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

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ClinicalTrials.gov Identifier: NCT02930655
Recruitment Status : Completed
First Posted : October 12, 2016
Last Update Posted : July 10, 2018
Sponsor:
Information provided by (Responsible Party):
Idorsia Pharmaceuticals Ltd.

Brief Summary:

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.


Condition or disease Intervention/treatment Phase
Fabry Disease Drug: Lucerastat Drug: Enzyme replacement therapy (ERT) Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single-center, Open-label, Randomized, Versus a Control Group, Phase 1b Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Oral Lucerastat in Adult Subjects With Fabry Disease Receiving Enzyme Replacement Therapy
Actual Study Start Date : February 1, 2015
Actual Primary Completion Date : February 1, 2016
Actual Study Completion Date : February 1, 2016

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Lucerastat group
Ten subjects with Fabry Disease received 1000 mg of oral lucerastat twice daily for 12 weeks in addition to their standard of care treatment (enzyme replace therapy).
Drug: Lucerastat
Hard gelatin capsules for oral administration formulated at a strength of 250 mg, and administered as 4 capsules in the morning and 4 capsules in the evening.
Other Name: ACT-434964

Drug: Enzyme replacement therapy (ERT)
All the subjects received an ERT as background therapy for at least 24 months prior to the screening visit and they had to continue receiving this treatment during the conduct of the study.
Other Names:
  • Fabrazyme
  • Replagal

Experimental: Control group
Four subjects with Fabry Disease under enzyme replace therapy (ERT) as standard of care treatment were included as a control group.
Drug: Enzyme replacement therapy (ERT)
All the subjects received an ERT as background therapy for at least 24 months prior to the screening visit and they had to continue receiving this treatment during the conduct of the study.
Other Names:
  • Fabrazyme
  • Replagal




Primary Outcome Measures :
  1. Change from baseline in blood pressure [ Time Frame: Up to Week 12 ]
  2. Change from baseline in heart rate [ Time Frame: Up to Week 12 ]
  3. Change from baseline in electrocardiogram (ECG) variables [ Time Frame: Up to Week 12 ]
    The duration (in ms) of the different ECG variables were measured using a standard 12-lead ECG

  4. Change from baseline in body weight [ Time Frame: Up to Week 12 ]
  5. Number of subjects with treatment-emergent adverse events and serious adverse events [ Time Frame: Up to Week 12 ]
  6. Number of subjects with adverse events leading to premature discontinuation of lucerastat or ERT [ Time Frame: Up to Week 12 ]
  7. Number of subjects with treatment-emergent abnormalities in laboratory variables [ Time Frame: Up to Week 12 ]

Secondary Outcome Measures :
  1. Change from baseline in plasma biomarkers of Fabry Disease [ Time Frame: Up to Week 12 ]
    Biomarkers reflecting glycolipid metabolism were measured (unit of measure: ng/mL)

  2. Change from baseline in urine biomarker of Fabry Disease [ Time Frame: Up to Week 12 ]
    Biomarker reflecting glycolipid metabolism was measured (unit of measure: ng/mg)

  3. Change from baseline in left ventricular ejection fraction (LVEF) [ Time Frame: Up to Week 12 ]
    LVEF was used to monitor cardiac function in subjects with Fabry Disease

  4. Change from baseline in left ventricular mass index (LVMi) [ Time Frame: Up to Week 12 ]
    LVMi was used to monitor cardiac function in subjects with Fabry Disease

  5. Change from baseline in estimated glomerular filtration rate (eGFR) [ Time Frame: Up to Week 12 ]
    eGFR was used to monitor renal function in subjects with Fabry Disease

  6. Change from baseline in urine albumin-to-creatinine ratio (UACR) [ Time Frame: Up to Week 12 ]
    UACR was used to monitor renal function in subjects with Fabry Disease

  7. Maximum plasma concentration (Cmax) of lucerastat [ Time Frame: At Week 4 visit, blood samples drawn at the following time points: pre-dose, 0.5h, 1h, 1.5h, 2h, 2.5h, 3h, 3.5h, 4h, 6h, 8h, 10h, 12h post-dose ]
    Cmax was determined directly from the observed plasma concentration-time curves of lucerastat. Blood samples for PK analyses were drawn at scheduled time points at the Week 4 visit

  8. Time to reach Cmax (tmax) of lucerastat [ Time Frame: At Week 4 visit, blood samples drawn at the following time points: pre-dose, 0.5h, 1h, 1.5h, 2h, 2.5h, 3h, 3.5h, 4h, 6h, 8h, 10h, 12h post-dose ]
    tmax was determined directly from the observed plasma concentration-time curves of lucerastat. Blood samples for PK analyses were drawn at scheduled time points at the Week 4 visit

  9. Area under the plasma concentration-time curve [AUC(tau)] of lucerastat [ Time Frame: At Week 4 visit, blood samples drawn at the following time points: pre-dose, 0.5h, 1h, 1.5h, 2h, 2.5h, 3h, 3.5h, 4h, 6h, 8h, 10h, 12h post-dose ]
    AUC(tau) corresponds to the area under the plasma concentration time curve of lucerastat over a dosing interval (tau = 12 hours)

  10. Terminal half-life [t(1/2)]of lucerastat [ Time Frame: At Week 4 visit, blood samples drawn at the following time points: pre-dose, 0.5h, 1h, 1.5h, 2h, 2.5h, 3h, 3.5h, 4h, 6h, 8h, 10h, 12h post-dose ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Signed informed consent form
  • Male and female adult subjects with a diagnosis of Fabry Disease (FD) based on historical assessments (residual α-GAL A activity level below lower limit of normal for males and presence of a galactosidase alpha mutation for females) and a history of clinical symptoms of FD
  • On ERT for at least 24 months without any change in dose within the last 6 months prior to screening

Exclusion Criteria:

  • Severe renal function impairment
  • Severe residual neurologic deficit
  • Clinically significant unstable cardiac disease
  • Any circumstances or conditions, which, in the opinion of the investigator, may have affected full participation in the study or compliance with the protocol

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02930655


Locations
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Germany
Investigator Site
Würzburg, Germany, 97080
Sponsors and Collaborators
Idorsia Pharmaceuticals Ltd.
Investigators
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Study Director: Nicolas Guérard Actelion

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Idorsia Pharmaceuticals Ltd.
ClinicalTrials.gov Identifier: NCT02930655     History of Changes
Other Study ID Numbers: AC-069-104
First Posted: October 12, 2016    Key Record Dates
Last Update Posted: July 10, 2018
Last Verified: July 2018

Keywords provided by Idorsia Pharmaceuticals Ltd.:
Fabry disease
lucerastat

Additional relevant MeSH terms:
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Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders