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

• Register: EUCTR
• Last refreshed on: 11 November 2024
• Main ID: EUCTR2018-002210-12-NO
• Date of registration: 22/09/2020
• Prospective Registration: Yes
• Primary sponsor: Idorsia Pharmaceuticals Ltd
• Public title: A study to determine the long-term safety and tolerability of oral 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: 26/03/2021
• Target sample size: 107
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-002210-12
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

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

Contacts

• Name: Idorsia Clinical Trial Information
• Affiliation: Idorsia Pharmaceuticals Ltd

• Name: Idorsia Clinical Trial Information
• Affiliation: Idorsia Pharmaceuticals Ltd

Key inclusion & exclusion criteria

Inclusion criteria:
1. Signed and dated ICF prior to any study-mandated procedure;
2. Subject completed the 6-month, double-blind treatment period in study ID 069A301;

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 103
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4

Exclusion criteria:
1. Pregnant / planning to become pregnant or lactating subject;
2. 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;
3. 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:
4. Subject’s eGFR per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 15 mL/min/1.73 m2;
5. Subject experienced an event of acute kidney injury Common Terminology Criteria for Adverse Event (CTCAE) grade 2 or above;
6. Subject experienced an event of stroke CTCAE grade 3 or above;
7. Subject experienced an event of heart failure leading to in-patient hospitalization or prolongation of ongoing hospitalization.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Fabry disease
MedDRA version: 20.0 Level: SOC Classification code 10010331 Term: Congenital, familial and genetic disorders System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 24.1 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: Lucerastat
Product Code: ACT-434964
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Lucerastat
Current Sponsor code: ACT-434964
Other descriptive name: OGT923
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: From enrollment up to EOS (which corresponds to FU1)

Primary end point(s): Treatment-emergent AEs and SAEs

Main Objective: To determine the long-term safety and tolerability of lucerastat in subjects with Fabry disease

Secondary Objective: To evaluate the effect of lucerastat on renal function and cardiac variables in subjects with Fabry disease;
To evaluate the long-term effect of lucerastat on biomarkers of Fabry disease.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: N.A

Secondary end point(s): N.A

Secondary ID(s)

2018-002210-12-GB

ID-069A302

Source(s) of Monetary Support

Idorsia Pharmaceuticals Ltd

Ethics review

Status: Approved
Approval date: 26/03/2021
Contact: