Trial · NCT00433147

A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

NCT00433147, 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 AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy. Trials, record NCT00433147. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00433147

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
NCT number: NCT00433147
Status: Completed
Phases: Phase 2
Enrollment: 30 participants, actual
Lead sponsor: Amicus Therapeutics
Conditions: Gaucher Disease, Type 1, Type 1 Gaucher Disease, Gaucher Disease
Interventions: Afegostat tartrate
First posted: 2007-02-09
Last updated: 2018-09-25
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 35	TEAEs were defined as any adverse event (AE) with a start date on or after administration of the study drug (on Day 1). A severe AE was 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 7 days after the last dose of study drug (Day 35) 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 Beta-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC)	Baseline, Day 28	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 35	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 Beta-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC)	Baseline, Day 28	PD Population: All participants who were included in the Safety Population and had a baseline and at least 1 post-baseline PD measurement.	pmol/mg/min	MEAN

Source ClinicalTrials.gov

Results by group16

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 25 mg Once Per Day		0		Participants
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)	Afegostat Tartrate 150 mg Once Per Day		0		Participants
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)	Afegostat Tartrate 150 mg Once Every Four Days		0		Participants
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)	Afegostat Tartrate 150 mg Once Every Seven Days		0		Participants
Change From Baseline To End Of Treatment In Beta-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC)	Afegostat Tartrate 25 mg Once Per Day	Baseline	8.732	Standard Deviation 3.1079	pmol/mg/min
Change From Baseline To End Of Treatment In Beta-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC)	Afegostat Tartrate 150 mg Once Per Day	Baseline	8.543	Standard Deviation 4.1560	pmol/mg/min
Change From Baseline To End Of Treatment In Beta-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC)	Afegostat Tartrate 150 mg Once Every Four Days	Baseline	9.371	Standard Deviation 3.5133	pmol/mg/min
Change From Baseline To End Of Treatment In Beta-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC)	Afegostat Tartrate 150 mg Once Every Seven Days	Baseline	12.035	Standard Deviation 5.4742	pmol/mg/min
Change From Baseline To End Of Treatment In Beta-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC)	Afegostat Tartrate 25 mg Once Per Day	Day 28	19.148	Standard Deviation 17.0210	pmol/mg/min
Change From Baseline To End Of Treatment In Beta-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC)	Afegostat Tartrate 150 mg Once Per Day	Day 28	23.557	Standard Deviation 14.6970	pmol/mg/min

1 to 10 of 16Source ClinicalTrials.gov

See all 16 results by group

Participant flow24

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Afegostat Tartrate 25 Milligrams (mg) Once Per Day	7
Overall Study	STARTED	Afegostat Tartrate 150 mg Once Per Day	7
Overall Study	STARTED	Afegostat Tartrate 150 mg Once Every Four Days	9
Overall Study	STARTED	Afegostat Tartrate 150 mg Once Every Seven Days	7
Overall Study	Safety Population	Afegostat Tartrate 25 Milligrams (mg) Once Per Day	7
Overall Study	Safety Population	Afegostat Tartrate 150 mg Once Per Day	7
Overall Study	Safety Population	Afegostat Tartrate 150 mg Once Every Four Days	9
Overall Study	Safety Population	Afegostat Tartrate 150 mg Once Every Seven Days	7
Overall Study	PD Population	Afegostat Tartrate 25 Milligrams (mg) Once Per Day	7
Overall Study	PD Population	Afegostat Tartrate 150 mg Once Per Day	6

1 to 10 of 24Source ClinicalTrials.gov

See all 24 participant flow

Adverse events132

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
other	Dry eye	Eye disorders	Afegostat Tartrate 25 mg Once Per Day	0	7
other	Dry eye	Eye disorders	Afegostat Tartrate 150 mg Once Per Day	1	7
other	Dry eye	Eye disorders	Afegostat Tartrate 150 mg Once Every Four Days	0	9
other	Dry eye	Eye disorders	Afegostat Tartrate 150 mg Once Every Seven Days	0	7
other	Lacrimation increased	Eye disorders	Afegostat Tartrate 25 mg Once Per Day	0	7
other	Lacrimation increased	Eye disorders	Afegostat Tartrate 150 mg Once Per Day	1	7
other	Lacrimation increased	Eye disorders	Afegostat Tartrate 150 mg Once Every Four Days	0	9
other	Lacrimation increased	Eye disorders	Afegostat Tartrate 150 mg Once Every Seven Days	0	7
other	Ocular hyperaemia	Eye disorders	Afegostat Tartrate 25 mg Once Per Day	0	7
other	Ocular hyperaemia	Eye disorders	Afegostat Tartrate 150 mg Once Per Day	1	7

1 to 10 of 132Source ClinicalTrials.gov

See all 132 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Afegostat Tartrate 25 mg Once Per Day
Total, all-cause mortality	Afegostat Tartrate 150 mg Once Per Day
Total, all-cause mortality	Afegostat Tartrate 150 mg Once Every Four Days
Total, all-cause mortality	Afegostat Tartrate 150 mg Once Every Seven Days
Total, other adverse events	Afegostat Tartrate 25 mg Once Per Day	4	7
Total, other adverse events	Afegostat Tartrate 150 mg Once Per Day	2	7
Total, other adverse events	Afegostat Tartrate 150 mg Once Every Four Days	4	9
Total, other adverse events	Afegostat Tartrate 150 mg Once Every Seven Days	1	7
Total, serious adverse events	Afegostat Tartrate 25 mg Once Per Day	0	7
Total, serious adverse events	Afegostat Tartrate 150 mg Once Per Day	0	7

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

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 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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT00433147

GET https://conductscience.org/api/trials/v1/NCT00433147/flow?page=2

API reference For AI assistants