Trial · NCT02228460
Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease
NCT02228460, Phase 2, Completed, Genzyme, a Sanofi Company; results first posted 2019-12-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease. Trials, record NCT02228460. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02228460
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease
- NCT number
- NCT02228460
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 11 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Fabry Disease
- Interventions
- GZ/SAR402671
- First posted
- 2014-08-29
- Last updated
- 2019-12-17
- Results first posted
- 2019-12-17
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | Baseline, Week 26 | Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26. |
| Primary | Mean Change From Baseline at Week 26 in Skin GL-3 Score in Superficial Skin Capillary Endothelium | Baseline, Week 26 | Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Change from Baseline in GL-3 score was obtained by subtracting Baseline value from post-baseline value at Week 26. A negative change from Baseline indicates less severe condition at Week 26. |
| Secondary | Change From Baseline in Plasma GL-3 Concentration at Week 26 | Baseline, Week 26 | Change from Baseline in plasma GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in plasma was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method. |
| Secondary | Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso-GL-3) Concentration at Week 26 | Baseline, Week 26 | Change from Baseline in plasma Lyso-GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of lyso-GL-3 in plasma was determined using a validated LC-MS/MS method. |
| Secondary | Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration at Week 26 | Baseline, Week 26 | Change from Baseline in plasma GL-1 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-1 in plasma was determined using a validated LC-MS/MS method. |
| Secondary | Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Endothelial Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | Baseline, Week 26 | Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26. |
| Secondary | Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Smooth Muscle Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | Baseline, Week 26 | Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26. |
| Secondary | Change From Baseline at Week 26 in Skin GL-3 Score in Perineurium Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | Baseline, Week 26 | Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26. |
| Secondary | Change From Baseline in Urine Globotriaosylceramide (GL-3) Concentration at Week 26 | Baseline, Week 26 | Change from Baseline in urine GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in urine was determined using a validated LC-MS/MS method. |
| Secondary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | From Baseline up to 212 days | Any untoward medical occurrence in a participant who received study drug was considered an adverse event (AE) without regard to possibility of causal relationship with this treatment. TEAEs were defined as AEs that developed or worsened during on-treatment period (period from the first administration of study drug through the last administration of the study drug plus 30 days or end of study participation for participant, whichever occurs first). |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | Baseline, Week 26 | Analysis was performed on full analysis set (FAS) that included all participants who received at least 1 dose of study treatment. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Mean Change From Baseline at Week 26 in Skin GL-3 Score in Superficial Skin Capillary Endothelium | Baseline, Week 26 | Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Plasma GL-3 Concentration at Week 26 | Baseline, Week 26 | Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26. | mcg/mL | MEAN |
| SECONDARY | Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso-GL-3) Concentration at Week 26 | Baseline, Week 26 | Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26. | ng/mL | MEAN |
| SECONDARY | Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration at Week 26 | Baseline, Week 26 | Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26. | mcg/mL | MEAN |
| SECONDARY | Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Endothelial Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | Baseline, Week 26 | Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Smooth Muscle Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | Baseline, Week 26 | Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline at Week 26 in Skin GL-3 Score in Perineurium Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | Baseline, Week 26 | Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Urine Globotriaosylceramide (GL-3) Concentration at Week 26 | Baseline, Week 26 | Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26. | mg/mmol Cr | MEAN |
| SECONDARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | From Baseline up to 212 days | Analysis was performed on safety population which included all enrolled participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group35
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | GZ/SAR402671 | Baseline Score: 1 / Week 26 Score: 1 | 4 | Participants | |
Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | GZ/SAR402671 | Baseline Score: 1 / Week 26 Score: 2 | 1 | Participants | |
Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | GZ/SAR402671 | Baseline Score: 2 / Week 26 Score: 1 | 3 | Participants | |
Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | GZ/SAR402671 | Baseline Score: 2 / Week 26 Score: 2 | 1 | Participants | |
| Mean Change From Baseline at Week 26 in Skin GL-3 Score in Superficial Skin Capillary Endothelium | GZ/SAR402671 | -0.22 | 95% Confidence Interval -0.73 to 0.29 | score on a scale | |
| Change From Baseline in Plasma GL-3 Concentration at Week 26 | GZ/SAR402671 | -3.62 | Standard Deviation 1.07 | mcg/mL | |
| Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso-GL-3) Concentration at Week 26 | GZ/SAR402671 | -30.99 | Standard Deviation 22.83 | ng/mL | |
| Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration at Week 26 | GZ/SAR402671 | -3.26 | Standard Deviation 1.43 | mcg/mL | |
| Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Endothelial Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | GZ/SAR402671 | Baseline Score: 1 / Week 26 Score: 1 | 1 | Participants | |
| Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Endothelial Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26 | GZ/SAR402671 | Baseline Score: 2 / Week 26 Score: 1 | 2 | Participants |
1 to 10 of 35Source ClinicalTrials.gov
See all 35 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | GZ/SAR402671 | 11 | |
| Overall Study | COMPLETED | GZ/SAR402671 | 9 | |
| Overall Study | NOT COMPLETED | GZ/SAR402671 | 2 |
Source ClinicalTrials.gov
Adverse events54
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 | Atrial fibrillation | Cardiac disorders | GZ/SAR402671 | 1 | 11 | |
| other | Tinnitus | Ear and labyrinth disorders | GZ/SAR402671 | 2 | 11 | |
| other | Blepharitis | Eye disorders | GZ/SAR402671 | 1 | 11 | |
| other | Chalazion | Eye disorders | GZ/SAR402671 | 1 | 11 | |
| other | Eye pain | Eye disorders | GZ/SAR402671 | 1 | 11 | |
| other | Lacrimation increased | Eye disorders | GZ/SAR402671 | 1 | 11 | |
| other | Lenticular opacities | Eye disorders | GZ/SAR402671 | 1 | 11 | |
| other | Retinal vascular disorder | Eye disorders | GZ/SAR402671 | 1 | 11 | |
| other | Vision blurred | Eye disorders | GZ/SAR402671 | 1 | 11 | |
| other | Constipation | Gastrointestinal disorders | GZ/SAR402671 | 1 | 11 |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | GZ/SAR402671 | 0 | 11 |
| Total, other adverse events | GZ/SAR402671 | 9 | 11 |
| Total, serious adverse events | GZ/SAR402671 | 3 | 11 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 36481125 | 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.
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