Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT01476163




Registration number
NCT01476163
Ethics application status
Date submitted
17/11/2011
Date registered
22/11/2011
Date last updated
23/01/2019

Titles & IDs
Public title
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
Scientific title
Physician Initiated Expanded Access Request for Treatment Use of Migalastat Hydrochloride (AT1001), an Investigational Treatment for Individual Patients With Fabry Disease (AT1001-188)
Secondary ID [1] 0 0
AT1001-188
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Expanded Access
Description of intervention(s) / exposure
Treatment: Drugs - migalastat HCl

Treatment: Drugs: migalastat HCl
150 mg capsule taken every other day by mouth. An inactive reminder capsule may be provided to take on the days in between migalastat HCl
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes

Eligibility
Key inclusion criteria
- Confirmed GLA mutation predicted to be responsive migalastat in the human embryonic
kidney (HEK-293) cell-based assay

- 16-74 years of age

- Strong clinical indication for treatment of Fabry disease

- No other treatment option including either unsuitable for ERT or unable to access ERT

- Appropriate female and male contraception

- Willing to receive treatment with migalastat HCl via this program including having
signed an authorization for sharing clinical data
Minimum age
16 Years
Maximum age
74 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Estimated glomerular filtration rate (eGFR) or GFR <30 mL/minute

- Scheduled for renal or other organ transplant or replacement therapy

- Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy
FABRAZYME® (agalsidase beta) or REPLAGAL™ (agalsidase alpha)

- Contraindication to migalastat, i.e., sensitivity to other iminosugar such as
miglustat, miglitol

- Treated with another investigational drug within 30 days of start of migalastat HCl
treatment

- Unable to comply with study requirements or deemed otherwise unsuitable for study
entry in the opinion of the investigator.

Study design
Purpose of the study
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Approved for marketing
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Maryland
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
New York

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Amicus Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This program allows physicians to request permission from Amicus Therapeutics (Amicus) for
treatment access to migalastat hydrochloride (HCl) for specific adult patients with Fabry
disease. Treatment is open label for 6 months with renewal every 6 months.
Trial website
https://clinicaltrials.gov/ct2/show/NCT01476163
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Monitor, Clinical Research
Address 0 0
Amicus Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT01476163