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Completed

NCT Number: NCT02930655

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

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.

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Key information

Conditions

Fabry Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses Vascular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Investigator Site

Würzburg, 97080, Germany

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Lucerastat

Drug

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 names: ACT-434964

Enzyme replacement therapy (ERT)

Drug

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 outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

Idorsia Pharmaceuticals Ltd.

Industry

Registry information

Official study 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

Important dates

Study start
2015
Primary completion
2016
Study completion
2016
First posted
Oct 12, 2016
Registry last updated
Jul 10, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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