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Substrate Reduction Therapy

Long-term Safety of Lucerastat for Fabry Disease

Phase 3

Waitlist Available

Research Sponsored by Idorsia Pharmaceuticals Ltd.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline to month 24, month 48 and month 72 (duration: up to 6 years)

Awards & highlights

Study Summary

This trial will study the long-term effects of taking lucerastat, a medication used to treat Fabry disease.

Who is the study for?

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.Check my eligibility

What is being tested?

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.See study design

What are the potential side effects?

While specific side effects are not listed here, the goal of the trial is to monitor how safe and tolerable Lucerastat is when taken by adults with Fabry disease over a long duration.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to month 24, month 48 and month 72 (duration: up to 6 years)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline to month 24, month 48 and month 72 (duration: up to 6 years)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to month 24, month 48 and month 72 (duration: up to 6 years) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Treatment-emergent adverse events (AEs)

Other outcome measures

Change in left ventricular mass index (LVMI)

Change in plasma globotriaosylceramide (Gb3)

Subject estimated glomerular filtration rate (eGFR) slope

+1 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: LucerastatExperimental Treatment1 Intervention

Dose will be based on subject's eGFR.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Lucerastat

Not yet FDA approved

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Idorsia Pharmaceuticals Ltd.Lead Sponsor

119 Previous Clinical Trials

34,445 Total Patients Enrolled

2 Trials studying Fabry Disease

196 Patients Enrolled for Fabry Disease

Clinical TrialsStudy DirectorIdorsia Pharmaceuticals Ltd.

2,201 Previous Clinical Trials

888,496 Total Patients Enrolled

1 Trials studying Fabry Disease

182 Patients Enrolled for Fabry Disease

Media Library

Lucerastat (Substrate Reduction Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03737214 — Phase 3

Fabry Disease Research Study Groups: Lucerastat

Fabry Disease Clinical Trial 2023: Lucerastat Highlights & Side Effects. Trial Name: NCT03737214 — Phase 3

Lucerastat (Substrate Reduction Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03737214 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are we still enrolling individuals for this experiment?

"According to the latest update on clinicaltrials.gov, this study is no longer looking for patients as of November 9th, 2022. This particular trial was first posted on December 18th, 2018. There are presently 24 other trials that might be searching for participants."

Answered by AI

What other research has been done on Lucerastat in the past?

"Lucerastat was first studied in 2018 at the London Health Sciences Centre - Victoria Hospital. To date, there have been six completed studies. Presently, one clinical trial is active with many of these trials taking place in Calgary and Utah."

Answered by AI

Are there any dangers associated with Lucerastat?

"Lucerastat received a score of 3 because it is a Phase 3 trial, and thus there is both efficacy and safety data available."

Answered by AI

How many people are being signed up for this clinical trial?

"As of now, this study is no longer recruiting patients. The trial was first announced on December 18th, 2018 but has not been updated since November 9th, 2022. There are other clinical trials investigating potential treatments for Fabry Disease and one looking into Lucerastat that are still actively enrolling participants."

Answered by AI

Are there many locations in the United States where this study is taking place?

"There are a total of 19 sites conducting this research at the moment, with locations in Calgary, Salt Lake City, Boston and 16 other cities. If you choose to participate in this study, it would be advantageous to select the site nearest you to limit travel."

Answered by AI

Is this a new clinical trial?

"Lucerastat has undergone clinical trials since 2018, with the first being completed that same year. Idorsia Pharmaceuticals Ltd. sponsored the initial study which involved 108 participants. After Phase 3 approval in 2018, there is only one active trial for Lucerastat currently taking place."

Answered by AI

Recent research and studies

Molecular Genetics and Metabolism ReportsJournal

Understanding and Modifying Fabry Disease: Rationale and Design of a Pivotal Phase 3 Study and Results from a Patient-reported Outcome Validation Study

Wanner, Christoph, Virginia Kimonis, Juan Politei, David G. Warnock, Nurcan Üçeyler, Aline Frey, Peter Cornelisse, and Derralyn Hughes. 2022. “Understanding and Modifying Fabry Disease: Rationale and Design of a Pivotal Phase 3 Study and Results from a Patient-reported Outcome Validation Study”. Molecular Genetics and Metabolism Reports. Elsevier BV. doi:10.1016/j.ymgmr.2022.100862.

clinicaltrials.govStudy

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

2018. "A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03737214.

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