Trial Outcomes & Findings for Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease (NCT NCT02194985)

NCT ID: NCT02194985

Last Updated: 2020-12-21

Results Overview

An 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 time of reporting; visits typically occurred every 6 months. Serious AEs were life threatening or resulted in death, resulted in disability/incapacity, hospitalization or prolonged hospitalization, or a congenital anomaly. The criteria for AE severity were: Mild: awareness of sign or symptom, does not interfere with normal everyday activities; Moderate: discomforting, interferes with normal everyday activities, but able to function; Severe: incapacitating, prevents normal everyday activities or significantly affects clinical status and requires medical intervention. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 84 participants
• Primary outcome timeframe: Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years
• Results posted on: 2020-12-21

Participant Flow

Participant milestones

Measure	Migalastat HCl 150 mg Migalastat hydrochloride (HCl) 150 milligram (mg) was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
Overall Study STARTED	84
Overall Study Received at Least 1 Dose of Study Drug	84
Overall Study COMPLETED	73
Overall Study NOT COMPLETED	11

Reasons for withdrawal

Measure	Migalastat HCl 150 mg Migalastat hydrochloride (HCl) 150 milligram (mg) was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
Overall Study Adverse Event	1
Overall Study Met Protocol Defined Stopping Criteria	3
Overall Study Physician Decision	4
Overall Study Lost to Follow-up	1
Overall Study Withdrawal by Subject	2

Baseline Characteristics

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

Baseline characteristics by cohort

Measure	Migalastat HCl 150 mg n=84 Participants Migalastat HCl 150 mg was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
Age, Continuous	51.9 years STANDARD_DEVIATION 12.27 • n=5 Participants
Sex: Female, Male Female	50 Participants n=5 Participants
Sex: Female, Male Male	34 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	41 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	38 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	6 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	78 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years

Population: Safety Population: All participants who received at least 1 dose of study drug after they enrolled into this open-label extension study.

An 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 time of reporting; visits typically occurred every 6 months. Serious AEs were life threatening or resulted in death, resulted in disability/incapacity, hospitalization or prolonged hospitalization, or a congenital anomaly. The criteria for AE severity were: Mild: awareness of sign or symptom, does not interfere with normal everyday activities; Moderate: discomforting, interferes with normal everyday activities, but able to function; Severe: incapacitating, prevents normal everyday activities or significantly affects clinical status and requires medical intervention. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.

Outcome measures

Measure	Migalastat HCl 150 mg n=84 Participants Migalastat HCl 150 mg was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
Number Of Participants Experiencing Adverse Events (AEs) Participants with at least 1 AE	80 Participants
Number Of Participants Experiencing Adverse Events (AEs) Participants with at least 1 serious AE	26 Participants
Number Of Participants Experiencing Adverse Events (AEs) Participants discontinued due to AEs	1 Participants
Number Of Participants Experiencing Adverse Events (AEs) Participants with AEs leading to death	0 Participants
Number Of Participants Experiencing Adverse Events (AEs) Participants with AEs related to study drug	24 Participants
Number Of Participants Experiencing Adverse Events (AEs) Participants with AEs unrelated to study drug	56 Participants
Number Of Participants Experiencing Adverse Events (AEs) Participants with at least 1 mild AE	19 Participants
Number Of Participants Experiencing Adverse Events (AEs) Participants with at least 1 moderate AE	46 Participants
Number Of Participants Experiencing Adverse Events (AEs) Participants with at least 1 severe AE	15 Participants

SECONDARY outcome

Timeframe: Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Population: Intent-to-Treat Population: all participants who took at least 1 dose of the study drug after they had enrolled into this study.

The annualized rate of change in 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 × (1/Serum Creatinine in mg/deciliter^1.154) × (1/Age in years^0.203) × 0.742 [if female] × 1.212 [if black] × 0.808 [if Japanese];

eGFR [CKD-EPI] = 141 × min(Serum creatinine [Scr]/k, 1)α × max(Scr/k, 1) - 1.209 × 0.993Age × 1.018 [if female] × 1.159 [if black],

where Scr is serum creatinine, k is 0.7 for females and 0.9 for males, α is -0.329 for females and -0.411 for males, min indicates the minimum of Scr/k or 1, and max indicates the maximum of Scr/k or 1.

Participants with at least a Baseline and a post-Baseline value are presented.

Outcome measures

Measure	Migalastat HCl 150 mg n=84 Participants Migalastat HCl 150 mg was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR) eGFR[MDRD]	-1.6107 mL/min/1.73 m^2 Standard Deviation 5.62761
Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR) eGFR[CKD-EPI]	-1.3528 mL/min/1.73 m^2 Standard Deviation 4.84795

SECONDARY outcome

Timeframe: Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Population: All participants who took at least 1 dose of the study drug after they had enrolled into this study and had analyzable data at the specified time points.

The change from baseline in eGFR was calculated using eGFR[CKD-EPI] and eGFR[MDRD] equations. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.

Outcome measures

Measure	Migalastat HCl 150 mg n=82 Participants Migalastat HCl 150 mg was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
Change From Baseline In eGFR At End Of Study Baseline eGFR[MDRD]	79.0 mL/min/1.73 m^2 Standard Deviation 22.56
Change From Baseline In eGFR At End Of Study End of Study eGFR[MDRD]	75.8 mL/min/1.73 m^2 Standard Deviation 22.90
Change From Baseline In eGFR At End Of Study Change from Baseline eGFR[MDRD]	-1.4 mL/min/1.73 m^2 Standard Deviation 10.62
Change From Baseline In eGFR At End Of Study Baseline eGFR[CKD-EPI]	84.70 mL/min/1.73 m^2 Standard Deviation 23.092
Change From Baseline In eGFR At End Of Study End of Study eGFR[CKD-EPI]	82.02 mL/min/1.73 m^2 Standard Deviation 23.890
Change From Baseline In eGFR At End Of Study Change from Baseline eGFR[CKD-EPI]	-0.93 mL/min/1.73 m^2 Standard Deviation 9.828

SECONDARY outcome

Timeframe: Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Population: All participants who took at least 1 dose of the study drug after they had enrolled into this study and had analyzable data at the specified time points.

Concentrations of lyso-Gb3 were measured in plasma using a qualified assay. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.

Outcome measures

Measure	Migalastat HCl 150 mg n=83 Participants Migalastat HCl 150 mg was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study Baseline	13.317 nmol/L Standard Deviation 17.5428
Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study End of Study	11.758 nmol/L Standard Deviation 16.0662
Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study Change from Baseline	-1.054 nmol/L Standard Deviation 4.4885

SECONDARY outcome

Timeframe: Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Population: All male participants who took at least 1 dose of the study drug after they had enrolled into this study and had analyzable data at the specified time points.

The activity of the α-galactosidase A (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 data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Results for male participants are reported. Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.

Outcome measures

Measure	Migalastat HCl 150 mg n=30 Participants Migalastat HCl 150 mg was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study Baseline	6.882 nmol/hr/mg Standard Deviation 6.6857
Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study End of Study	5.290 nmol/hr/mg Standard Deviation 5.4054
Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study Change from Baseline	-1.375 nmol/hr/mg Standard Deviation 3.3432

SECONDARY outcome

Timeframe: Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Population: All participants who took at least 1 dose of the study drug after they had enrolled into this study and had analyzable data at the specified time points.

A 24-hour urine sample was collected to measure 24-hour urine protein. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.

Outcome measures

Measure	Migalastat HCl 150 mg n=83 Participants Migalastat HCl 150 mg was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
Change From Baseline In 24-hour Urine Protein To End Of Study Baseline	478.9 mg/day Standard Deviation 948.59
Change From Baseline In 24-hour Urine Protein To End Of Study End of Study	394.0 mg/day Standard Deviation 547.77
Change From Baseline In 24-hour Urine Protein To End Of Study Change from Baseline	5.4 mg/day Standard Deviation 233.49

SECONDARY outcome

Timeframe: Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Population: All participants who took at least 1 dose of the study drug after they had enrolled into this study and had analyzable data at the specified time points.

LVM was measured by echocardiography. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.

Outcome measures

Measure	Migalastat HCl 150 mg n=70 Participants Migalastat HCl 150 mg was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
Change From Baseline In Left Ventricular Mass (LVM) To End Of Study Baseline	178.879 gram Standard Deviation 78.6761
Change From Baseline In Left Ventricular Mass (LVM) To End Of Study End of Study	157.896 gram Standard Deviation 47.9947
Change From Baseline In Left Ventricular Mass (LVM) To End Of Study Change from Baseline	-0.803 gram Standard Deviation 18.8522

SECONDARY outcome

Timeframe: Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Population: All participants who took at least 1 dose of the study drug after they had enrolled into this study and had analyzable data at the specified time points.

LVMi was measured by echocardiography. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.

Outcome measures

Measure	Migalastat HCl 150 mg n=84 Participants Migalastat HCl 150 mg was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study Baseline	96.513 g/m^2 Standard Deviation 36.5691
Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study End of Study	83.912 g/m^2 Standard Deviation 22.7633
Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study Change from Baseline	-0.809 g/m^2 Standard Deviation 11.8056

SECONDARY outcome

Timeframe: Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Population: All participants who took at least 1 dose of the study drug after they had enrolled into this study and had analyzable data at the specified time points.

The SF-36 is a participant self-rated questionnaire that is a general measure of perceived health status comprising 36 questions, which yields an 8-scale health profile (Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional, and Mental Health). Scores on each item are summed and averaged (range: 0=worst to 100=best). Scores were normed to the US population. Higher score indicates less disability. A positive change from baseline indicates improvement. Baseline was defined as the data collected in the last visit of the previous (feeder) study. Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.

Outcome measures

Measure	Migalastat HCl 150 mg n=84 Participants Migalastat HCl 150 mg was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Baseline Physical Functioning	48.313 units on a scale Standard Deviation 9.2345
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire End of Study Physical Functioning	46.415 units on a scale Standard Deviation 10.2466
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Change from Baseline Physical Functioning	-1.276 units on a scale Standard Deviation 7.1627
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Baseline Role Physical	46.197 units on a scale Standard Deviation 10.0761
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire End of Study Role Physical	45.929 units on a scale Standard Deviation 10.5723
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Change from Baseline Role Physical	0.390 units on a scale Standard Deviation 7.3500
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Baseline Bodily Pain	46.889 units on a scale Standard Deviation 10.5983
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire End of Study Bodily Pain	46.737 units on a scale Standard Deviation 10.3368
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Change from Baseline Bodily Pain	0.425 units on a scale Standard Deviation 8.1342
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Baseline General Health	44.016 units on a scale Standard Deviation 10.2497
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire End of Study General Health	42.352 units on a scale Standard Deviation 10.1185
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Change from Baseline General Health	-0.811 units on a scale Standard Deviation 5.8672
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Baseline Vitality	46.375 units on a scale Standard Deviation 11.8451
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire End of Study Vitality	45.429 units on a scale Standard Deviation 12.4340
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Change from Baseline Vitality	-0.674 units on a scale Standard Deviation 8.5076
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Baseline Social Functioning	47.433 units on a scale Standard Deviation 10.4985
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire End of Study Social Functioning	47.648 units on a scale Standard Deviation 9.5165
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Change from Baseline Social Functioning	0.401 units on a scale Standard Deviation 8.4356
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Baseline Role Emotional	47.547 units on a scale Standard Deviation 10.3897
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire End of Study Role Emotional	46.141 units on a scale Standard Deviation 10.9054
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Change from Baseline Role Emotional	-0.929 units on a scale Standard Deviation 10.6679
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Baseline Mental Health	48.501 units on a scale Standard Deviation 10.6878
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire End of Study Mental Health	49.578 units on a scale Standard Deviation 10.5732
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Change from Baseline Mental Health	0.872 units on a scale Standard Deviation 6.2528
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Baseline Physical Component	46.184 units on a scale Standard Deviation 10.2268
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire End of Study Physical Component	44.825 units on a scale Standard Deviation 10.4292
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Change from Baseline Physical Component	-0.508 units on a scale Standard Deviation 6.2494
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Baseline Mental Component	47.917 units on a scale Standard Deviation 11.5480
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire End of Study Mental Component	48.175 units on a scale Standard Deviation 10.9497
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire Change from Baseline Mental Component	0.187 units on a scale Standard Deviation 8.2714

Adverse Events

Migalastat HCl 150 mg

Serious events: 26 serious events

Other events: 74 other events

Deaths: 0 deaths

Serious adverse events

Measure	Migalastat HCl 150 mg n=84 participants at risk Migalastat HCl 150 mg was administered orally every other day.
Cardiac disorders Atrial fibrillation	2.4% 2/84 • Baseline to up to 4.4 years (includes safety follow-up)
Cardiac disorders Atrioventricular block complete	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Cardiac disorders Left ventricular hypertrophy	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Cardiac disorders Ventricular tachycardia	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Eye disorders Visual impairment	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Abdominal pain	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Abdominal pain upper	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Abdominal wall haematoma	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Barrett's oesophagus	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Large intestine perforation	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
General disorders Device malfunction	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Hepatobiliary disorders Biliary dyskinesia	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Immune system disorders Drug hypersensitivity	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Infections and infestations Appendicitis perforated	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Infections and infestations Biliary sepsis	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Infections and infestations Diverticulitis	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Infections and infestations Infective exacerbation of bronchiectasis	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Infections and infestations Pneumonia	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Injury, poisoning and procedural complications Rib fracture	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Investigations Heart rate increased	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Metabolism and nutrition disorders Dehydration	2.4% 2/84 • Baseline to up to 4.4 years (includes safety follow-up)
Metabolism and nutrition disorders Diabetes mellitus	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Metabolism and nutrition disorders Lipomatosis	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Musculoskeletal and connective tissue disorders Arthritis	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Musculoskeletal and connective tissue disorders Osteoarthritis	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Musculoskeletal and connective tissue disorders Periarthritis	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Musculoskeletal and connective tissue disorders Tendon calcification	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Breast cancer	2.4% 2/84 • Baseline to up to 4.4 years (includes safety follow-up)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Invasive lobular breast carcinoma	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Nervous system disorders Convulsion	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Nervous system disorders Embolic stroke	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Nervous system disorders Headache	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Nervous system disorders Neuralgia	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Nervous system disorders Syncope	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Nervous system disorders Transient ischaemic attack	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Psychiatric disorders Depression	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Psychiatric disorders Suicidal ideation	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Renal and urinary disorders Renal failure	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Reproductive system and breast disorders Uterine prolapse	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Respiratory, thoracic and mediastinal disorders Dyspnoea	2.4% 2/84 • Baseline to up to 4.4 years (includes safety follow-up)
Respiratory, thoracic and mediastinal disorders Pneumothorax	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Skin and subcutaneous tissue disorders Subcutaneous emphysema	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Vascular disorders Air embolism	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Vascular disorders Hypertension	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)
Vascular disorders Hypotension	1.2% 1/84 • Baseline to up to 4.4 years (includes safety follow-up)

Other adverse events

Measure	Migalastat HCl 150 mg n=84 participants at risk Migalastat HCl 150 mg was administered orally every other day.
Ear and labyrinth disorders Vertigo	8.3% 7/84 • Baseline to up to 4.4 years (includes safety follow-up)
Endocrine disorders Hypothyroidism	6.0% 5/84 • Baseline to up to 4.4 years (includes safety follow-up)
Cardiac disorders Palpitations	7.1% 6/84 • Baseline to up to 4.4 years (includes safety follow-up)
Ear and labyrinth disorders Tinnitus	8.3% 7/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Nausea	11.9% 10/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Diarrhoea	10.7% 9/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Abdominal pain	8.3% 7/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Abdominal pain upper	7.1% 6/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Constipation	7.1% 6/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Dyspepsia	6.0% 5/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Gastrooesophageal reflux disease	6.0% 5/84 • Baseline to up to 4.4 years (includes safety follow-up)
Gastrointestinal disorders Vomiting	6.0% 5/84 • Baseline to up to 4.4 years (includes safety follow-up)
General disorders Fatigue	19.0% 16/84 • Baseline to up to 4.4 years (includes safety follow-up)
General disorders Oedema peripheral	14.3% 12/84 • Baseline to up to 4.4 years (includes safety follow-up)
General disorders Pyrexia	10.7% 9/84 • Baseline to up to 4.4 years (includes safety follow-up)
General disorders Pain	6.0% 5/84 • Baseline to up to 4.4 years (includes safety follow-up)
Infections and infestations Nasopharyngitis	20.2% 17/84 • Baseline to up to 4.4 years (includes safety follow-up)
Infections and infestations Upper respiratory tract infection	14.3% 12/84 • Baseline to up to 4.4 years (includes safety follow-up)
Infections and infestations Urinary tract infection	14.3% 12/84 • Baseline to up to 4.4 years (includes safety follow-up)
Infections and infestations Influenza	11.9% 10/84 • Baseline to up to 4.4 years (includes safety follow-up)
Infections and infestations Sinusitis	10.7% 9/84 • Baseline to up to 4.4 years (includes safety follow-up)
Infections and infestations Bronchitis	7.1% 6/84 • Baseline to up to 4.4 years (includes safety follow-up)
Injury, poisoning and procedural complications Overdose	14.3% 12/84 • Baseline to up to 4.4 years (includes safety follow-up)
Investigations Albumin urine present	7.1% 6/84 • Baseline to up to 4.4 years (includes safety follow-up)
Investigations Blood uric acid increased	7.1% 6/84 • Baseline to up to 4.4 years (includes safety follow-up)
Investigations Protein urine present	7.1% 6/84 • Baseline to up to 4.4 years (includes safety follow-up)
Investigations Blood creatinine increased	6.0% 5/84 • Baseline to up to 4.4 years (includes safety follow-up)
Investigations Glomerular filtration rate decreased	6.0% 5/84 • Baseline to up to 4.4 years (includes safety follow-up)
Metabolism and nutrition disorders Hypercholesterolaemia	6.0% 5/84 • Baseline to up to 4.4 years (includes safety follow-up)
Musculoskeletal and connective tissue disorders Arthralgia	19.0% 16/84 • Baseline to up to 4.4 years (includes safety follow-up)
Musculoskeletal and connective tissue disorders Pain in extremity	16.7% 14/84 • Baseline to up to 4.4 years (includes safety follow-up)
Musculoskeletal and connective tissue disorders Musculoskeletal pain	11.9% 10/84 • Baseline to up to 4.4 years (includes safety follow-up)
Musculoskeletal and connective tissue disorders Back pain	10.7% 9/84 • Baseline to up to 4.4 years (includes safety follow-up)
Musculoskeletal and connective tissue disorders Muscle spasms	8.3% 7/84 • Baseline to up to 4.4 years (includes safety follow-up)
Musculoskeletal and connective tissue disorders Tendonitis	8.3% 7/84 • Baseline to up to 4.4 years (includes safety follow-up)
Nervous system disorders Paraesthesia	15.5% 13/84 • Baseline to up to 4.4 years (includes safety follow-up)
Nervous system disorders Headache	14.3% 12/84 • Baseline to up to 4.4 years (includes safety follow-up)
Nervous system disorders Dizziness	11.9% 10/84 • Baseline to up to 4.4 years (includes safety follow-up)
Nervous system disorders Hypoaesthesia	11.9% 10/84 • Baseline to up to 4.4 years (includes safety follow-up)
Nervous system disorders Migraine	6.0% 5/84 • Baseline to up to 4.4 years (includes safety follow-up)
Nervous system disorders Neuralgia	6.0% 5/84 • Baseline to up to 4.4 years (includes safety follow-up)
Psychiatric disorders Depression	9.5% 8/84 • Baseline to up to 4.4 years (includes safety follow-up)
Psychiatric disorders Insomnia	7.1% 6/84 • Baseline to up to 4.4 years (includes safety follow-up)
Renal and urinary disorders Proteinuria	14.3% 12/84 • Baseline to up to 4.4 years (includes safety follow-up)
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	8.3% 7/84 • Baseline to up to 4.4 years (includes safety follow-up)
Respiratory, thoracic and mediastinal disorders Asthma	7.1% 6/84 • Baseline to up to 4.4 years (includes safety follow-up)
Respiratory, thoracic and mediastinal disorders Cough	7.1% 6/84 • Baseline to up to 4.4 years (includes safety follow-up)
Respiratory, thoracic and mediastinal disorders Dyspnoea	7.1% 6/84 • Baseline to up to 4.4 years (includes safety follow-up)
Respiratory, thoracic and mediastinal disorders Dyspnoea exertional	7.1% 6/84 • Baseline to up to 4.4 years (includes safety follow-up)
Vascular disorders Hypotension	6.0% 5/84 • Baseline to up to 4.4 years (includes safety follow-up)
Musculoskeletal and connective tissue disorders Muscular weakness	8.3% 7/84 • Baseline to up to 4.4 years (includes safety follow-up)

Additional Information

Medical Affairs

Amicus Therapeutics

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

Email: MedInfoUSA@amicusrx.com

Results disclosure agreements

• Principal investigator is a sponsor employee The investigator can publish only the results from this trial, provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review prior to submission for publication. If requested and prior to publication, the investigator will remove information deemed confidential or proprietary by the sponsor and will withhold publication 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