Trial · NCT03425539

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

NCT03425539, Phase 3, Completed, Idorsia Pharmaceuticals Ltd.; results first posted 2024-08-09

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease. Trials, record NCT03425539. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03425539

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Trial

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

Title: Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
NCT number: NCT03425539
Status: Completed
Phases: Phase 3
Enrollment: 118 participants, actual
Lead sponsor: Idorsia Pharmaceuticals Ltd.
Conditions: Fabry Disease
Interventions: Lucerastat, Placebo
First posted: 2018-02-07
Last updated: 2024-08-09
Results first posted: 2024-08-09

Source ClinicalTrials.gov

Registered outcomes4

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Neuropathic Pain Monthly Score: Change From Baseline to Month 6	From baseline to Month 6 (duration: 6 months)	Neuropathic pain on the modified BPI-SF3: subjects rated their neuropathic pain intensity ("neuropathic pain at its worst in the last 24 hours") on an 11-point scale, from 0 (no neuropathic pain) to 10 (worst imaginable neuropathic pain).
Secondary	Plasma Globotriaosylceramide (Gb3; in ng/ml): Change From Baseline to Month 6	From baseline to Month 6 (duration: 6 months)
Secondary	Abdominal Pain Monthly Score: Change From Baseline to Month 6	From baseline to Month 6 (duration: 6 months)	Abdominal pain on the 11-point Numerical Rating Scale (NRS-11): subjects rated their abdominal pain intensity ("abdominal pain at its worst in the last 24 hours") on an 11-point scale, from 0 (no pain) to 10 (worst imaginable pain).
Secondary	Number of Days With Diarrhea: Change From Baseline to Month 6	From baseline to Month 6 (duration: 6 months)	A subject was considered to have diarrhea on a specific day if at least one stool of a Bristol Stool Scale (BSS) consistency Type 6 or 7 was reported. The number of days with diarrhea at baseline and Month 6 was the number of days with diarrhea over the 4 weeks prior to the randomization visit or the Month 6 visit, respectively, adjusted for the number of days with data available.

Source ClinicalTrials.gov

Outcomes4

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Neuropathic Pain Monthly Score: Change From Baseline to Month 6	From baseline to Month 6 (duration: 6 months)	The analysis included all subjects who were randomized and took at least one dose of study treatment.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Plasma Globotriaosylceramide (Gb3; in ng/ml): Change From Baseline to Month 6	From baseline to Month 6 (duration: 6 months)	The analysis included all subjects who were randomized and took at least one dose of study treatment.	ng/ml	LEAST_SQUARES_MEAN
SECONDARY	Abdominal Pain Monthly Score: Change From Baseline to Month 6	From baseline to Month 6 (duration: 6 months)	The analysis included randomized and treated subjects with gastrointestinal symptoms at baseline (diarrhea on at least 8 days and/or moderate or severe abdominal pain in the 4 weeks prior to randomization).	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Number of Days With Diarrhea: Change From Baseline to Month 6	From baseline to Month 6 (duration: 6 months)	The analysis included randomized and treated subjects with gastrointestinal symptoms at baseline (diarrhea on at least 8 days and/or moderate or severe abdominal pain in the 4 weeks prior to randomization) and a non-missing change from baseline to Month 6 value.	Number of days within 4 weeks	MEAN

Source ClinicalTrials.gov

Results by group8

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Neuropathic Pain Monthly Score: Change From Baseline to Month 6	Lucerastat	-1.64	95% Confidence Interval -2.11 to -1.16	score on a scale
Neuropathic Pain Monthly Score: Change From Baseline to Month 6	Placebo	-2.05	95% Confidence Interval -2.73 to -1.37	score on a scale
Plasma Globotriaosylceramide (Gb3; in ng/ml): Change From Baseline to Month 6	Lucerastat	-672.68	95% Confidence Interval -798.91 to -546.46	ng/ml
Plasma Globotriaosylceramide (Gb3; in ng/ml): Change From Baseline to Month 6	Placebo	200.84	95% Confidence Interval 14.29 to 387.40	ng/ml
Abdominal Pain Monthly Score: Change From Baseline to Month 6	Lucerastat	-1.37	95% Confidence Interval -1.86 to -0.87	score on a scale
Abdominal Pain Monthly Score: Change From Baseline to Month 6	Placebo	-1.68	95% Confidence Interval -2.38 to -0.98	score on a scale
Number of Days With Diarrhea: Change From Baseline to Month 6	Lucerastat	-3.45	Standard Deviation 4.96	Number of days within 4 weeks
Number of Days With Diarrhea: Change From Baseline to Month 6	Placebo	-3.95	Standard Deviation 9.29	Number of days within 4 weeks

Source ClinicalTrials.gov

Participant flow8

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Lucerastat	80
Overall Study	STARTED	Placebo	38
Overall Study	Treated	Lucerastat	80
Overall Study	Treated	Placebo	37
Overall Study	COMPLETED	Lucerastat	75
Overall Study	COMPLETED	Placebo	34
Overall Study	NOT COMPLETED	Lucerastat	5
Overall Study	NOT COMPLETED	Placebo	4

Source ClinicalTrials.gov

Adverse events64

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	Vertigo	Ear and labyrinth disorders	Lucerastat	1	80	1
other	Vertigo	Ear and labyrinth disorders	Placebo	2	37	2
other	Abdominal discomfort	Gastrointestinal disorders	Lucerastat	1	80	1
other	Abdominal discomfort	Gastrointestinal disorders	Placebo	2	37	2
other	Abdominal distension	Gastrointestinal disorders	Lucerastat	4	80	4
other	Abdominal distension	Gastrointestinal disorders	Placebo	4	37	4
other	Abdominal pain	Gastrointestinal disorders	Lucerastat	4	80	5
other	Abdominal pain	Gastrointestinal disorders	Placebo	1	37	2
other	Diarrhoea	Gastrointestinal disorders	Lucerastat	9	80	10
other	Diarrhoea	Gastrointestinal disorders	Placebo	1	37	1

1 to 10 of 64Source ClinicalTrials.gov

See all 64 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Lucerastat	0	80
Total, all-cause mortality	Placebo	0	37
Total, other adverse events	Lucerastat	46	80
Total, other adverse events	Placebo	21	37
Total, serious adverse events	Lucerastat	5	80
Total, serious adverse events	Placebo	1	37

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
Lucerastat vs Placebo: deaths from any cause	Trial comparisons
Lucerastat vs Placebo: 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.

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/NCT03425539

GET https://conductscience.org/api/trials/v1/NCT03425539/events?page=2

API reference For AI assistants