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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01196871




Registration number
NCT01196871
Ethics application status
Date submitted
7/09/2010
Date registered
9/09/2010
Date last updated
19/12/2018

Titles & IDs
Public title
Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease
Scientific title
An Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Subjects With Fabry Disease
Secondary ID [1] 0 0
AT1001-013
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
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Migalastat HCl
Treatment: Other - Agalsidase Beta
Treatment: Other - Agalsidase Alfa

Experimental: Agalsidase Beta (0.5 mg/kg)-Migalastat (150 mg) -

Experimental: Agalsidase Beta (1.0 mg/kg)-Migalastat (150 mg) -

Experimental: Agalsidase Alfa (0.2 mg/kg)-Migalastat (150 mg) -

Experimental: Agalsidase Beta (0.5 mg/kg)-Migalastat (450 mg) -

Experimental: Agalsidase Beta (1.0 mg/kg)-Migalastat (450 mg) -

Experimental: Agalsidase Alfa (0.2 mg/kg)-Migalastat (450 mg) -


Treatment: Drugs: Migalastat HCl
Oral capsules, single dose

Treatment: Other: Agalsidase Beta
IV infusion, single dose

Treatment: Other: Agalsidase Alfa
IV infusion, single dose
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change In Area Under The Plasma Concentration Versus Time Curve (AUC) For Active a-Galactosidase A (a-Gal A) Levels After Administration Of Migalastat
Timepoint [1] 0 0
0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Primary outcome [2] 0 0
Change In Maximum Observed Plasma Concentration (Cmax) For Active a-Gal A Levels After Administration Of Migalastat
Timepoint [2] 0 0
0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Primary outcome [3] 0 0
Change In Time To Maximum Observed Plasma Concentration (Tmax) And Terminal Elimination Half-life (T1/2) For Active a-Gal A Levels After Administration Of Migalastat
Timepoint [3] 0 0
0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Primary outcome [4] 0 0
Change In AUC For Total a-Gal A Protein Levels After Administration Of Migalastat
Timepoint [4] 0 0
0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Primary outcome [5] 0 0
Change In Percentage Of AUCinfinity Extrapolated From The Last Time Point At Which Concentration Is Quantified To Infinity (AUCextrapolated %) For Total a-Gal A Protein Levels After Administration Of Migalastat
Timepoint [5] 0 0
0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Primary outcome [6] 0 0
Change In Cmax For Total a-Gal A Protein Levels After Administration Of Migalastat
Timepoint [6] 0 0
0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Primary outcome [7] 0 0
Change In Tmax And T1/2 For Total a-Gal A Protein Levels After Administration Of Migalastat
Timepoint [7] 0 0
0 hr, 2 hr, 2 days, 7 days, 14 days post dose
Primary outcome [8] 0 0
Change In AUC For Migalastat After Administration Of Agalsidase
Timepoint [8] 0 0
0 hr, 1 day post dose
Primary outcome [9] 0 0
Change In Cmax For Migalastat After Administration Of Agalsidase
Timepoint [9] 0 0
0 hr, 1 day post dose
Primary outcome [10] 0 0
Change In Tmax And T1/2 For Migalastat After Administration Of Agalsidase
Timepoint [10] 0 0
0 hr, 1 day post dose
Secondary outcome [1] 0 0
Change From Baseline To Day 7 In Active a-Gal A In Skin Following Treatment With Agalsidase Alone And Co-administration With Migalastat
Timepoint [1] 0 0
Baseline, Day 7

Eligibility
Key inclusion criteria
* Male diagnosed with Fabry disease and between 18 and 65 years of age, inclusive
* Body mass index between 18-35 kg per meter squared
* Had initiated treatment with agalsidase at least 1 month prior to screening, and had received at least 2 infusions before screening
* Had stable dose level, dosing regimen, and form of agalsidase for at least 1 month before screening
* Had an estimated creatinine clearance greater than or equal to 50 milliliters (mL)/minute at screening
* Agreed to use medically accepted methods of contraception during the study and for 30 days after study completion
* Were willing and able to provide written informed consent
Minimum age
18 Years
Maximum age
65 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Had a documented transient ischemic attack, ischemic stroke, unstable angina, or myocardial infarction within the 3 months before screening
* Had clinically significant unstable cardiac disease (for example, cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or New York Heart Association class III or IV congestive heart failure)
* History of allergy or sensitivity to study drug (including excipients) or other iminosugars (such as miglustat, miglitol)
* Required a concomitant medication prohibited by the protocol: Glyset® (miglitol), or Zavesca® (miglustat)
* Any investigational/experimental drug or device within 30 days of screening, except for use of investigational enzyme replacement therapy for Fabry disease
* Had any intercurrent illness or condition that might have precluded the participant from fulfilling the protocol requirements or suggested to the investigator that the potential participant might have had an unacceptable risk by participating in this study

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
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
Other
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
2/02/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
9/10/2012
Sample size
Target
Accrual to date
Final
20
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Nedlands
Recruitment hospital [2] 0 0
- Parkville
Recruitment postcode(s) [1] 0 0
- Nedlands
Recruitment postcode(s) [2] 0 0
- Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Iowa
Country [4] 0 0
United States of America
State/province [4] 0 0
Kansas
Country [5] 0 0
United States of America
State/province [5] 0 0
Virginia
Country [6] 0 0
Belgium
State/province [6] 0 0
Edegem
Country [7] 0 0
Canada
State/province [7] 0 0
Montreal
Country [8] 0 0
Netherlands
State/province [8] 0 0
Amsterdam

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

Ethics approval
Ethics application status

Summary
Brief summary
The objective was to determine the effects of a single dose of migalastat hydrochloride (HCl) (migalastat) 150 and 450 milligrams (mg) on the safety and plasma pharmacokinetics (PK) of agalsidase and the effects of agalsidase on the safety and PK of migalastat 150 mg.
Trial website
https://clinicaltrials.gov/study/NCT01196871
Trial related presentations / publications
Warnock DG, Bichet DG, Holida M, Goker-Alpan O, Nicholls K, Thomas M, Eyskens F, Shankar S, Adera M, Sitaraman S, Khanna R, Flanagan JJ, Wustman BA, Barth J, Barlow C, Valenzano KJ, Lockhart DJ, Boudes P, Johnson FK. Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active alpha-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase. PLoS One. 2015 Aug 7;10(8):e0134341. doi: 10.1371/journal.pone.0134341. eCollection 2015.
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/study/NCT01196871