Trial · NCT00925301
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
NCT00925301, Phase 3, Completed, Amicus Therapeutics; results first posted 2018-10-30
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease. Trials, record NCT00925301. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00925301
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
- NCT number
- NCT00925301
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 67 participants, actual
- Lead sponsor
- Amicus Therapeutics
- Conditions
- Fabry Disease
- Interventions
- migalastat hydrochloride, Placebo
- First posted
- 2009-06-22
- Last updated
- 2018-10-30
- Other registries' IDs
- eudract:2009-013459-31
- Results first posted
- 2018-10-30
- Limitations and caveats
During Phase 3, prior to unblinding, the assay used for enrollment was validated (GLP HEK assay). Mutant forms of α-Gal A that met assay criteria were categorized as amenable; additional analyses were subsequently performed on this target population.
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions | Baseline, Month 6 | Renal biopsies were taken at Baseline and Month 6 (Stage 1). The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Baseline and Month 6. Assessments were made using digital images. A responder was defined as a participant with a ≥50% reduction from Baseline to Month 6 in the average number of kidney IC GL-3 inclusions. |
| Secondary | Percent Change In Kidney IC GL-3 Inclusions From Baseline To Month 6 | Baseline, Month 6 | Renal biopsies were taken at Baseline and Month 6. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Baseline and Month 6. Assessments were made using digital images. |
| Secondary | Change From Baseline Through Month 24 In Urine GL-3 Levels | Baseline, Months 6, 12, and 24 | The effect of migalastat versus placebo on urine GL-3 levels was measured by liquid chromatography-mass spectrometry/mass spectrometry. The 24-hour urine samples were collected at Baseline, Month 6 (Stage 1), Month 12 (Stage 2), and Month 24 (OLE). Results are presented as changes in nanograms (ng)/mg creatinine from Baseline to the end of the 3 stages. |
| Other | Change From Month 6 To Month 12 In Average Number Of Kidney IC GL-3 Inclusions | Month 6, Month 12 | Renal biopsies were taken at Month 6 and Month 12. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Month 6 and Month 12. Assessments were made using digital images. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions | Baseline, Month 6 | ITT: All randomized participants regardless of their participation in the study beyond randomization. As per the statistical analysis plan, analysis excluded participants who were missing baseline kidney biopsy results. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percent Change In Kidney IC GL-3 Inclusions From Baseline To Month 6 | Baseline, Month 6 | ITT: All randomized participants regardless of their participation in the study beyond randomization. | percent change | MEAN |
| SECONDARY | Change From Baseline Through Month 24 In Urine GL-3 Levels | Baseline, Months 6, 12, and 24 | Stage 1: ITT, all randomized participants regardless of participation in the study beyond randomization. Stage 2: participants who completed Stage 1 and entered Stage 2. OLE: participants who completed Stage 2 and entered the optional OLE. Once assay and sample issues were identified, later samples were not further analyzed; all data was listed. | ng/mg creatinine | MEAN |
| OTHER_PRE_SPECIFIED | Change From Month 6 To Month 12 In Average Number Of Kidney IC GL-3 Inclusions | Month 6, Month 12 | mITT with amenable mutations: Randomized participants who switched from placebo to migalastat during Stage 2, received at least 1 dose of study drug, and underwent a renal biopsy at both Baseline and Month 6. Amenable mutations are mutant forms of α-galactosidase A (α Gal-A) amenable to migalastat. Amenable mutations based on the GLP HEK assay. | kidney IC GL-3 inclusions | LEAST_SQUARES_MEAN |
| POST_HOC | Change From Baseline To Month 6 In Average Number Of Kidney IC GL-3 Inclusions | Baseline, Month 6 | ITT-amenable: Randomized participants with amenable mutations who received study drug during Stage 1. Amenable mutations are mutant forms of α Gal-A amenable to migalastat. Amenable mutations based on the GLP HEK assay. Participants were analyzed according to their original randomized treatment group. | kidney IC GL-3 inclusions | MEAN |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions | Migalastat | Responder | 13 | Participants | |
| Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions | Placebo | Responder | 9 | Participants | |
| Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions | Migalastat | Non-Responder | 19 | Participants | |
| Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions | Placebo | Non-Responder | 23 | Participants | |
| Percent Change In Kidney IC GL-3 Inclusions From Baseline To Month 6 | Migalastat | -7.948 | Standard Deviation 105.2736 | percent change | |
| Percent Change In Kidney IC GL-3 Inclusions From Baseline To Month 6 | Placebo | 12.985 | Standard Deviation 90.5131 | percent change | |
| Change From Baseline Through Month 24 In Urine GL-3 Levels | Migalastat-Migalastat | Stage 1 | -234.80 | Standard Deviation 853.451 | ng/mg creatinine |
| Change From Baseline Through Month 24 In Urine GL-3 Levels | Placebo-Migalastat | Stage 1 | -186.24 | Standard Deviation 957.119 | ng/mg creatinine |
| Change From Baseline Through Month 24 In Urine GL-3 Levels | Migalastat-Migalastat | Stage 2 | -179.63 | Standard Deviation 699.157 | ng/mg creatinine |
| Change From Baseline Through Month 24 In Urine GL-3 Levels | Placebo-Migalastat | Stage 2 | -537.95 | Standard Deviation 1169.883 | ng/mg creatinine |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow56
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| 6-Month Double-blind (Stage 1) | STARTED | Migalastat (0-6 Months) | 34 | |
| 6-Month Double-blind (Stage 1) | STARTED | Placebo (0-6 Months) | 33 | |
| 6-Month Double-blind (Stage 1) | STARTED | Migalastat (>6-12 Months) | 0 | |
| 6-Month Double-blind (Stage 1) | STARTED | Migalastat (>12-24 Months) | 0 | |
| 6-Month Double-blind (Stage 1) | Received at Least 1 Dose of Study Drug | Migalastat (0-6 Months) | 34 | |
| 6-Month Double-blind (Stage 1) | Received at Least 1 Dose of Study Drug | Placebo (0-6 Months) | 33 | |
| 6-Month Double-blind (Stage 1) | Received at Least 1 Dose of Study Drug | Migalastat (>6-12 Months) | 0 | |
| 6-Month Double-blind (Stage 1) | Received at Least 1 Dose of Study Drug | Migalastat (>12-24 Months) | 0 | |
| 6-Month Double-blind (Stage 1) | Intent-to-Treat (ITT) | Migalastat (0-6 Months) | 34 | |
| 6-Month Double-blind (Stage 1) | Intent-to-Treat (ITT) | Placebo (0-6 Months) | 33 |
1 to 10 of 56Source ClinicalTrials.gov
See all 56 participant flowAdverse events264
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 | Fatigue | General disorders | Migalastat (0-6 Months) | 4 | 34 | |
| other | Fatigue | General disorders | Placebo (0-6 Months) | 4 | 33 | |
| other | Fatigue | General disorders | All Migalastat (>6-12 Months) | 2 | 63 | |
| other | Fatigue | General disorders | All Migalastat (>12-24 Months) | 3 | 57 | |
| other | Pyrexia | General disorders | Migalastat (0-6 Months) | 4 | 34 | |
| other | Pyrexia | General disorders | Placebo (0-6 Months) | 1 | 33 | |
| other | Pyrexia | General disorders | All Migalastat (>6-12 Months) | 1 | 63 | |
| other | Pyrexia | General disorders | All Migalastat (>12-24 Months) | 0 | 57 | |
| other | Depression | Psychiatric disorders | Migalastat (0-6 Months) | 1 | 34 | |
| other | Depression | Psychiatric disorders | Placebo (0-6 Months) | 0 | 33 |
1 to 10 of 264Source ClinicalTrials.gov
See all 264 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Migalastat (0-6 Months) | ||
| Total, all-cause mortality | Placebo (0-6 Months) | ||
| Total, all-cause mortality | All Migalastat (>6-12 Months) | ||
| Total, all-cause mortality | All Migalastat (>12-24 Months) | ||
| Total, other adverse events | Migalastat (0-6 Months) | 31 | 34 |
| Total, other adverse events | Placebo (0-6 Months) | 30 | 33 |
| Total, other adverse events | All Migalastat (>6-12 Months) | 50 | 63 |
| Total, other adverse events | All Migalastat (>12-24 Months) | 46 | 57 |
| Total, serious adverse events | Migalastat (0-6 Months) | 2 | 34 |
| Total, serious adverse events | Placebo (0-6 Months) | 4 | 33 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers5
The papers the trial lists on ClinicalTrials.gov.
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/NCT00925301
GET https://conductscience.org/api/trials/v1/NCT00925301/flow?page=2