A Study With TEPEZZA in Patients With Diffuse Cutaneous Systemic Sclerosis (dcSSc)

Recruiting at 9 trial locations

Overseen By HorizonTherapeutics

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Amgen

Must be taking: CellCept, Myfortic, Prednisone

Must not be taking: Rituximab, Immunosuppressives, Biologics

Disqualifiers: Limited SSc, Other autoimmune, Renal crisis, others

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 12

Duration: 24 weeks

25 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing TEPEZZA, a medicine that blocks a protein causing inflammation and scarring, in patients with a skin condition called diffuse cutaneous systemic sclerosis. The study will see if TEPEZZA is safe and effective when given over several months. The goal is to find out if it can help reduce symptoms in these patients.

Will I have to stop taking my current medications?

The trial allows participants to continue taking CellCept (mycophenolate mofetil) or Myfortic (mycophenolic acid) and low-dose prednisone if they have been on a stable dose for a specified period before the trial. Other non-steroidal immunosuppressive agents and certain biologics must be stopped before joining the trial.

What safety data exists for the treatment known as TEPEZZA?

The safety of marketed drugs, including those like TEPEZZA, is monitored for serious or life-threatening side effects. Safety-related information is curated in resources like ChEMBL, which includes data on drugs that have been withdrawn or have black box warnings (strongest safety warnings by the FDA). This information helps in understanding the general safety of drugs in humans. ^{1 2 3 4 5}

Research Team

MD

Principal Investigator

Amgen

Eligibility Criteria

Inclusion Criteria

Written informed consent.

You must have developed the first signs of dcSSc (except Raynaud's phenomenon) no more than 5 years ago.

You are male or female between the ages of 18 and 80 years, inclusive.

Exclusion Criteria

You have been diagnosed with autoimmune diseases, except for fibromyalgia, scleroderma associated myopathy, and Sjogren's syndrome.

Corticosteroid use for conditions other than dcSSc within 4 weeks prior to Screening (topical steroids for dermatological conditions and inhaled steroids are allowed)

Previous treatment with rituximab (Rituxan® or MabThera®) within 12 months prior to the first infusion

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

1 visit (in-person)

Treatment

Participants receive 8 infusions of TEPEZZA or placebo every 3 weeks for 24 weeks

24 weeks

8 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 weeks

3 visits (in-person), 2 contacts (phone or email)

Treatment Details

Interventions

• TEPEZZA

Participant Groups

Group I: TEPEZZA 20mg/kg Active Control 1 Intervention

Approximately 15 participants will receive 8 infusions of TEPEZZA q3W for a total of 21 weeks. TEPEZZA 10mg/kg will be administered on Day 1 and TEPEZZA 20mg/kg will be administered q3W for the remaining 7 infusions.

Group II: Placebo Placebo Group 1 Intervention

Approximately 10 participants will receive 8 infusions of placebo q3W for a total of 21 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 Therapeutics USA, Inc.

Lead Sponsor

Trials

4

Recruited

420+

Findings from Research

The safety of marketed drugs is a significant concern, as some commonly prescribed medications can lead to serious or life-threatening side effects in patients.

The ChEMBL resource will provide a curated drug safety data set, including toxicity classifications and black box warnings, which will be freely available and regularly updated to aid in drug safety research and discovery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drug Safety Data Curation and Modeling in ChEMBL: Boxed Warnings and Withdrawn Drugs. Hunter, FMI., Bento, AP., Bosc, N., et al.[2023]

Out of 123,129 reports of adverse drug reactions (ADRs) in Italy from 2001 to 2012, 8,338 (6.8%) were related to pediatric patients, with a notable 39.4% classified as serious, often requiring hospitalization, especially in very young children.

The most common drugs involved in these ADRs were anti-infectives (44.9%), followed by nervous system medications (15.6%) and anti-inflammatory drugs (10.2%), highlighting specific safety concerns in pediatric populations that differ from adults.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pediatric drug safety surveillance in Italian pharmacovigilance network: an overview of adverse drug reactions in the years 2001 - 2012. Ferrajolo, C., Capuano, A., Trifirò, G., et al.[2014]

In a review of 6579 medical records from New Zealand public hospitals in 1998, 12.9% of hospital admissions were associated with an adverse event, indicating a significant occurrence of safety issues in acute care settings.

Most adverse events had minor impacts on patients, with less than 15% leading to permanent disability or death, but they significantly increased hospital stays by an average of over nine days, highlighting the need for improved patient safety measures.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse events in New Zealand public hospitals I: occurrence and impact. Davis, P., Lay-Yee, R., Briant, R., et al.[2022]

A systematic review of 108 adverse drug event (ADE) reporting systems revealed 1782 unique data fields, highlighting significant variability in how ADEs are reported, which complicates data comparison and aggregation across different systems.

Despite consistent reporting concepts, the lack of standardized terminology and the use of multiple drug and disease dictionaries hinder effective drug safety monitoring, suggesting a need for a common standardized dataset to improve ADE reporting and surveillance.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse drug event reporting systems: a systematic review. Bailey, C., Peddie, D., Wickham, ME., et al.[2021]

References

1. United States pubmed.ncbi.nlm.nih.gov

Drug Safety Data Curation and Modeling in ChEMBL: Boxed Warnings and Withdrawn Drugs. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Pediatric drug safety surveillance in Italian pharmacovigilance network: an overview of adverse drug reactions in the years 2001 - 2012. [2014]

3. England pubmed.ncbi.nlm.nih.gov

Adverse drug events in hospitalized children at Ethiopian University Hospital: a prospective observational study. [2022]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Adverse events in New Zealand public hospitals I: occurrence and impact. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Adverse drug event reporting systems: a systematic review. [2021]