Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01196871
• Date of registration: 07/09/2010
• Prospective Registration: Yes
• Primary sponsor: Amicus Therapeutics
• 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
• Date of first enrolment: February 2, 2011
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01196871
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia, Belgium, Canada, France, Netherlands, United Kingdom, United States

Contacts

• Name: Medical Monitor Clinical Research
• Affiliation: Amicus Therapeutics

Key inclusion & exclusion criteria

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

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



Age minimum: 18 Years
Age maximum: 65 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Biological: Agalsidase Beta

Drug: Migalastat HCl

Biological: Agalsidase Alfa

Primary Outcome(s)

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 [Time Frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose]

Change In Cmax For Migalastat After Administration Of Agalsidase [Time Frame: 0 hr, 1 day post dose]

Change In AUC For Total a-Gal A Protein Levels After Administration Of Migalastat [Time Frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose]

Change In Area Under The Plasma Concentration Versus Time Curve (AUC) For Active a-Galactosidase A (a-Gal A) Levels After Administration Of Migalastat [Time Frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose]

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 [Time Frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose]

Change In Tmax And T1/2 For Migalastat After Administration Of Agalsidase [Time Frame: 0 hr, 1 day post dose]

Change In AUC For Migalastat After Administration Of Agalsidase [Time Frame: 0 hr, 1 day post dose]

Change In Maximum Observed Plasma Concentration (Cmax) For Active a-Gal A Levels After Administration Of Migalastat [Time Frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose]

Change In Tmax And T1/2 For Total a-Gal A Protein Levels After Administration Of Migalastat [Time Frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose]

Change In Cmax For Total a-Gal A Protein Levels After Administration Of Migalastat [Time Frame: 0 hr, 2 hr, 2 days, 7 days, 14 days post dose]

Secondary Outcome(s)

Change From Baseline To Day 7 In Active a-Gal A In Skin Following Treatment With Agalsidase Alone And Co-administration With Migalastat [Time Frame: Baseline, Day 7]

Secondary ID(s)

AT1001-013

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 01/11/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01196871