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

• ClinicalTrials.gov Identifier: NCT02194985
• Recruitment Status: Completed
• First Posted: July 21, 2014
• Results First Posted: December 21, 2020
• Last Update Posted: December 21, 2020
• Sponsor: Amicus Therapeutics
• Information provided by (Responsible Party): Amicus Therapeutics

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Fabry Disease
• Intervention: Drug: migalastat HCl 150 mg
• Enrollment: 84

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Migalastat HCl 150 mg
Arm/Group Description	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).
Period Title: Overall Study
Started	84
Received at Least 1 Dose of Study Drug	84
Completed	73
Not Completed	11
Reason Not Completed
Adverse Event	1
Met Protocol Defined Stopping Criteria	3
Physician Decision	4
Lost to Follow-up	1
Withdrawal by Subject	2

Baseline Characteristics

Arm/Group Title		Migalastat HCl 150 mg
Arm/Group Description		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).
Overall Number of Baseline Participants		84
Baseline Analysis Population Description		All participants who took at least 1 dose of the study drug after they had enrolled into this study.
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	84 participants
		51.9 (12.27)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	84 participants
	Female	50 59.5%
	Male	34 40.5%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	84 participants
	Hispanic or Latino	5 6.0%
	Not Hispanic or Latino	41 48.8%
	Unknown or Not Reported	38 45.2%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	84 participants
	American Indian or Alaska Native	0 0.0%
	Asian	6 7.1%
	Native Hawaiian or Other Pacific Islander	0 0.0%
	Black or African American	0 0.0%
	White	78 92.9%
	More than one race	0 0.0%
	Unknown or Not Reported	0 0.0%

Outcome Measures

1. Primary Outcome

Title	Number Of Participants Experiencing Adverse Events (AEs)
Description	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.
Time Frame	Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Migalastat HCl 150 mg
Arm/Group Description:	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).
Overall Number of Participants Analyzed	84
Measure Type: Count of Participants; Unit of Measure: Participants
Participants with at least 1 AE	80 95.2%
Participants with at least 1 serious AE	26 31.0%
Participants discontinued due to AEs	1 1.2%
Participants with AEs leading to death	0 0.0%
Participants with AEs related to study drug	24 28.6%
Participants with AEs unrelated to study drug	56 66.7%
Participants with at least 1 mild AE	19 22.6%
Participants with at least 1 moderate AE	46 54.8%
Participants with at least 1 severe AE	15 17.9%

2. Secondary Outcome

Title	Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)
Description	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.
Time Frame	Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Migalastat HCl 150 mg
Arm/Group Description:	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).
Overall Number of Participants Analyzed	84
Mean (Standard Deviation); Unit of Measure: mL/min/1.73 m^2
eGFR[MDRD]	-1.6107 (5.62761)
eGFR[CKD-EPI]	-1.3528 (4.84795)

3. Secondary Outcome

Title	Change From Baseline In eGFR At End Of Study
Description	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.
Time Frame	Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title		Migalastat HCl 150 mg
Arm/Group Description:		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).
Overall Number of Participants Analyzed		82
Mean (Standard Deviation); Unit of Measure: mL/min/1.73 m^2
Baseline eGFR[MDRD]	Number Analyzed	82 participants
		79.0 (22.56)
End of Study eGFR[MDRD]	Number Analyzed	73 participants
		75.8 (22.90)
Change from Baseline eGFR[MDRD]	Number Analyzed	72 participants
		-1.4 (10.62)
Baseline eGFR[CKD-EPI]	Number Analyzed	82 participants
		84.70 (23.092)
End of Study eGFR[CKD-EPI]	Number Analyzed	73 participants
		82.02 (23.890)
Change from Baseline eGFR[CKD-EPI]	Number Analyzed	72 participants
		-0.93 (9.828)

4. Secondary Outcome

Title	Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study
Description	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.
Time Frame	Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title		Migalastat HCl 150 mg
Arm/Group Description:		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).
Overall Number of Participants Analyzed		83
Mean (Standard Deviation); Unit of Measure: nmol/L
Baseline	Number Analyzed	83 participants
		13.317 (17.5428)
End of Study	Number Analyzed	75 participants
		11.758 (16.0662)
Change from Baseline	Number Analyzed	75 participants
		-1.054 (4.4885)

5. Secondary Outcome

Title	Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study
Description	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.
Time Frame	Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title		Migalastat HCl 150 mg
Arm/Group Description:		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).
Overall Number of Participants Analyzed		30
Mean (Standard Deviation); Unit of Measure: nmol/hr/mg
Baseline	Number Analyzed	27 participants
		6.882 (6.6857)
End of Study	Number Analyzed	30 participants
		5.290 (5.4054)
Change from Baseline	Number Analyzed	24 participants
		-1.375 (3.3432)

6. Secondary Outcome

Title	Change From Baseline In 24-hour Urine Protein To End Of Study
Description	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.
Time Frame	Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title		Migalastat HCl 150 mg
Arm/Group Description:		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).
Overall Number of Participants Analyzed		83
Mean (Standard Deviation); Unit of Measure: mg/day
Baseline	Number Analyzed	83 participants
		478.9 (948.59)
End of Study	Number Analyzed	66 participants
		394.0 (547.77)
Change from Baseline	Number Analyzed	66 participants
		5.4 (233.49)

7. Secondary Outcome

Title	Change From Baseline In Left Ventricular Mass (LVM) To End Of Study
Description	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.
Time Frame	Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title		Migalastat HCl 150 mg
Arm/Group Description:		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).
Overall Number of Participants Analyzed		70
Mean (Standard Deviation); Unit of Measure: gram
Baseline	Number Analyzed	70 participants
		178.879 (78.6761)
End of Study	Number Analyzed	25 participants
		157.896 (47.9947)
Change from Baseline	Number Analyzed	24 participants
		-0.803 (18.8522)

8. Secondary Outcome

Title	Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study
Description	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.
Time Frame	Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title		Migalastat HCl 150 mg
Arm/Group Description:		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).
Overall Number of Participants Analyzed		84
Mean (Standard Deviation); Unit of Measure: g/m^2
Baseline	Number Analyzed	68 participants
		96.513 (36.5691)
End of Study	Number Analyzed	25 participants
		83.912 (22.7633)
Change from Baseline	Number Analyzed	84 participants
		-0.809 (11.8056)

9. Secondary Outcome

Title	Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire
Description	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.
Time Frame	Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title		Migalastat HCl 150 mg
Arm/Group Description:		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).
Overall Number of Participants Analyzed		84
Mean (Standard Deviation); Unit of Measure: units on a scale
Baseline Physical Functioning	Number Analyzed	84 participants
		48.313 (9.2345)
End of Study Physical Functioning	Number Analyzed	75 participants
		46.415 (10.2466)
Change from Baseline Physical Functioning	Number Analyzed	75 participants
		-1.276 (7.1627)
Baseline Role Physical	Number Analyzed	84 participants
		46.197 (10.0761)
End of Study Role Physical	Number Analyzed	75 participants
		45.929 (10.5723)
Change from Baseline Role Physical	Number Analyzed	75 participants
		0.390 (7.3500)
Baseline Bodily Pain	Number Analyzed	84 participants
		46.889 (10.5983)
End of Study Bodily Pain	Number Analyzed	75 participants
		46.737 (10.3368)
Change from Baseline Bodily Pain	Number Analyzed	75 participants
		0.425 (8.1342)
Baseline General Health	Number Analyzed	84 participants
		44.016 (10.2497)
End of Study General Health	Number Analyzed	75 participants
		42.352 (10.1185)
Change from Baseline General Health	Number Analyzed	75 participants
		-0.811 (5.8672)
Baseline Vitality	Number Analyzed	84 participants
		46.375 (11.8451)
End of Study Vitality	Number Analyzed	75 participants
		45.429 (12.4340)
Change from Baseline Vitality	Number Analyzed	75 participants
		-0.674 (8.5076)
Baseline Social Functioning	Number Analyzed	84 participants
		47.433 (10.4985)
End of Study Social Functioning	Number Analyzed	75 participants
		47.648 (9.5165)
Change from Baseline Social Functioning	Number Analyzed	75 participants
		0.401 (8.4356)
Baseline Role Emotional	Number Analyzed	84 participants
		47.547 (10.3897)
End of Study Role Emotional	Number Analyzed	75 participants
		46.141 (10.9054)
Change from Baseline Role Emotional	Number Analyzed	75 participants
		-0.929 (10.6679)
Baseline Mental Health	Number Analyzed	84 participants
		48.501 (10.6878)
End of Study Mental Health	Number Analyzed	75 participants
		49.578 (10.5732)
Change from Baseline Mental Health	Number Analyzed	75 participants
		0.872 (6.2528)
Baseline Physical Component	Number Analyzed	84 participants
		46.184 (10.2268)
End of Study Physical Component	Number Analyzed	75 participants
		44.825 (10.4292)
Change from Baseline Physical Component	Number Analyzed	75 participants
		-0.508 (6.2494)
Baseline Mental Component	Number Analyzed	84 participants
		47.917 (11.5480)
End of Study Mental Component	Number Analyzed	75 participants
		48.175 (10.9497)
Change from Baseline Mental Component	Number Analyzed	75 participants
		0.187 (8.2714)

Adverse Events

Time Frame	Baseline to up to 4.4 years (includes safety follow-up)
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Migalastat HCl 150 mg
Arm/Group Description	Migalastat HCl 150 mg was administered orally every other day.
All-Cause Mortality
	Migalastat HCl 150 mg
	Affected / at Risk (%)
Total	0/84 (0.00%)
Serious Adverse Events
	Migalastat HCl 150 mg
	Affected / at Risk (%)
Total	26/84 (30.95%)
Cardiac disorders
Atrial fibrillation † 1	2/84 (2.38%)
Atrioventricular block complete † 1	1/84 (1.19%)
Left ventricular hypertrophy † 1	1/84 (1.19%)
Ventricular tachycardia † 1	1/84 (1.19%)
Eye disorders
Visual impairment † 1	1/84 (1.19%)
Gastrointestinal disorders
Abdominal pain † 1	1/84 (1.19%)
Abdominal pain upper † 1	1/84 (1.19%)
Abdominal wall haematoma † 1	1/84 (1.19%)
Barrett's oesophagus † 1	1/84 (1.19%)
Large intestine perforation † 1	1/84 (1.19%)
General disorders
Device malfunction † 1	1/84 (1.19%)
Hepatobiliary disorders
Biliary dyskinesia † 1	1/84 (1.19%)
Immune system disorders
Drug hypersensitivity † 1	1/84 (1.19%)
Infections and infestations
Appendicitis perforated † 1	1/84 (1.19%)
Biliary sepsis † 1	1/84 (1.19%)
Diverticulitis † 1	1/84 (1.19%)
Infective exacerbation of bronchiectasis † 1	1/84 (1.19%)
Pneumonia † 1	1/84 (1.19%)
Injury, poisoning and procedural complications
Rib fracture † 1	1/84 (1.19%)
Investigations
Heart rate increased † 1	1/84 (1.19%)
Metabolism and nutrition disorders
Dehydration † 1	2/84 (2.38%)
Diabetes mellitus † 1	1/84 (1.19%)
Lipomatosis † 1	1/84 (1.19%)
Musculoskeletal and connective tissue disorders
Arthritis † 1	1/84 (1.19%)
Osteoarthritis † 1	1/84 (1.19%)
Periarthritis † 1	1/84 (1.19%)
Tendon calcification † 1	1/84 (1.19%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Breast cancer † 1	2/84 (2.38%)
Invasive lobular breast carcinoma † 1	1/84 (1.19%)
Nervous system disorders
Convulsion † 1	1/84 (1.19%)
Embolic stroke † 1	1/84 (1.19%)
Headache † 1	1/84 (1.19%)
Neuralgia † 1	1/84 (1.19%)
Syncope † 1	1/84 (1.19%)
Transient ischaemic attack † 1	1/84 (1.19%)
Psychiatric disorders
Depression † 1	1/84 (1.19%)
Suicidal ideation † 1	1/84 (1.19%)
Renal and urinary disorders
Renal failure † 1	1/84 (1.19%)
Reproductive system and breast disorders
Uterine prolapse † 1	1/84 (1.19%)
Respiratory, thoracic and mediastinal disorders
Dyspnoea † 1	2/84 (2.38%)
Pneumothorax † 1	1/84 (1.19%)
Skin and subcutaneous tissue disorders
Subcutaneous emphysema † 1	1/84 (1.19%)
Vascular disorders
Air embolism † 1	1/84 (1.19%)
Hypertension † 1	1/84 (1.19%)
Hypotension † 1	1/84 (1.19%)
1 Term from vocabulary, MedDRA 16.1; † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Migalastat HCl 150 mg
	Affected / at Risk (%)
Total	74/84 (88.10%)
Cardiac disorders
Palpitations † 1	6/84 (7.14%)
Ear and labyrinth disorders
Tinnitus † 1	7/84 (8.33%)
Vertigo † 1	7/84 (8.33%)
Endocrine disorders
Hypothyroidism † 1	5/84 (5.95%)
Gastrointestinal disorders
Nausea † 1	10/84 (11.90%)
Diarrhoea † 1	9/84 (10.71%)
Abdominal pain † 1	7/84 (8.33%)
Abdominal pain upper † 1	6/84 (7.14%)
Constipation † 1	6/84 (7.14%)
Dyspepsia † 1	5/84 (5.95%)
Gastrooesophageal reflux disease † 1	5/84 (5.95%)
Vomiting † 1	5/84 (5.95%)
General disorders
Fatigue † 1	16/84 (19.05%)
Oedema peripheral † 1	12/84 (14.29%)
Pyrexia † 1	9/84 (10.71%)
Pain † 1	5/84 (5.95%)
Infections and infestations
Nasopharyngitis † 1	17/84 (20.24%)
Upper respiratory tract infection † 1	12/84 (14.29%)
Urinary tract infection † 1	12/84 (14.29%)
Influenza † 1	10/84 (11.90%)
Sinusitis † 1	9/84 (10.71%)
Bronchitis † 1	6/84 (7.14%)
Injury, poisoning and procedural complications
Overdose † 1	12/84 (14.29%)
Investigations
Albumin urine present † 1	6/84 (7.14%)
Blood uric acid increased † 1	6/84 (7.14%)
Protein urine present † 1	6/84 (7.14%)
Blood creatinine increased † 1	5/84 (5.95%)
Glomerular filtration rate decreased † 1	5/84 (5.95%)
Metabolism and nutrition disorders
Hypercholesterolaemia † 1	5/84 (5.95%)
Musculoskeletal and connective tissue disorders
Arthralgia † 1	16/84 (19.05%)
Pain in extremity † 1	14/84 (16.67%)
Musculoskeletal pain † 1	10/84 (11.90%)
Back pain † 1	9/84 (10.71%)
Muscle spasms † 1	7/84 (8.33%)
Tendonitis † 1	7/84 (8.33%)
Muscular weakness † 1	7/84 (8.33%)
Nervous system disorders
Paraesthesia † 1	13/84 (15.48%)
Headache † 1	12/84 (14.29%)
Dizziness † 1	10/84 (11.90%)
Hypoaesthesia † 1	10/84 (11.90%)
Migraine † 1	5/84 (5.95%)
Neuralgia † 1	5/84 (5.95%)
Psychiatric disorders
Depression † 1	8/84 (9.52%)
Insomnia † 1	6/84 (7.14%)
Renal and urinary disorders
Proteinuria † 1	12/84 (14.29%)
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain † 1	7/84 (8.33%)
Asthma † 1	6/84 (7.14%)
Cough † 1	6/84 (7.14%)
Dyspnoea † 1	6/84 (7.14%)
Dyspnoea exertional † 1	6/84 (7.14%)
Vascular disorders
Hypotension † 1	5/84 (5.95%)
1 Term from vocabulary, MedDRA 16.1; † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

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.

Results Point of Contact

• Name/Title: Medical Affairs
• Organization: Amicus Therapeutics
• Phone: +1-877-426-4287 (877-4-AMICUS)
• EMail: MedInfoUSA@amicusrx.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Narita I, Ohashi T, Sakai N, Hamazaki T, Skuban N, Castelli JP, Lagast H, Barth JA. Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study. Clin Exp Nephrol. 2020 Feb;24(2):157-166. doi: 10.1007/s10157-019-01810-w. Epub 2019 Dec 30.

• Responsible Party: Amicus Therapeutics
• ClinicalTrials.gov Identifier: NCT02194985
• Other Study ID Numbers: AT1001-042; 2014-002701-38 ( EudraCT Number )
• First Submitted: July 17, 2014
• First Posted: July 21, 2014
• Results First Submitted: October 21, 2020
• Results First Posted: December 21, 2020
• Last Update Posted: December 21, 2020