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A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03737214
Recruitment Status : Active, not recruiting
First Posted : November 9, 2018
Last Update Posted : January 17, 2023
Sponsor:
Information provided by (Responsible Party):
Idorsia Pharmaceuticals Ltd.

Study Description
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Brief Summary:
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease

Condition or disease Intervention/treatment Phase
Fabry Disease Drug: Lucerastat Phase 3

Detailed Description:
Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country or until all subjects have reached Month 24 (for subjects participating only in Stage 1) or Month 48 (for subjects participating in Stages 1 and 2) or Month 72 (for subjects participating in Stages 1, 2 and 3), whichever is earliest.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 107 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Multi-center, open-label, uncontrolled, single-arm, extension study
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease
Actual Study Start Date : December 18, 2018
Estimated Primary Completion Date : October 29, 2025
Estimated Study Completion Date : November 29, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Lucerastat
Dose will be based on subject's eGFR.
 Drug: Lucerastat
Administered in hard gelatin capsules containing 250 mg of lucerastat.


Outcome Measures
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Primary Outcome Measures :
  1. Treatment-emergent adverse events (AEs) [ Time Frame: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 73 months (72 months OL treatment period plus 1 month Follow-up) ]

Other Outcome Measures:
  1. Treatment-emergent serious adverse events (SAEs) [ Time Frame: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 73 months (72 months OL treatment period plus 1 month Follow-up) ]
  2. Subject estimated glomerular filtration rate (eGFR) slope [ Time Frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years) ]
  3. Change in left ventricular mass index (LVMI) [ Time Frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years) ]
  4. Change in plasma globotriaosylceramide (Gb3) [ Time Frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years) ]

Eligibility Criteria
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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 ICF prior to any study-mandated procedure;
  • Subject completed the 6-month, double-blind treatment period in study ID 069A301
  • Woman of childbearing potential only if agreement 1) to follow a specified contraception scheme, 2) to undertake monthly urine pregnancy tests , 3) not to donate ova.
  • Fertile male only if agreement 1) to use a condom, 2) to not father a child, 3) not to donate sperm.

Exclusion Criteria:

  • Pregnant / planning to be become pregnant or lactating subject;
  • Subject considered to be at high risk of developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
  • Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results as per investigator judgment.

In addition, the subject must not be enrolled in study ID-069A302 if at any time during study ID-069A301, one of the following criteria was met:

  • Subject's eGFR per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 15 mL/min/1.73 m2;
  • Subject experienced an event of acute kidney injury Common Terminology Criteria for Adverse Event (CTCAE) grade 2 or above;
  • Subject experienced an event of stroke CTCAE grade 3 or above;
  • Subject experienced an event of heart failure leading to in-patient hospitalization or prolongation of ongoing hospitalization.
Contacts and Locations
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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): NCT03737214


Locations
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Sponsors and Collaborators
Idorsia Pharmaceuticals Ltd.
Investigators
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Study Director: Clinical Trials Idorsia Pharmaceuticals Ltd.
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Idorsia Pharmaceuticals Ltd.
ClinicalTrials.gov Identifier: NCT03737214    
Other Study ID Numbers: ID-069A302
2018-002210-12 ( EudraCT Number )
First Posted: November 9, 2018    Key Record Dates
Last Update Posted: January 17, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
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