Trial · NCT01458119
Open-Label Phase 3 Long-Term Safety Study of Migalastat
NCT01458119, Phase 3, Terminated, Amicus Therapeutics; results first posted 2018-10-02
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Open-Label Phase 3 Long-Term Safety Study of Migalastat. Trials, record NCT01458119. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01458119
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-Label Phase 3 Long-Term Safety Study of Migalastat
- NCT number
- NCT01458119
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 85 participants, actual
- Lead sponsor
- Amicus Therapeutics
- Conditions
- Fabry Disease
- Interventions
- migalastat hydrochloride
- First posted
- 2011-10-24
- Last updated
- 2018-10-02
- Other registries' IDs
- eudract:2011-004800-40
- Results first posted
- 2018-10-02
- Limitations and caveats
The study was discontinued for logistical reasons and not due to either safety concerns or lack of efficacy. The investigators were offered participation in a similar long-term migalastat study for participants ongoing at discontinuation.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) | Baseline to End of Follow-up (30 days after the end of this 42-month study), with AE reporting occurring at each study visit, which occurred once every 6 months. | An adverse event (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 the time of reporting; visits typically occurred every 6 months. A TEAE was defined as an AE starting on or after the first study drug administration date. Serious AEs were life-threatening or resulted in death, persistent or significant incapacitation, inpatient or prolonged hospitalization, or a congenital anomaly. The criteria for AE severity were: Mild: minimal discomfort, does not interfere with normal everyday activities; Moderate: sufficiently discomforting, interferes with normal everyday activities; Severe: prevents normal everyday activities. The number of participants experiencing TEAEs is presented for those who received migalastat treatment. 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, Every 6 m until the End of Study (42 m) | The annualized rate of change of 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 \* (Serum Creatinine)\^-1.154 \* (Age)\^-0.203 \* 1.212 (if black or African American) \* 0.742 (if female); eGFR \[CKD-EPI\] = 141 \* min(serum creatinine/kappa,1)\^alpha \* max(serum creatinine/kappa, 1)\^-1.209 \* 0.993\^age \* 1.1018(if female) \* 1.159(if black or African American), where kappa is 0.7 for females and 0.9 for males, alpha is -0.329 for females and -0.411 for males, min is minimum of serum creatinine/kappa or 1, and max is the maximum of serum creatinine/kappa or 1. The number of participants with at least a Baseline and a post-Baseline value are presented. |
Source ClinicalTrials.gov
Outcomes2
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 Treatment-emergent Adverse Events (TEAEs) | Baseline to End of Follow-up (30 days after the end of this 42-month study), with AE reporting occurring at each study visit, which occurred once every 6 months. | Safety Population: All participants who received at least 1 dose of study drug after they enrolled into this open-label extension study. | participants | NUMBER |
| SECONDARY | Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR) | Baseline, Every 6 m until the End of Study (42 m) | ITT-Amenable Population: All participants who received at least 1 dose of study drug. Participants with mutant forms of α-Galactosidase (Gal) A determined to be amenable to migalastat based on the GLP-HEK assay are referred to as with amenable mutations. Number of participants analyzed are those with at least a Baseline and a post-Baseline value. | milliliters/minute/1.73 meters^2 | MEAN |
Source ClinicalTrials.gov
Results by group11
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 Treatment-emergent Adverse Events (TEAEs) | Migalastat | Participants with at least 1 TEAE | 74 | participants | |
| Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) | Migalastat | Participants with at least 1 serious TEAE | 22 | participants | |
| Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) | Migalastat | Participants discontinued due to TEAEs | 1 | participants | |
| Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) | Migalastat | Participants with adverse events leading to death | 2 | participants | |
| Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) | Migalastat | Participants with TEAEs related to study drug | 14 | participants | |
| Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) | Migalastat | Participants with TEAEs unrelated to study drug | 60 | participants | |
| Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) | Migalastat | Participants with at least 1 mild TEAE | 22 | participants | |
| Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) | Migalastat | Participants with at least 1 moderate TEAE | 40 | participants | |
| Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) | Migalastat | Participants with at least 1 severe TEAE | 12 | participants | |
| Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR) | Migalastat ITT-Amenable Population | EGFR [CKD-EPI] | 1.54 | 95% Confidence Interval -1.63 to 4.71 | milliliters/minute/1.73 meters^2 |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 results by groupParticipant flow7
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Migalastat | 85 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Migalastat | 85 | |
| Overall Study | Safety Population | Migalastat | 85 | |
| Overall Study | Intent to Treat (ITT) Population | Migalastat | 85 | |
| Overall Study | ITT-Amenable Population | Migalastat | 68 | |
| Overall Study | COMPLETED | Migalastat | 65 | |
| Overall Study | NOT COMPLETED | Migalastat | 20 |
Source ClinicalTrials.gov
Adverse events52
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 | Hypertension | Vascular disorders | Migalastat | 6 | 85 | |
| other | Contusion | Injury, poisoning and procedural complications | Migalastat | 5 | 85 | |
| other | Atrial fibrillation | Cardiac disorders | Migalastat | 7 | 85 | |
| other | Palpitations | Cardiac disorders | Migalastat | 5 | 85 | |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Migalastat | 6 | 85 | |
| other | Headache | Nervous system disorders | Migalastat | 11 | 85 | |
| other | Dizziness | Nervous system disorders | Migalastat | 9 | 85 | |
| other | Paraesthesia | Nervous system disorders | Migalastat | 5 | 85 | |
| other | Fatigue | General disorders | Migalastat | 9 | 85 | |
| other | Oedema peripheral | General disorders | Migalastat | 6 | 85 |
1 to 10 of 52Source ClinicalTrials.gov
See all 52 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Migalastat | ||
| Total, other adverse events | Migalastat | 57 | 85 |
| Total, serious adverse events | Migalastat | 22 | 85 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 27509102 | 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/NCT01458119
GET https://conductscience.org/api/trials/v1/NCT01458119/events?page=2