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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Idorsia Pharmaceuticals Ltd.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trial duration

13 Mar 2019 → ongoing

Decision date (initial)

2024-11-08

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Idorsia Pharmaceuticals Ltd

External identifiers

EU CT number

2024-513884-20-00

EudraCT number

2018-002210-12

ClinicalTrials.gov

NCT03737214

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Pharmacodynamic, Efficacy, Safety

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

Secondary objectives 2

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

Conditions and MedDRA coding

Fabry disease

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

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;

Exclusion criteria 7

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.
4. If at any time during study ID-069A301, the following criteria was met: Subject’s eGFR per the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation < 15 mL/min/1.73 m2.
5. If at any time during study ID-069A301, the following criteria was met: Subject experienced an event of acute kidney injury Common Terminology Criteria for Adverse Event (CTCAE) grade 2 or above;
6. If at any time during study ID-069A301, the following criteria was met: Subject experienced an event of stroke CTCAE grade 3 or above;
7. If at any time during study ID-069A301, the following criteria was met: Subject experienced an event of heart failure leading to in-patient hospitalization or prolongation of ongoing hospitalization.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Treatment-emergent AEs and SAEs

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Idorsia Pharmaceuticals Ltd.

Sponsor organisation

Idorsia Pharmaceuticals Ltd.

Address

Hegenheimermattweg 91

City

Allschwil

Postcode

4123

Country

Switzerland

Scientific contact point

Organisation

Idorsia Pharmaceuticals Ltd.

Contact name

Idorsia Clinical Trial Information

Public contact point

Organisation

Idorsia Pharmaceuticals Ltd.

Contact name

Idorsia Clinical Trial Information

Third parties 14

Locations

7 EU/EEA countries · 15 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 60 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications