Long-term Safety of Lucerastat for Fabry Disease

Recruiting at 76 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Idorsia Pharmaceuticals Ltd.

Disqualifiers: Pregnancy, Acute kidney injury, Stroke, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24-72 months

107 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

What is the purpose of this trial?

This trial is testing the safety and tolerability of a medication called lucerastat, which is taken by mouth. It is aimed at adults with Fabry disease, a condition where harmful fats build up in the body. Lucerastat helps to reduce these fat levels.

Research Team

Clinical Trials

Principal Investigator

Idorsia Pharmaceuticals Ltd.

Eligibility Criteria

Adults with Fabry disease can join this trial if they've finished a previous 6-month study (ID 069A301). Women must use contraception and have monthly pregnancy tests. Men should use condoms and not father children. People with conditions that could affect the study or those at high risk for organ issues, pregnant, or lactating individuals cannot participate.

Inclusion Criteria

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

Subject completed the 6-month, double-blind treatment period in study ID 069A301

Signed ICF prior to any study-mandated procedure

Exclusion Criteria

Pregnant / planning to be become pregnant or lactating subject

The doctor thinks you are likely to show signs of organ problems during the study.

I have not had severe kidney issues, stroke, or heart failure that required a hospital stay during the previous study.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral lucerastat to evaluate long-term safety and tolerability

24-72 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may continue treatment until lucerastat is commercially available or until study completion

Long-term

Treatment Details

Interventions

• Lucerastat

Trial Overview The trial is testing the long-term safety of Lucerastat, an oral medication for adults with Fabry disease. It aims to understand how well patients tolerate this drug over an extended period following their initial treatment in a prior study.

Participant Groups

Group I: Lucerastat Experimental Treatment 1 Intervention

Dose will be based on subject's eGFR.

Who Is Running the Clinical Trial?

Idorsia Pharmaceuticals Ltd.

Lead Sponsor

Trials

124

Recruited

36,400+

Antonio Olivieri

Idorsia Pharmaceuticals Ltd.

Chief Medical Officer since 2024

Not specified

André C. Muller

Idorsia Pharmaceuticals Ltd.

Chief Executive Officer

Not specified