Trial Outcomes & Findings for Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study (NCT NCT00526071)

Last Updated: 2018-10-03

Results Overview

A TEAE was defined as any adverse event (AE) with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe AE was defined as an AE that was incapacitating and required medical intervention. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through End of Study (EOS) or follow-up (for participants who did not enroll in Study AT1001-041) are presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

23 participants

Primary outcome timeframe

Day 1 (after dosing) through EOS (up to 56 months) or follow-up (28 days after EOS)

Results posted on

2018-10-03

Participant Flow

An open label, long-term extension study for participants who completed one of 4 preceding Phase 2 studies (FAB-CL-201 \[NCT00214500\], FAB-CL-202 \[NCT00283959\], FAB-CL-203 \[NCT00283933\], or FAB-CL-204 \[NCT00304512\]). Participants could enter this study directly upon completion of the previous study or at a later point.

This study included a dose escalation period (DEP), in which migalastat was administered at 250 milligrams (mg) for 2 months. If there were no safety concerns the dose was increased to 500 mg for up to 10 months, depending on the date of amendment approval. Before and after the DEP, all but 1 participant (post-DEP 300 mg instead) received 150 mg.

Participant milestones

Participant milestones
Measure	Migalastat Participants received migalastat 150 mg given orally once every other day (QOD) before and after the DEP. During the DEP participants received 250 mg once daily (QD) for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site. One participant received migalastat 300 mg (QD for 3 days and 4 days off per week) until the end of the study.
Overall Study STARTED	23
Overall Study Safety Population	23
Overall Study Pharmacodynamic (PD) Population	23
Overall Study Amenable Population; Men	11
Overall Study Amenable Population; Women	5
Overall Study Non-amenable Population; Men	3
Overall Study Non-amenable Population; Women	4
Overall Study COMPLETED	17
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Migalastat Participants received migalastat 150 mg given orally once every other day (QOD) before and after the DEP. During the DEP participants received 250 mg once daily (QD) for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site. One participant received migalastat 300 mg (QD for 3 days and 4 days off per week) until the end of the study.
Overall Study Adverse Event	1
Overall Study Withdrawal by Subject	1
Overall Study Lack of Response to Study Drug	4

Baseline Characteristics

Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Migalastat n=23 Participants Participants received migalastat 150 mg given orally QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site. One participant received migalastat 300 mg (QD for 3 days and 4 days off per week) until the end of the study.
Age, Continuous	43.1 years STANDARD_DEVIATION 11.33 • n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	14 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1 (after dosing) through EOS (up to 56 months) or follow-up (28 days after EOS)

Population: Safety Population: all participants who received at least 1 dose of study drug.

Outcome measures

Outcome measures
Measure	Migalastat n=23 Participants Participants received migalastat 150 mg given orally QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site. One participant received migalastat 300 mg (QD for 3 days and 4 days off per week) until the end of the study.	Amenable Population; Women Amenable participants were those with mutant forms of α-Gal A determined to be responsive to migalastat treatment based on the CT-HEK assay. Migalastat was administered orally, 150 mg QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site.	Non-amenable Population; Men Non-amenable participants were those without mutant forms of α-Gal A as determined by the CT-HEK assay. Migalastat was administered orally, 150 mg QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site.	Non-amenable Population; Women Non-amenable participants were those without mutant forms of α-Gal A as determined by the CT-HEK assay. Migalastat was administered orally, 150 mg QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site.
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)	9 Participants	—	—	—

SECONDARY outcome

Timeframe: Baseline, Month 42

Population: PD Population: all participants who received at least 1 dose of study drug and who had a baseline measurement and at least 1 postbaseline PD measure.

The activity of the α-Gal A enzyme was measured in leukocyte lysate by a validated fluorometric assay method, using 4-methylumbelliferone as a reference. The activity values obtained were normalized to protein (measured using a colorimetric assay). Baseline was defined as the last non-missing pre-treatment value for each participant in his or her respective preceding migalastat Phase 2 study. A negative change from Baseline indicates that α-Gal A activity decreased. α-Gal A activity levels are presented for Baseline and Month 42.

Outcome measures

Outcome measures
Measure	Migalastat n=11 Participants Participants received migalastat 150 mg given orally QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site. One participant received migalastat 300 mg (QD for 3 days and 4 days off per week) until the end of the study.	Amenable Population; Women n=5 Participants Amenable participants were those with mutant forms of α-Gal A determined to be responsive to migalastat treatment based on the CT-HEK assay. Migalastat was administered orally, 150 mg QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site.	Non-amenable Population; Men n=3 Participants Non-amenable participants were those without mutant forms of α-Gal A as determined by the CT-HEK assay. Migalastat was administered orally, 150 mg QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site.	Non-amenable Population; Women n=4 Participants Non-amenable participants were those without mutant forms of α-Gal A as determined by the CT-HEK assay. Migalastat was administered orally, 150 mg QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site.
Absolute Change From Baseline In α-Galactosidase A (α-Gal A) Activity In Leukocytes To Month 42 Baseline	2.08 nanomoles (nm)/hour (hr)/mg protein Standard Deviation 2.433	16.84 nanomoles (nm)/hour (hr)/mg protein Standard Deviation 7.955	0.09 nanomoles (nm)/hour (hr)/mg protein Standard Deviation 0.047	14.62 nanomoles (nm)/hour (hr)/mg protein Standard Deviation 8.944
Absolute Change From Baseline In α-Galactosidase A (α-Gal A) Activity In Leukocytes To Month 42 Month 42	7.88 nanomoles (nm)/hour (hr)/mg protein Standard Deviation 8.691	18.31 nanomoles (nm)/hour (hr)/mg protein Standard Deviation 14.186	—	9.77 nanomoles (nm)/hour (hr)/mg protein Standard Deviation 5.071

SECONDARY outcome

Timeframe: 0 (predose on Day 1; start of DEP), 3 hr (postdose at Month 2; during DEP])

Population: Safety Population: all participants who received at least 1 dose of study drug.

The concentration of migalastat was evaluated in plasma following a dose of 250 mg and 500 mg. Blood samples were taken at trough (predose or Time 0; just prior to the third dose during a 3 day on, 4 days off dosing regimen) and at peak (3 hr postdose; after the third dose during a 3 day on, 4 days off dosing regimen) during the Day 1 (250 mg) and Month 2 (500 mg) visits. This outcome presents the lowest and highest concentrations of migalastat measured in any of the participants for predose and 3 hr postdose.

Outcome measures

Outcome measures
Measure	Migalastat n=23 Participants Participants received migalastat 150 mg given orally QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site. One participant received migalastat 300 mg (QD for 3 days and 4 days off per week) until the end of the study.	Amenable Population; Women Amenable participants were those with mutant forms of α-Gal A determined to be responsive to migalastat treatment based on the CT-HEK assay. Migalastat was administered orally, 150 mg QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site.	Non-amenable Population; Men Non-amenable participants were those without mutant forms of α-Gal A as determined by the CT-HEK assay. Migalastat was administered orally, 150 mg QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site.	Non-amenable Population; Women Non-amenable participants were those without mutant forms of α-Gal A as determined by the CT-HEK assay. Migalastat was administered orally, 150 mg QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site.
Pharmacokinetics Of Migalastat As Assessed By Plasma Concentration Predose Lowest Concentration	5.94 nanograms/milliliter	—	—	—
Pharmacokinetics Of Migalastat As Assessed By Plasma Concentration Predose Highest Concentration	313 nanograms/milliliter	—	—	—
Pharmacokinetics Of Migalastat As Assessed By Plasma Concentration 250 mg 3 hr Postdose Lowest Concentration	908 nanograms/milliliter	—	—	—
Pharmacokinetics Of Migalastat As Assessed By Plasma Concentration 250 mg 3 hr Postdose Highest Concentration	5250 nanograms/milliliter	—	—	—
Pharmacokinetics Of Migalastat As Assessed By Plasma Concentration 500 mg 3 hr Postdose Lowest Concentration	113 nanograms/milliliter	—	—	—
Pharmacokinetics Of Migalastat As Assessed By Plasma Concentration 500 mg 3 hr Postdose Highest Concentration	8500 nanograms/milliliter	—	—	—

Adverse Events

Migalastat

Serious events: 7 serious events

Other events: 23 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Migalastat n=23 participants at risk Participants received migalastat 150 mg given orally QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site. One participant received migalastat 300 mg (QD for 3 days and 4 days off per week) until the end of the study.
Cardiac disorders Atrial Fibrillation	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Cardiac disorders Atrial Flutter	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Cardiac disorders Atrioventricular Block	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Cardiac disorders Cardiac Failure Congestive	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Cardiac disorders Ventricular Fibrillation	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Endocrine disorders Hyperthyroidism	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Gastrointestinal disorders Dyspepsia	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
General disorders Sensation of Foreign Body	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Injury, poisoning and procedural complications Ankle Fracture	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Injury, poisoning and procedural complications Post Procedural Haemorrhage	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Metabolism and nutrition disorders Dehydration	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Metabolism and nutrition disorders Malnutrition	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Nervous system disorders Transient Ischaemic Attack	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Nervous system disorders Cerebrovascular Accident	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Nervous system disorders Syncope	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Respiratory, thoracic and mediastinal disorders Epistaxis	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Respiratory, thoracic and mediastinal disorders Pneumonia Aspiration	4.3% 1/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.

Other adverse events

Other adverse events
Measure	Migalastat n=23 participants at risk Participants received migalastat 150 mg given orally QOD before and after the DEP. During the DEP participants received 250 mg QD for 3 days, 4 days off per week for 2 months then 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site. One participant received migalastat 300 mg (QD for 3 days and 4 days off per week) until the end of the study.
Infections and infestations Influenza	26.1% 6/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Infections and infestations Nasopharyngitis	21.7% 5/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Infections and infestations Sinusitis	21.7% 5/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Infections and infestations Upper respiratory tract infection	17.4% 4/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Infections and infestations Urinary tract infection	17.4% 4/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Infections and infestations Cystitis	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Infections and infestations Lower respiratory tract infection	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Infections and infestations Rhinitis	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Gastrointestinal disorders Abdominal pain upper	21.7% 5/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Gastrointestinal disorders Diarrhoea	21.7% 5/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Gastrointestinal disorders Abdominal pain	17.4% 4/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Gastrointestinal disorders Gastrooesophageal reflux disease	17.4% 4/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Gastrointestinal disorders Vomiting	17.4% 4/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Gastrointestinal disorders Abdominal distension	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Gastrointestinal disorders Abdominal pain lower	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Gastrointestinal disorders Constipation	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Gastrointestinal disorders Dry mouth	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Gastrointestinal disorders Nausea	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Arthralgia	39.1% 9/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Back pain	34.8% 8/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Pain in extremity	26.1% 6/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Myalgia	21.7% 5/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Flank pain	13.0% 3/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Muscle spasms	13.0% 3/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Joint swelling	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Muscle twitching	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Muscular weakness	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Musculoskeletal chest pain	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Neck pain	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Musculoskeletal and connective tissue disorders Osteoarthritis	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
General disorders Fatigue	34.8% 8/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
General disorders Oedema peripheral	13.0% 3/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
General disorders Pyrexia	13.0% 3/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
General disorders Asthenia	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
General disorders Chest pain	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
General disorders Influenza like illness	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
General disorders Irritability	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
General disorders Pain	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Nervous system disorders Headache	26.1% 6/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Nervous system disorders Dizziness	17.4% 4/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Nervous system disorders Paraesthesia	13.0% 3/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Nervous system disorders Migraine	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Investigations Alanine aminotransferase increased	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Investigations Platelet count increased	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Investigations Weight increased	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Respiratory, thoracic and mediastinal disorders Cough	13.0% 3/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	13.0% 3/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Respiratory, thoracic and mediastinal disorders Dyspnoea	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Respiratory, thoracic and mediastinal disorders Epistaxis	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Respiratory, thoracic and mediastinal disorders Sinus congestion	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Cardiac disorders Palpitations	21.7% 5/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Cardiac disorders Sinus bradycardia	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Injury, poisoning and procedural complications Fall	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Injury, poisoning and procedural complications Limb injury	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Injury, poisoning and procedural complications Post procedural haematuria	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Skin and subcutaneous tissue disorders Dermal cyst	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Skin and subcutaneous tissue disorders Dry skin	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Skin and subcutaneous tissue disorders Rash	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Ear and labyrinth disorders Vertigo	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Psychiatric disorders Insomnia	17.4% 4/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Psychiatric disorders Depression	13.0% 3/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Psychiatric disorders Anxiety	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Renal and urinary disorders Haematuria	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Eye disorders Vision blurred	13.0% 3/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Metabolism and nutrition disorders Vitamin d deficiency	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Blood and lymphatic system disorders Lymphadenopathy	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.
Congenital, familial and genetic disorders Fabry's disease	8.7% 2/23 • Day 1 after dosing through EOS (up to 56 months) or follow-up (28 days after EOS). Follow-up was not required for participants who enrolled in Study AT1001-041.

Additional Information

Amicus Therapeutics

Medical Affairs

Phone: +1-877-426-4287 (877-4-AMICUS)

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The investigator can only publish the results from this trial provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review. If requested, the investigator will remove information deemed confidential or proprietary by the sponsor and will withhold publication for an additional period of time to allow the sponsor to take appropriate measures to establish and preserve its proprietary rights.
Publication restrictions are in place

Restriction type: OTHER