HZN-825 for Scleroderma

Recruiting at 92 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

174 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests HZN-825, a medication, in people with diffuse cutaneous systemic sclerosis. It aims to see if the drug can improve lung function and overall health by affecting the disease process.

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.

What data supports the effectiveness of the drug HZN-825 for treating scleroderma?

The systematic review of biological drugs for scleroderma suggests that these types of treatments, which include drugs like tocilizumab and belimumab, show trends of improvement in skin and lung function, indicating that similar drugs like HZN-825 might also be effective in treating scleroderma. ^{1 2 3 4 5}

How does the drug HZN-825 differ from other treatments for scleroderma?

HZN-825 is unique because it is being studied specifically for its potential to treat scleroderma, a condition with no current cure, by targeting the underlying disease process rather than just alleviating symptoms. Unlike some existing treatments that focus on symptom management, HZN-825 may offer a novel approach by addressing the disease's progression. ^{2 3 4 5 6}

Research Team

MD

Principal Investigator

Amgen

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Effectiveness of etanercept in bleomycin-induced experimental scleroderma. [2015]

2. Germany pubmed.ncbi.nlm.nih.gov

Collagen V nasal tolerance in experimental model of systemic sclerosis. [2007]

3. Canada pubmed.ncbi.nlm.nih.gov

Comparison of aminobenzoate potassium and placebo in the treatment of scleroderma. [2013]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Impact of Innovative Treatment Using Biological Drugs for the Modulation of Diffuse Cutaneous Systemic Sclerosis: A Systematic Review. [2023]

5. Canada pubmed.ncbi.nlm.nih.gov

The modified Rodnan skin score is an accurate reflection of skin biopsy thickness in systemic sclerosis. [2022]

6. Netherlands pubmed.ncbi.nlm.nih.gov

Transgenic analysis of scleroderma: understanding key pathogenic events in vivo. [2007]