Trial · NCT00446550
A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
NCT00446550, Phase 2, Completed, Amicus Therapeutics; results first posted 2018-08-15
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease. Trials, record NCT00446550. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00446550
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
- NCT number
- NCT00446550
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 19 participants, actual
- Lead sponsor
- Amicus Therapeutics
- Conditions
- Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
- Interventions
- afegostat tartrate
- First posted
- 2007-03-13
- Last updated
- 2018-08-15
- Results first posted
- 2018-08-15
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 Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) | Day 1 (after dosing) through Day 183 | A TEAE was defined as any adverse event (AE) with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe AE defined as an AE that was incapacitating and required medical intervention. The number of participants who experienced 1 or more severe TEAEs after dosing on Day 1 through the end of follow-up (Day 183) is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module. |
| Secondary | Change From Baseline To End Of Treatment In β-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC) | Baseline, Day 169 | GCase is a biomarker used to assess the PD effects of afegostat tartrate. Blood samples were collected to assess GCase levels in WBC. The baseline value was defined as the last non-missing value before the start of study drug. |
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 Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) | Day 1 (after dosing) through Day 183 | Safety Population: all participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline To End Of Treatment In β-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC) | Baseline, Day 169 | PD Population: all participants who were included in the Safety Population and had a baseline and at least 1 post-baseline PD measurement. | picomole/minute/mg | MEAN |
Source ClinicalTrials.gov
Results by group4
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) | Afegostat Tartrate Treatment Regimen 1 | 2 | Participants | ||
| Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) | Afegostat Tartrate Treatment Regimen 2 | 0 | Participants | ||
| Change From Baseline To End Of Treatment In β-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC) | Afegostat Tartrate Treatment Regimen 1 | 10.2 | Standard Deviation 15.87 | picomole/minute/mg | |
| Change From Baseline To End Of Treatment In β-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC) | Afegostat Tartrate Treatment Regimen 2 | 3.9 | Standard Deviation 4.12 | picomole/minute/mg |
Source ClinicalTrials.gov
Participant flow10
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Afegostat Tartrate Treatment Regimen 1 | 11 | |
| Overall Study | STARTED | Afegostat Tartrate Treatment Regimen 2 | 8 | |
| Overall Study | Safety Population | Afegostat Tartrate Treatment Regimen 1 | 11 | |
| Overall Study | Safety Population | Afegostat Tartrate Treatment Regimen 2 | 8 | |
| Overall Study | PD Population | Afegostat Tartrate Treatment Regimen 1 | 11 | |
| Overall Study | PD Population | Afegostat Tartrate Treatment Regimen 2 | 8 | |
| Overall Study | COMPLETED | Afegostat Tartrate Treatment Regimen 1 | 10 | |
| Overall Study | COMPLETED | Afegostat Tartrate Treatment Regimen 2 | 8 | |
| Overall Study | NOT COMPLETED | Afegostat Tartrate Treatment Regimen 1 | 1 | |
| Overall Study | NOT COMPLETED | Afegostat Tartrate Treatment Regimen 2 | 0 |
Source ClinicalTrials.gov
Adverse events134
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 | Splenomegaly | Blood and lymphatic system disorders | Afegostat Tartrate Treatment Regimen 1 | 0 | 11 | |
| other | Splenomegaly | Blood and lymphatic system disorders | Afegostat Tartrate Treatment Regimen 2 | 1 | 8 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Afegostat Tartrate Treatment Regimen 1 | 1 | 11 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Afegostat Tartrate Treatment Regimen 2 | 0 | 8 | |
| other | Abnormal sensation in eye | Eye disorders | Afegostat Tartrate Treatment Regimen 1 | 1 | 11 | |
| other | Abnormal sensation in eye | Eye disorders | Afegostat Tartrate Treatment Regimen 2 | 1 | 8 | |
| other | Chalazion | Eye disorders | Afegostat Tartrate Treatment Regimen 1 | 0 | 11 | |
| other | Chalazion | Eye disorders | Afegostat Tartrate Treatment Regimen 2 | 1 | 8 | |
| other | Conjunctival haemorrhage | Eye disorders | Afegostat Tartrate Treatment Regimen 1 | 0 | 11 | |
| other | Conjunctival haemorrhage | Eye disorders | Afegostat Tartrate Treatment Regimen 2 | 1 | 8 |
1 to 10 of 134Source ClinicalTrials.gov
See all 134 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Afegostat Tartrate Treatment Regimen 1 | ||
| Total, all-cause mortality | Afegostat Tartrate Treatment Regimen 2 | ||
| Total, other adverse events | Afegostat Tartrate Treatment Regimen 1 | 10 | 11 |
| Total, other adverse events | Afegostat Tartrate Treatment Regimen 2 | 8 | 8 |
| Total, serious adverse events | Afegostat Tartrate Treatment Regimen 1 | 0 | 11 |
| Total, serious adverse events | Afegostat Tartrate Treatment Regimen 2 | 0 | 8 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Afegostat Tartrate Treatment Regimen 1 vs Afegostat Tartrate Treatment Regimen 2: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) | Trial comparisons |
| Afegostat Tartrate Treatment Regimen 1 vs Afegostat Tartrate Treatment Regimen 2: serious adverse events | Trial comparisons |
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/NCT00446550
GET https://conductscience.org/api/trials/v1/NCT00446550/events?page=2