NCT02930655 is a Phase 1 clinical trial of Lucerastat in Fabry Disease, sponsored by Idorsia Pharmaceuticals Ltd..

Who is sponsoring NCT02930655?

NCT02930655 is sponsored by Idorsia Pharmaceuticals Ltd..

What drugs are being tested in NCT02930655?

Interventions in NCT02930655: Lucerastat, Enzyme replacement therapy (ERT).

What condition does NCT02930655 study?

NCT02930655 studies Fabry Disease.

Is NCT02930655 still recruiting?

NCT02930655 is currently completed. Last updated 10 July 2018.

Last reviewed 2018-07-10 · How we verify

NCT02930655

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease

Completed Phase 1 Last updated 10 July 2018

What this trial tests

Phase 1 trial testing Lucerastat in Fabry Disease in 14 participants. Completed in 1 February 2016.

Timeline

1 February 2015

Primary endpoint
1 February 2016

1 February 2016

Quick facts

Lead sponsor	Idorsia Pharmaceuticals Ltd.
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	14
Start date	1 February 2015
Primary completion	1 February 2016
Estimated completion	1 February 2016
Sites	1 location across Germany

Drugs / interventions tested

Lucerastat — full drug profile →
Enzyme replacement therapy (ERT) — full drug profile →

Conditions studied

Fabry Disease — all drugs for Fabry Disease →

Sponsor

Idorsia Pharmaceuticals Ltd. — full company profile →

Who can join

18 and older, any sex, with Fabry Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The primary purpose of this study was to assess the safety and tolerability of lucerastat in adults with Fabry Disease receiving Enzyme Replacement Therapy (ERT). The secondary objectives were to investigate the effects of lucerastat on plasma and urine levels of biomarkers, to assess its effects on renal and cardiac functions and to determine the pharmacokinetic profile of lucerastat at steady-state.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Fabry Disease: Molecular Basis, Pathophysiology, Diagnostics and Potential Therapeutic Directions.
Kok K, Zwiers KC, Boot RG, Overkleeft HS, et al · · 2021 · cited 81× · PMID 33673160 · DOI 10.3390/biom11020271
Therapeutic Approaches in Lysosomal Storage Diseases.
Fernández-Pereira C, San Millán-Tejado B, Gallardo-Gómez M, Pérez-Márquez T, et al · · 2021 · cited 43× · PMID 34944420 · DOI 10.3390/biom11121775
Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: safety, tolerability, and pharmacokinetics in healthy subjects.
Guérard N, Morand O, Dingemanse J. · · 2017 · cited 32× · PMID 28088251 · DOI 10.1186/s13023-017-0565-9
Fabry disease: Mechanism and therapeutics strategies.
Li X, Ren X, Zhang Y, Ding L, et al · · 2022 · cited 27× · PMID 36386210 · DOI 10.3389/fphar.2022.1025740
Inflammation, Oxidative Stress, and Endothelial Dysfunction in the Pathogenesis of Vascular Damage: Unraveling Novel Cardiovascular Risk Factors in Fabry Disease.
Faro DC, Di Pino FL, Monte IP. · · 2024 · cited 25× · PMID 39125842 · DOI 10.3390/ijms25158273
Precision Medicine for Lysosomal Disorders.
Pinto E Vairo F, Rojas Málaga D, Kubaski F, Fischinger Moura de Souza C, et al · · 2020 · cited 15× · PMID 32722587 · DOI 10.3390/biom10081110
Challenges in Treating Genodermatoses: New Therapies at the Horizon.
Morren MA, Legius E, Giuliano F, Hadj-Rabia S, et al · · 2021 · cited 10× · PMID 35069188 · DOI 10.3389/fphar.2021.746664
Overcoming Resistance in Anderson-Fabry Disease: Current Therapeutic Challenges and Future Perspectives.
Carella MC, Forleo C, Caretto P, Naccarati ML, et al · · 2024 · cited 5× · PMID 39685654 · DOI 10.3390/jcm13237195

Verify or expand the search:

Other trials of Lucerastat

Trials testing the same drug.

NCT03425539 — Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease · Phase 3 · completed
NCT03380455 — Effect of Cimetidine on the Pharmacokinetics of Lucerastat in Healthy Subjects · Phase 1 · completed

Other recruiting trials for Fabry Disease

Currently open trials in the same condition.

NCT07187440 — A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease · recruiting
NCT06776419 — the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease · recruiting
NCT06539624 — Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease · NA · recruiting
NCT07277361 — Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment · recruiting
NCT06270316 — Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease · Phase 1, PHASE2 · recruiting

Other Idorsia Pharmaceuticals Ltd. trials

Trials by the same sponsor.

NCT06799884 — A Drug-drug Interaction Trial in Healthy Female Participants to Investigate the Effect of Aprocitentan on Combined Hormo · Phase 1 · completed
NCT05877222 — A Phase 1 Trial to Investigate the Biological Equivalence of 5 × 10 mg Tablets and 2 × 25 mg Tablets of Daridorexant in · Phase 1 · completed
NCT05597020 — A Study to Find Out if Daridorexant is Safe and Efficacious in Patients With Insomnia and Comorbid Nocturia · Phase 4 · completed
NCT05632393 — A Study to Measure Daridorexant in Breast Milk of Healthy Lactating Women · Phase 1 · completed
NCT05702177 — A Study of the Effect of Daridorexant on Nighttime Body Posture, the Noise Level Required to Wake up, and the Ability to · Phase 1 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT02930655 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Idorsia Pharmaceuticals Ltd.
Last refreshed: 10 July 2018

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02930655.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing