Long-term Safety of Lucerastat for Fabry Disease

Not currently recruiting at 80 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the safety and tolerability of a drug called lucerastat for people with Fabry disease, a genetic disorder affecting the heart, kidneys, and skin. The focus is on patient responses to long-term use of lucerastat. A special sub-study examines how the drug affects the kidneys of men with the classic form of Fabry disease. Individuals who completed a previous 6-month treatment study with lucerastat and have Fabry disease might be suitable candidates for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new treatment.

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.

Is there any evidence suggesting that lucerastat is likely to be safe for humans?

Research has shown that lucerastat is generally safe for adults with Fabry disease. In earlier studies, most participants did not experience serious side effects. Some reported mild issues like stomach discomfort or headaches, but these were not severe.

Lucerastat has undergone extensive testing, including studies on long-term safety. These studies suggest that people can use lucerastat for extended periods without major problems. The treatment works to lower harmful substances in the body, and its safety has been carefully monitored.

Lucerastat's progression through advanced testing stages indicates strong safety evidence. Researchers have gathered enough information to be confident about its general safety for treating Fabry disease. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Fabry disease?

Unlike the standard treatments for Fabry Disease, which generally involve enzyme replacement therapy, Lucerastat offers a novel approach by targeting substrate reduction. Lucerastat works by inhibiting the production of specific lipids that accumulate due to the disease, potentially reducing symptoms and organ damage in a different way than current therapies. Researchers are excited about Lucerastat because it is administered orally, which could provide a more convenient option compared to the intravenous infusions required by existing treatments. This unique mechanism and delivery method may improve the quality of life for patients with Fabry Disease.

What evidence suggests that lucerastat might be an effective treatment for Fabry disease?

Research has shown that lucerastat, the investigational treatment in this trial, may help treat Fabry disease. Studies have found that lucerastat lowers levels of Gb3, a harmful substance, in cells from Fabry patients, regardless of their specific genetic mutation. Early results suggest that lucerastat can slow the worsening of kidney function, a major concern for those with Fabry disease. Additionally, the treatment is generally well tolerated, with most patients not experiencing severe side effects. Overall, lucerastat reduces harmful substances in the body, potentially leading to symptom relief and slower disease progression. ^{1 3 6 7 8}

Who Is on the Research Team?

Clinical Trials

Principal Investigator

Idorsia Pharmaceuticals Ltd.

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03425539

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult ... Study Overview. Brief Summary. This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9248229/

Understanding and modifying Fabry disease - PubMed Central In Fabry patient fibroblasts, lucerastat resulted in a decrease of Gb3 and lysosomal staining irrespective of mutation type [24].

3. idorsia.com idorsia.com/investors/news-and-events/media-releases/media-release-details?id=2608540

Idorsia | Media release Lucerastat was well tolerated and biochemically it did exactly what we were expecting; as previously seen, in this study we saw a substantial ...

4. asn-online.org asn-online.org/education/kidneyweek/2024/program-abstract.aspx?controlId=4130405

Kidney Week This unexpected renal effect was confirmed at Month-18, where lucerastat markedly slowed eGFR decline in the 93 participants with pre-randomization eGFR data, ...

5. ojrd.biomedcentral.com ojrd.biomedcentral.com/articles/10.1186/s13023-020-01582-7

The effect of the glucosylceramide synthase inhibitor ... The desired outcome is a reduction of net globotriaosylceramide load in tissues leading to symptomatic improvement and a delayed progression ...

6. clinicaltrials.eu clinicaltrials.eu/trial/study-on-long-term-safety-of-oral-lucerastat-for-adults-with-fabry-disease/

Study on Long-Term Safety of Oral Lucerastat for Adults ... This study analyzes the long-term safety and tolerability of oral Lucerastat (ACT-434964) in treating adults with Fabry disease, ...

7. idorsia.com idorsia.com/our-innovation/portfolio/lucerastat.html

Lucerastat This demonstrates that lucerastat has the potential to reduce Gb3 levels in key target organs and, therefore, to show clinical efficacy in Fabry disease.

8. clinicaltrials.gov clinicaltrials.gov/study/NCT02930655

A Study to Assess the Safety and Tolerability of Lucerastat ... The primary purpose of this study was to assess the safety and tolerability of lucerastat in adults with Fabry Disease receiving Enzyme Replacement Therapy ...