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

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ClinicalTrials.gov Identifier: NCT03737214
Recruitment Status : Recruiting
First Posted : November 9, 2018
Last Update Posted : July 18, 2019
Sponsor:
Information provided by (Responsible Party):
Idorsia Pharmaceuticals Ltd.

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 108 participants
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 : June 22, 2022
Estimated Study Completion Date : July 22, 2022

Resource links provided by the National Library of Medicine


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.




Primary Outcome Measures :
  1. Treatment-emergent adverse events (AEs) [ Time Frame: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 25 months (24 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 25 months (24 months OL treatment period plus 1 month Follow-up) ]
  2. Subject estimated glomerular filtration rate (eGFR) slope [ Time Frame: From baseline to Month 24 (duration: 2 years) ]
  3. Change in left ventricular mass index (LVMI) [ Time Frame: From baseline to Month 24 (duration: 2 years) ]
  4. Change in plasma globotriaosylceramide (Gb3) [ Time Frame: From baseline to Month 24 (duration: 2 years) ]


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 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 up to 30 days after study treatment discontinuation 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.

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


Contacts
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Contact: Clinical Trial Disclosure Desk +18566613721 clinical-trials-disclosure@idorsia.com

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Sponsors and Collaborators
Idorsia Pharmaceuticals Ltd.
Investigators
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Study Director: Clinical Trials Idorsia Pharmaceuticals Ltd.

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Responsible Party: Idorsia Pharmaceuticals Ltd.
ClinicalTrials.gov Identifier: NCT03737214     History of Changes
Other Study ID Numbers: ID-069A302
2018-002210-12 ( EudraCT Number )
First Posted: November 9, 2018    Key Record Dates
Last Update Posted: July 18, 2019
Last Verified: July 2019
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