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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.

Tracking Information
First Submitted Date  ICMJE November 6, 2018
First Posted Date  ICMJE November 9, 2018
Last Update Posted Date January 17, 2023
Actual Study Start Date  ICMJE December 18, 2018
Estimated Primary Completion Date October 29, 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 12, 2023)
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) ]
Original Primary Outcome Measures  ICMJE
 (submitted: November 7, 2018)
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) ]
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures
 (submitted: January 12, 2023)
  • 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) ]
  • Subject estimated glomerular filtration rate (eGFR) slope [ Time Frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years) ]
  • Change in left ventricular mass index (LVMI) [ Time Frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years) ]
  • Change in plasma globotriaosylceramide (Gb3) [ Time Frame: From baseline to Month 24, Month 48 and Month 72 (duration: up to 6 years) ]
Original Other Pre-specified Outcome Measures
 (submitted: November 7, 2018)
  • 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) ]
  • Subject estimated glomerular filtration rate (eGFR) slope [ Time Frame: From baseline to Month 24 (duration: 2 years) ]
  • Change in left ventricular mass index (LVMI) [ Time Frame: From baseline to Month 24 (duration: 2 years) ]
  • Change in plasma globotriaosylceramide (Gb3) [ Time Frame: From baseline to Month 24 (duration: 2 years) ]
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
Official Title  ICMJE 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
Brief Summary A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE 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
Condition  ICMJE Fabry Disease
Intervention  ICMJE Drug: Lucerastat
Administered in hard gelatin capsules containing 250 mg of lucerastat.
Study Arms  ICMJE Experimental: Lucerastat
Dose will be based on subject's eGFR.
Intervention: Drug: Lucerastat
Publications * Wanner C, Kimonis V, Politei J, Warnock DG, Uceyler N, Frey A, Cornelisse P, Hughes D. Understanding and modifying Fabry disease: Rationale and design of a pivotal Phase 3 study and results from a patient-reported outcome validation study. Mol Genet Metab Rep. 2022 Mar 26;31:100862. doi: 10.1016/j.ymgmr.2022.100862. eCollection 2022 Jun.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: January 12, 2023)
107
Original Estimated Enrollment  ICMJE
 (submitted: November 7, 2018)
108
Estimated Study Completion Date  ICMJE November 29, 2025
Estimated Primary Completion Date October 29, 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Austria,   Belgium,   Canada,   France,   Germany,   Norway,   Poland,   Spain,   United Kingdom,   United States
Removed Location Countries Czechia,   Ireland,   Italy,   Netherlands,   Switzerland
 
Administrative Information
NCT Number  ICMJE NCT03737214
Other Study ID Numbers  ICMJE ID-069A302
2018-002210-12 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Idorsia Pharmaceuticals Ltd.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Idorsia Pharmaceuticals Ltd.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Clinical Trials Idorsia Pharmaceuticals Ltd.
PRS Account Idorsia Pharmaceuticals Ltd.
Verification Date January 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP