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

a study on Fabry Disease

Summary

for people ages 18 years and up (full criteria)
at Irvine, California and other locations
study started
estimated completion

Description

Summary

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

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

Keywords

Fabry Disease 1-Deoxynojirimycin Lucerastat

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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.

You CAN'T join if...

  • 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.

Locations

  • University of California Irvine accepting new patients
    Irvine California 92696 United States
  • University of California - Los Angeles not yet accepting patients
    Los Angeles California 90025 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Idorsia Pharmaceuticals Ltd.
ID
NCT03737214
Phase
Phase 3
Study Type
Interventional
Last Updated