Trial · NCT01218659
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
NCT01218659, Phase 3, Completed, Amicus Therapeutics; results first posted 2018-11-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease. Trials, record NCT01218659. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01218659
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
- NCT number
- NCT01218659
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 68 participants, actual
- Lead sponsor
- Amicus Therapeutics
- Conditions
- Fabry Disease
- Interventions
- migalastat hydrochloride, agalsidase
- First posted
- 2010-10-11
- Last updated
- 2018-11-01
- Other registries' IDs
- eudract:2010-022636-37
- Results first posted
- 2018-11-01
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate | Baseline to Month 18 | To assess renal function, measured glomerular filtration rate (GFR) was measured by the plasma clearance of unlabeled iohexol (mGFR-iohexol), a non-ionic contrast agent. The annualized rate of change in mGFR-iohexol from Baseline to Month 18 was analyzed using an analysis of covariance (ANCOVA) model with the following factors as covariates: treatment group, sex, age, Baseline GFR (mGFR-iohexol), and Baseline 24-hour (hr) urine protein. A threshold of \<2.2 milliliter (mL)/minute (min)/1.73 meter squared (m\^2)/year was established to compare migalastat to ERT. This difference of 2.2 mL/min/1.73 m2/year is based on the smallest expected rate of decline in estimated glomerular filtration rate (eGFR) for participants treated with agalsidase alfa for 18 months. |
| Primary | Annualized Rate Of Change From Baseline To Month 18 In eGFR | Baseline to Month 18 | The eGFR assessed by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation was calculated using the following: eGFR-CKD-EPI = 141 x min (Serum Creatinine/κ,1)\^(α) x max(Serum Creatinine/κ,1)\^(-1.209) x 0.993\^(Age) x 1.1018 (if female) x 1.159 (if African American or black) where: κ 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 Serum Creatinine/κ or 1; max indicates the maximum of Serum Creatinine/κ or 1. The annualized rate of change in eGFR-CKD-EPI from Baseline to Month 18 was analyzed using an ANCOVA model with the following factors as covariates: treatment group, sex, age, Baseline GFR (eGFR-CKD-EPI), and Baseline 24-hr urine protein. A threshold of \<2.2 mL/min/1.73m\^2/year was established to compare migalastat to ERT. This difference of 2.2 mL/min/1.73 m2/year is based on the smallest expected rate of decline in eGFR for participants treated with agalsidase alfa for 18 months. |
| Secondary | Annualized Rate Of Change From Baseline To Month 18 In eGFR By The Modification Of Diet In Renal Disease Equation | Baseline to Month 18 | The GFR estimated by the Modification Of Diet In Renal Disease equation (eGFR-MDRD) was calculated using the following equation: eGFR-MDRD = 175 x (Serum Creatinine)\^(-1.154) x (Age)\^(-0.203) x 1.212 (if participant's race is black or African American) x 0.742 (if participant is female). The eGFR-MDRD from Baseline to Month 18 was analyzed using an ANCOVA model with the following factors as covariates: treatment group, sex, age, Baseline GFR (eGFR-CKD-EPI), and Baseline 24-hr urine protein. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate | Baseline to Month 18 | mITT Population: All randomized participants with an α Gal-A mutation that is amenable to migalastat, based on the GLP HEK assay, who received at least 1 dose of study drug, had Baseline and postbaseline mGFR-iohexol values, and postbaseline measure of the estimated GFR using the CKD-EPI equation. | mL/min/1.73 m^2/year | LEAST_SQUARES_MEAN |
| PRIMARY | Annualized Rate Of Change From Baseline To Month 18 In eGFR | Baseline to Month 18 | mITT Population: All randomized participants with an α Gal-A mutation that is amenable to migalastat, based on the GLP HEK assay, who received at least 1 dose of study drug, had Baseline and postbaseline mGFR-iohexol values, and postbaseline measure of the estimated GFR using the CKD-EPI equation. | mL/min/1.73 m^2/year | LEAST_SQUARES_MEAN |
| SECONDARY | Annualized Rate Of Change From Baseline To Month 18 In eGFR By The Modification Of Diet In Renal Disease Equation | Baseline to Month 18 | mITT Population: All randomized participants with an α Gal-A mutation that is amenable to migalastat, based on the GLP HEK assay, who received at least 1 dose of study drug, had Baseline and postbaseline mGFR-iohexol values, and postbaseline measure of the estimated GFR using the CKD-EPI equation. | mL/min/1.73 m^2/year | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate | Migalastat (0-18 Months) | -4.35 | 95% Confidence Interval -7.65 to -1.06 | mL/min/1.73 m^2/year | |
| Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate | ERT (0-18 Months) | -3.24 | 95% Confidence Interval -7.81 to 1.33 | mL/min/1.73 m^2/year | |
| Annualized Rate Of Change From Baseline To Month 18 In eGFR | Migalastat (0-18 Months) | -0.40 | 95% Confidence Interval -2.27 to 1.48 | mL/min/1.73 m^2/year | |
| Annualized Rate Of Change From Baseline To Month 18 In eGFR | ERT (0-18 Months) | -1.03 | 95% Confidence Interval -3.64 to 1.58 | mL/min/1.73 m^2/year | |
| Annualized Rate Of Change From Baseline To Month 18 In eGFR By The Modification Of Diet In Renal Disease Equation | Migalastat (0-18 Months) | -1.51 | 95% Confidence Interval -3.43 to 0.40 | mL/min/1.73 m^2/year | |
| Annualized Rate Of Change From Baseline To Month 18 In eGFR By The Modification Of Diet In Renal Disease Equation | ERT (0-18 Months) | -1.53 | 95% Confidence Interval -4.20 to 1.13 | mL/min/1.73 m^2/year |
Source ClinicalTrials.gov
Participant flow27
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| 18-Month Randomized Period | STARTED | Migalastat (0-18 Months) | 36 | |
| 18-Month Randomized Period | STARTED | ERT (0-18 Months) | 24 | |
| 18-Month Randomized Period | STARTED | All Migalastat (0-30 Months) | 0 | |
| 18-Month Randomized Period | Safety Population | Migalastat (0-18 Months) | 36 | |
| 18-Month Randomized Period | Safety Population | ERT (0-18 Months) | 21 | |
| 18-Month Randomized Period | Safety Population | All Migalastat (0-30 Months) | 0 | |
| 18-Month Randomized Period | COMPLETED | Migalastat (0-18 Months) | 34 | |
| 18-Month Randomized Period | COMPLETED | ERT (0-18 Months) | 18 | |
| 18-Month Randomized Period | COMPLETED | All Migalastat (0-30 Months) | 0 | |
| 18-Month Randomized Period | NOT COMPLETED | Migalastat (0-18 Months) | 2 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 participant flowAdverse events240
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 (0-18 Months) | 2 | 36 | |
| other | Palpitations | Cardiac disorders | ERT (0-18 Months) | 1 | 21 | |
| other | Palpitations | Cardiac disorders | All Migalastat (0-30 Months) | 5 | 51 | |
| other | Tinnitus | Ear and labyrinth disorders | Migalastat (0-18 Months) | 3 | 36 | |
| other | Tinnitus | Ear and labyrinth disorders | ERT (0-18 Months) | 0 | 21 | |
| other | Tinnitus | Ear and labyrinth disorders | All Migalastat (0-30 Months) | 5 | 51 | |
| other | Diarrhoea | Gastrointestinal disorders | Migalastat (0-18 Months) | 5 | 36 | |
| other | Diarrhoea | Gastrointestinal disorders | ERT (0-18 Months) | 2 | 21 | |
| other | Diarrhoea | Gastrointestinal disorders | All Migalastat (0-30 Months) | 11 | 51 | |
| other | Nausea | Gastrointestinal disorders | Migalastat (0-18 Months) | 5 | 36 |
1 to 10 of 240Source ClinicalTrials.gov
See all 240 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Migalastat (0-18 Months) | ||
| Total, all-cause mortality | ERT (0-18 Months) | ||
| Total, all-cause mortality | All Migalastat (0-30 Months) | ||
| Total, other adverse events | Migalastat (0-18 Months) | 34 | 36 |
| Total, other adverse events | ERT (0-18 Months) | 20 | 21 |
| Total, other adverse events | All Migalastat (0-30 Months) | 50 | 51 |
| Total, serious adverse events | Migalastat (0-18 Months) | 7 | 36 |
| Total, serious adverse events | ERT (0-18 Months) | 7 | 21 |
| Total, serious adverse events | All Migalastat (0-30 Months) | 16 | 51 |
Source ClinicalTrials.gov
Papers6
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/NCT01218659
GET https://conductscience.org/api/trials/v1/NCT01218659/flow?page=2