Trial · NCT02194985
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
NCT02194985, Phase 3, Completed, Amicus Therapeutics; results first posted 2020-12-21
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease. Trials, record NCT02194985. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02194985
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
- NCT number
- NCT02194985
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 84 participants, actual
- Lead sponsor
- Amicus Therapeutics
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl 150 mg
- First posted
- 2014-07-21
- Last updated
- 2020-12-21
- Other registries' IDs
- eudract:2014-002701-38
- Results first posted
- 2020-12-21
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number Of Participants Experiencing Adverse Events (AEs) | Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years | 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. |
| Secondary | Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR) | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. |
| Secondary | Change From Baseline In eGFR At End Of Study | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. |
| Secondary | Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. |
| Secondary | Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. |
| Secondary | Change From Baseline In 24-hour Urine Protein To End Of Study | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. |
| Secondary | Change From Baseline In Left Ventricular Mass (LVM) To End Of Study | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. |
| Secondary | Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. |
| Secondary | Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number Of Participants Experiencing Adverse Events (AEs) | Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years | Safety Population: All participants who received at least 1 dose of study drug after they enrolled into this open-label extension study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR) | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | Intent-to-Treat Population: all participants who took at least 1 dose of the study drug after they had enrolled into this study. | mL/min/1.73 m^2 | MEAN |
| SECONDARY | Change From Baseline In eGFR At End Of Study | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. | mL/min/1.73 m^2 | MEAN |
| SECONDARY | Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. | nmol/L | MEAN |
| SECONDARY | Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. | nmol/hr/mg | MEAN |
| SECONDARY | Change From Baseline In 24-hour Urine Protein To End Of Study | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. | mg/day | MEAN |
| SECONDARY | Change From Baseline In Left Ventricular Mass (LVM) To End Of Study | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. | gram | MEAN |
| SECONDARY | Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. | g/m^2 | MEAN |
| SECONDARY | Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire | Baseline to approximately 30 days after last treatment, median duration of 3.1 years | 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. | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group62
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number Of Participants Experiencing Adverse Events (AEs) | Migalastat HCl 150 mg | Participants with at least 1 AE | 80 | Participants | |
| Number Of Participants Experiencing Adverse Events (AEs) | Migalastat HCl 150 mg | Participants with at least 1 serious AE | 26 | Participants | |
| Number Of Participants Experiencing Adverse Events (AEs) | Migalastat HCl 150 mg | Participants discontinued due to AEs | 1 | Participants | |
| Number Of Participants Experiencing Adverse Events (AEs) | Migalastat HCl 150 mg | Participants with AEs leading to death | 0 | Participants | |
| Number Of Participants Experiencing Adverse Events (AEs) | Migalastat HCl 150 mg | Participants with AEs related to study drug | 24 | Participants | |
| Number Of Participants Experiencing Adverse Events (AEs) | Migalastat HCl 150 mg | Participants with AEs unrelated to study drug | 56 | Participants | |
| Number Of Participants Experiencing Adverse Events (AEs) | Migalastat HCl 150 mg | Participants with at least 1 mild AE | 19 | Participants | |
| Number Of Participants Experiencing Adverse Events (AEs) | Migalastat HCl 150 mg | Participants with at least 1 moderate AE | 46 | Participants | |
| Number Of Participants Experiencing Adverse Events (AEs) | Migalastat HCl 150 mg | Participants with at least 1 severe AE | 15 | Participants | |
| Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR) | Migalastat HCl 150 mg | eGFR[MDRD] | -1.6107 | Standard Deviation 5.62761 | mL/min/1.73 m^2 |
1 to 10 of 62Source ClinicalTrials.gov
See all 62 results by groupParticipant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Migalastat HCl 150 mg | 84 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Migalastat HCl 150 mg | 84 | |
| Overall Study | COMPLETED | Migalastat HCl 150 mg | 73 | |
| Overall Study | NOT COMPLETED | Migalastat HCl 150 mg | 11 |
Source ClinicalTrials.gov
Adverse events96
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Palpitations | Cardiac disorders | Migalastat HCl 150 mg | 6 | 84 | |
| other | Tinnitus | Ear and labyrinth disorders | Migalastat HCl 150 mg | 7 | 84 | |
| other | Vertigo | Ear and labyrinth disorders | Migalastat HCl 150 mg | 7 | 84 | |
| other | Hypothyroidism | Endocrine disorders | Migalastat HCl 150 mg | 5 | 84 | |
| other | Nausea | Gastrointestinal disorders | Migalastat HCl 150 mg | 10 | 84 | |
| other | Diarrhoea | Gastrointestinal disorders | Migalastat HCl 150 mg | 9 | 84 | |
| other | Abdominal pain | Gastrointestinal disorders | Migalastat HCl 150 mg | 7 | 84 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Migalastat HCl 150 mg | 6 | 84 | |
| other | Constipation | Gastrointestinal disorders | Migalastat HCl 150 mg | 6 | 84 | |
| other | Dyspepsia | Gastrointestinal disorders | Migalastat HCl 150 mg | 5 | 84 |
1 to 10 of 96Source ClinicalTrials.gov
See all 96 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Migalastat HCl 150 mg | 0 | 84 |
| Total, other adverse events | Migalastat HCl 150 mg | 74 | 84 |
| Total, serious adverse events | Migalastat HCl 150 mg | 26 | 84 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 31889231 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02194985
GET https://conductscience.org/api/trials/v1/NCT02194985/results?page=2