HZN-825 for Scleroderma

No longer recruiting at 115 trial locations

Overseen By Horizon Therapeutics

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Amgen

Must not be taking: Investigational agents

Disqualifiers: Malignant condition, Pregnancy, Non-compliance, others

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

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

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called HZN-825 for individuals with scleroderma, a condition that can harden and tighten the skin. The researchers aim to evaluate the treatment's effectiveness over a year and assess its safety, particularly by monitoring breathing ability. Participants should have previously taken part in a related study and completed specific assessments. This trial is not suitable for those planning to use other experimental treatments or who have developed new serious health issues. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to early-stage findings.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, you cannot use another investigational agent during the trial.

Is there any evidence suggesting that HZN-825 is likely to be safe for humans?

Research has shown that HZN-825 has been studied for safety and tolerability. In earlier studies, participants with diffuse cutaneous systemic sclerosis took HZN-825 for a year. These studies examined safety concerns, such as unwanted side effects or more serious problems. Although detailed results are not yet available, ongoing research indicates that researchers are closely monitoring the treatment for any negative effects. The advancement of HZN-825 to this stage of study suggests confidence in its safety for further testing. However, participants should remain vigilant and report any issues during the trial. ^{1 2 3 4 5}

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

HZN-825 is unique because it is administered orally, which is a convenient alternative to the more common intravenous treatments used for scleroderma. Unlike current treatments that primarily focus on managing symptoms, HZN-825 targets the underlying disease process by modulating the lysophosphatidic acid receptor 1 (LPA1), which is believed to play a role in fibrosis. This novel mechanism of action offers the potential to not only alleviate symptoms but also slow disease progression, generating excitement among researchers for its potential benefits.

What evidence suggests that HZN-825 might be an effective treatment for scleroderma?

Research has shown that HZN-825, the treatment under study in this trial, may help treat diffuse cutaneous systemic sclerosis. Studies have found that patients taking HZN-825 experienced improvements, including a noticeable decrease in skin thickness, a common issue with this disease. The treatment also appears to aid breathing by improving lung function, as indicated by better results for forced vital capacity (FVC), a measure of lung performance. While further research is ongoing, these early findings suggest HZN-825 could be a helpful option for managing this challenging condition. ^{1 2 6 7 8}

Who Is on the Research Team?

MD

Principal Investigator

Amgen

Are You a Good Fit for This Trial?

This trial is for adults who've completed a previous HZNP-HZN-825-301 trial for diffuse cutaneous systemic sclerosis, even if they stopped early for non-safety reasons. It's not open to pregnant women, those at reproductive age not using birth control, or anyone with new risks that could make the trial unsafe.

Inclusion Criteria

Completed the double-blind Treatment Period (Week 52) in Trial HZNP-HZN-825-301

Participants prematurely discontinued from trial drug in Trial HZNP-HZN-825-301 for reasons other than safety or toxicity can be included at the discretion of the Investigator after completing Trial HZNP-HZN-825-301 scheduled visits, including Week 52 assessments.

Exclusion Criteria

I can follow the trial's requirements and have no health issues that could interfere.

I agree to use effective birth control during and for 4 weeks after the trial.

Any new development with the participant's disease or condition or any significant laboratory test abnormality during the course of Trial HZNP-HZN-825-301 that, in the opinion of the Investigator, would potentially put the subject at unacceptable risk

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive open-label treatment with HZN-825 for 52 weeks

52 weeks

Regular visits as per trial protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• HZN-825

Trial Overview The study tests HZN-825 over 52 weeks in patients with diffuse cutaneous systemic sclerosis. The focus is on lung function improvement (FVC %) and safety by monitoring adverse events up to four weeks after the last dose.

How Is the Trial Designed?

Group I: HZN-825 Experimental Treatment 1 Intervention

HZN-825 will be administered by mouth (PO) twice daily (BID) for 52 weeks

Who Is Running the Clinical Trial?

Amgen

Lead Sponsor

Trials

1,508

Recruited

1,433,000+

Founded

1980

Headquarters

Thousand Oaks, USA

Known For

Human Therapeutics

Top Products

Enbrel, Prolia, Neulasta, Otezla

Robert A. Bradway

Amgen

Chief Executive Officer since 2012

MBA from Harvard Business School

Paul Burton

Amgen

Chief Medical Officer since 2023

MD from University of London, PhD in Molecular and Cellular Biology from Imperial College London

Horizon Pharma Ireland, Ltd., Dublin Ireland

Lead Sponsor

Trials

31

Recruited

3,700+

Published Research Related to This Trial

Biological drugs like tocilizumab and belimumab show promising trends in improving pulmonary function and skin fibrosis in patients with diffuse cutaneous systemic sclerosis (dcSSc), based on a systematic review of 6 controlled trials involving 426 patients.

While these treatments may enhance anti-fibrotic activity, they did not demonstrate significant benefits in overall health status as measured by the Health Assessment Questionnaire-Disability Index compared to control groups.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Impact of Innovative Treatment Using Biological Drugs for the Modulation of Diffuse Cutaneous Systemic Sclerosis: A Systematic Review. Fernández-Lázaro, D., Iglesias-Lázaro, M., Garrosa, E., et al.[2023]

A 48-week study involving 146 patients with scleroderma found that aminobenzoate potassium (KPAB) did not significantly improve skin mobility or thickening compared to a placebo, indicating it may not be effective for this condition.

While KPAB was generally well tolerated, with a compliance rate of over 75%, some patients experienced adverse drug reactions, primarily gastrointestinal issues and headaches, leading to withdrawals from the study.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparison of aminobenzoate potassium and placebo in the treatment of scleroderma. Clegg, DO., Reading, JC., Mayes, MD., et al.[2013]

In a study involving 32 mice with bleomycin-induced scleroderma, etanercept significantly reduced markers of dermal sclerosis, including serum TGF-beta1 levels and collagen accumulation, indicating its potential efficacy in managing scleroderma.

Thalidomide did not show significant effects in this model, suggesting that targeting TNF-alpha with etanercept may be a more effective approach for treating the condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness of etanercept in bleomycin-induced experimental scleroderma. Koca, SS., Isik, A., Ozercan, IH., et al.[2015]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04781543

NCT04781543 | A Multicenter Trial to Evaluate the Efficacy ... A Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients With Diffuse Cutaneous Systemic Sclerosis.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05626751

An Open-label Extension Trial of HZNP-HZN-825-301 in ... The primary efficacy objective is to assess the efficacy of 52 weeks of open-label treatment with HZN-825 in participants with diffuse cutaneous systemic ...

3. youtube.com youtube.com/watch?v=P30kbEWFlfY

Phase 2b Trial Testing HZN-825 to Treat Diffuse Cutaneous ... Phase 2b Trial Testing HZN-825 to Treat Diffuse Cutaneous Systemic Sclerosis Has Begun.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11319916/

Prediction of Disease Progression and Clinical Response ... Maximum decreases from baseline in mRSS were 50.4% (ziritaxestat) and 34.7% (placebo). Study designs based on 300 patients randomized 2:1 or 1:1 to 600 mg of ...

5. horizonscandb.pcori.org horizonscandb.pcori.org/report/topics/628

Fipaxalparant (AMG 670) to treat diffuse cutaneous ... Fipaxalparant (formerly AMG 670/HZN-825) is an oral small-molecule drug under clinical development to treat diffuse cutaneous systemic sclerosis.

6. ctv.veeva.com ctv.veeva.com/study/a-multicenter-trial-to-evaluate-the-efficacy-safety-tolerability-and-pharmacokinetics-of-hzn-825-i

A Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability ... A Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients With Diffuse Cutaneous Systemic ...

7. clinicaltrialsregister.eu clinicaltrialsregister.eu/ctr-search/trial/2021-006271-42/PT

Clinical Trials Register The primary safety objective is to examine the safety and tolerability of 52 weeks of open-label treatment with HZN 825, inclusive of, but not ...

8. amgentrials.com amgentrials.com/study/?id=HZNP-HZN-825-302

An Open-label Extension Trial of HZNP-HZN-825-301 in ... 2. The primary safety objective is to examine the safety and tolerability of 52 weeks of open-label treatment with HZN-825, inclusive of, but not limited to, ...