treatment period of another Phase 2 trial of AT1001 in Fabry disease
2.Women of childbearing potential must have a negative result on their pregnancy test
3.Male and female subjects agree to use reliable methods of contraception during study treatment and for 4 weeks after study treatment termination
4.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
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Exclusion criteria:
1. Subject had a major protocol violation in the preceding AT1001 trial and was discontinued.
2.Subject has undergone, or is scheduled to undergo kidney transplantation or is currently on dialysis
3.Subject is treated or has been treated with another investigational drug (except AT1001) within 30 days of study start
4.Subject has been treated with Fabrazyme (agalsidase beta), Replagal (agalsidase alfa), Glyset (miglitol) or Zavesca(miglustat) within 2 weeks prior to enrollment
Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
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Health Condition(s) or Problem(s) studied
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Fabry Disease
MedDRA version: 14.1
Level: PT
Classification code 10016016
Term: Fabry's disease
System Organ Class: 10010331 - Congenital, familial and genetic disorders
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Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
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Intervention(s)
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Product Name: migalastat hydrochloride
Product Code: AT1001
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: migalastat hydrochloride
CAS Number: 75172-81-5
Current Sponsor code: AT1001
Other descriptive name: 1-deoxygalactonojirimycin hydrochloride
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Product Name: Reminder capsules
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Not applicable
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0-
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Primary Outcome(s)
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Secondary Objective: To gain information about the pharmacodynamics and pharmacokinetics of orally administered AT1001 in patients with Fabry disease
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Timepoint(s) of evaluation of this end point: End of study visit
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Primary end point(s): •Treatment emergent marked laboratory abnormalities up to end of study (EOS) visit
•Treatment emergent adverse events up to 48 hours after study medication discontinuation
•Serious adverse events up to 28 days after study medication discontinuation
•Change in concomitant treatments
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Main Objective: To evaluate the long-term safety and tolerability of oral AT1001 in patients with Fabry disease
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Secondary Outcome(s)
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Secondary end point(s): Levels of a-Gal A in leukocytes
Levels of GL-3 in plasma and
Levels of AT1001 in plasma
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Timepoint(s) of evaluation of this end point: Monitored throughout study
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Secondary ID(s)
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FAB-CL-205
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Source(s) of Monetary Support
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Amicus Therapeutics
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Ethics review
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Status: Approved
Approval date:
Contact:
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