Long-term Safety of Lucerastat for Fabry Disease

Recruiting in Palo Alto (17 mi)

+76 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Idorsia Pharmaceuticals Ltd.

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

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.

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

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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.

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Participant Groups

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.

1Treatment groups

Experimental Treatment

Group I: LucerastatExperimental Treatment1 Intervention

Dose will be based on subject's eGFR.