A research study to study the effects of a new oral drug called lucerastat in adults with Fabry disease

• Register: EUCTR
• Last refreshed on: 8 October 2021
• Main ID: EUCTR2017-003369-85-IE
• Date of registration: 01/07/2019
• Prospective Registration: Yes
• Primary sponsor: Idorsia Pharmaceuticals Ltd
• Public title: A research study to study the effects of a new oral drug called lucerastat in adults with Fabry disease
• Scientific title: A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease. - MODIFY
• Date of first enrolment: 18/05/2020
• Target sample size: 99
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-003369-85
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• 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, Germany, Ireland, Italy, Netherlands, Norway, Poland, Spain, Switzerland, United Kingdom, United States

Contacts

• Name: Clinical Trial Disclosure Desk
• Affiliation: Idorsia Pharmaceuticals Ltd

• Name: Clinical Trial Disclosure Desk
• Affiliation: Idorsia Pharmaceuticals Ltd

Key inclusion & exclusion criteria

Inclusion criteria:
1. Signed and dated ICF prior to any study-mandated procedure.
2. Male or female subjects; 18-years old and above.
3. FD diagnosis confirmed with local genetic test results (i.e., presence of at least 1 mutation in GLA, the gene coding for a-
galactosidase A).
4. Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening.

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 89
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion criteria:
1. Pregnant, planning to be become pregnant up to 30 days after study treatment discontinuation or lactating subject;
2. Severe renal insufficiency defined as an estimated glomerular filtration rate (eGFR) per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 30 mL/min/1.73 m2 at screening;
3. Subject on regular dialysis for the treatment of chronic kidney disease;
4. Subject has undergone, or is on a waiting list for, or is scheduled to undergo kidney or other organ transplantation;
5. Known and documented transient ischemic attack, stroke, unstable angina or myocardial infarction within 6 months prior to screening;
6. Clinically significant unstable cardiac disease in the opinion of the investigator (e.g., uncontrolled symptomatic arrhythmia, New York Heart Association class III or IV congestive heart failure);
7. Any other subject at high risk for developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
8. Subject planned for imminent initiation of treatment with ERT.


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: 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-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: • To determine the effect of lucerastat on neuropathic pain in subjects with Fabry disease (FD).

Secondary Objective: • To determine the effects of lucerastat on gastro-intestinal (GI) symptoms (abdominal pain and diarrhea) in subjects with FD and GI symptom(s) at baseline.
• To confirm the effect of lucerastat on biomarkers of FD.
• To determine the safety and tolerability of lucerastat in subjects with FD.

Timepoint(s) of evaluation of this end point: From baseline to Month 6

Primary end point(s): The primary efficacy endpoint is change from baseline to Month 6 in the "modified" Brief Pain Inventory-Short Form item 3 (BPI-SF3) score of "neuropathic pain at its worst in the last 24 hours".

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: From baseline to Month 6

Secondary end point(s): • Change from baseline to Month 6 in the 11-point Numerical Rating Scale (NRS-11) score of “abdominal pain at its worst in the last 24 hours” in subjects with GI symptoms at baseline.
• Change from baseline to Month 6 in the number of days with at least one stool of a Bristol Stool Scale (BSS) consistency Type 6 or 7 in subjects with GI symptoms at baseline.
• Change from baseline to Month 6 in plasma globotriaosylceramide (Gb3).

Secondary ID(s)

2017-003369-85-GB

ID-069A301

Source(s) of Monetary Support

Idorsia Pharmaceuticals Ltd

Ethics review

Status: Approved
Approval date: 18/05/2020
Contact: