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

• Register: ClinicalTrials.gov
• Last refreshed on: 22 January 2024
• Main ID: NCT03737214
• Date of registration: 06/11/2018
• Prospective Registration: Yes
• Primary sponsor: Idorsia Pharmaceuticals Ltd.
• Public title: A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
• Scientific 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
• Date of first enrolment: December 18, 2018
• Target sample size: 107
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03737214
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Austria, Belgium, Canada, Czechia, France, Germany, Ireland, Italy, Netherlands, Norway, Poland, Spain, Switzerland, United Kingdom, United States

Contacts

• Name: Clinical Trials
• Affiliation: Idorsia Pharmaceuticals Ltd.

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: Lucerastat

Primary Outcome(s)

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)]

Secondary ID(s)

2018-002210-12

ID-069A302

Source(s) of Monetary Support

Please refer to primary and secondary sponsors