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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01458119




Registration number
NCT01458119
Ethics application status
Date submitted
20/10/2011
Date registered
24/10/2011
Date last updated
2/10/2018

Titles & IDs
Public title
Open-Label Phase 3 Long-Term Safety Study of Migalastat
Scientific title
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
Secondary ID [1] 0 0
2011-004800-40
Secondary ID [2] 0 0
AT1001-041
Universal Trial Number (UTN)
Trial acronym
AT1001-041
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
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - migalastat hydrochloride

Experimental: Migalastat - Migalastat 150-mg capsule taken orally QOD. The median duration of exposure was 23.5 m.


Treatment: Drugs: migalastat hydrochloride
Oral capsule QOD

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) - An adverse event (AE) was defined as any untoward medical occurrence in a participant administered migalastat that did not necessarily have a causal relationship with the treatment. Each AE was recorded at the time of reporting; visits typically occurred every 6 months. A TEAE was defined as an AE starting on or after the first study drug administration date. Serious AEs were life-threatening or resulted in death, persistent or significant incapacitation, inpatient or prolonged hospitalization, or a congenital anomaly. The criteria for AE severity were: Mild: minimal discomfort, does not interfere with normal everyday activities; Moderate: sufficiently discomforting, interferes with normal everyday activities; Severe: prevents normal everyday activities. The number of participants experiencing TEAEs is presented for those who received migalastat treatment. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Timepoint [1] 0 0
Baseline to End of Follow-up (30 days after the end of this 42-month study), with AE reporting occurring at each study visit, which occurred once every 6 months.
Secondary outcome [1] 0 0
Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR) - The annualized rate of change of the eGFR was assessed per participant by the slope of the simple linear regression between the observed values and the assessment times. It was calculated by using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (eGFR [CKD-EPI]) and the Modification of Diet in Renal Disease (MDRD) equation (eGFR [MDRD]). The equations are as follows:
eGFR [MDRD] = 175 * (Serum Creatinine)^-1.154 * (Age)^-0.203 * 1.212 (if black or African American) * 0.742 (if female); eGFR [CKD-EPI] = 141 * min(serum creatinine/kappa,1)^alpha * max(serum creatinine/kappa, 1)^-1.209 * 0.993^age * 1.1018(if female) * 1.159(if black or African American), where kappa is 0.7 for females and 0.9 for males, alpha is -0.329 for females and -0.411 for males, min is minimum of serum creatinine/kappa or 1, and max is the maximum of serum creatinine/kappa or 1. The number of participants with at least a Baseline and a post-Baseline value are presented.
Timepoint [1] 0 0
Baseline, Every 6 m until the End of Study (42 m)

Eligibility
Key inclusion criteria
- Completed migalastat treatment in a previous Fabry disease protocol

- Both male and female participants were enrolled

- Age 16 years or older

- Male and female participants had to agree to use protocol-identified acceptable
contraception
Minimum age
16 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Estimated glomerular filtration rate (eGFR) in the previous study was <30
milliliters/minute/1.73 square meters (mL/min/1.73 m^2) unless there was a measured
GFR available within 3 m of the Baseline Visit that was >30 mL/min/1.73 m^2

- Had undergone, or was scheduled to undergo, kidney transplantation or was currently on
dialysis

- Pregnant or breast feeding

- Treated with another investigational drug (except migalastat) within 30 days of study
start

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

- Had documented transient ischemic attack, stroke, unstable angina, or myocardial
infarction within the 12 m before the Baseline Visit

- Had clinically significant, unstable cardiac disease in the opinion of the
investigator

- Had a history of allergy or sensitivity to migalastat (including excipients) or to
other iminosugars

- Required treatment with Glyset (miglitol) or Zavesca (miglustat)

- Had any intercurrent illness or condition that may have precluded the participant from
fulfilling the protocol requirements

- Had a severe or unsuitable concomitant medical condition

- Had a clinically significant abnormal laboratory value and a clinically significant
electrocardiogram finding at the Baseline Visit.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
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)
Recruitment hospital [1] 0 0
- Adelaide
Recruitment hospital [2] 0 0
- Parkville
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Georgia
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
Kansas
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
Oregon
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania
Country [9] 0 0
United States of America
State/province [9] 0 0
Texas
Country [10] 0 0
United States of America
State/province [10] 0 0
Virginia
Country [11] 0 0
United States of America
State/province [11] 0 0
Washington
Country [12] 0 0
Argentina
State/province [12] 0 0
Pilar
Country [13] 0 0
Belgium
State/province [13] 0 0
Edegem
Country [14] 0 0
Brazil
State/province [14] 0 0
Porto Alegre
Country [15] 0 0
Canada
State/province [15] 0 0
Montreal
Country [16] 0 0
Denmark
State/province [16] 0 0
Copenhagen
Country [17] 0 0
Egypt
State/province [17] 0 0
Cairo
Country [18] 0 0
France
State/province [18] 0 0
Garches
Country [19] 0 0
Italy
State/province [19] 0 0
Roma
Country [20] 0 0
Spain
State/province [20] 0 0
Barcelona
Country [21] 0 0
Turkey
State/province [21] 0 0
Ankara
Country [22] 0 0
United Kingdom
State/province [22] 0 0
London
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Salford

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

Ethics approval
Ethics application status

Summary
Brief summary
This was a long-term, open-label study of migalastat (123 milligrams [mg] of migalastat
[equivalent to 150 mg of migalastat hydrochloride]) (migalastat) in participants with Fabry
disease who completed treatment in a previous monotherapy trial with migalastat.
Trial website
https://clinicaltrials.gov/show/NCT01458119
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/show/NCT01458119