TEPEZZA for Scleroderma

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effects of TEPEZZA for individuals with diffuse cutaneous systemic sclerosis (dcSSc), a condition that thickens and tightens the skin. Researchers aim to observe how TEPEZZA influences proteins and markers related to inflammation and scarring. The trial compares TEPEZZA to a placebo (a substance with no active drug) to determine its true impact. Individuals with dcSSc and noticeable skin thickening for less than five years might be suitable candidates for this trial. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

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.

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

Previous studies have shown that TEPEZZA can cause some side effects. Common ones include fatigue, high blood sugar, and hearing problems. Some participants also reported muscle cramps, nausea, and changes in taste.

Serious side effects are less common but may include hearing loss and reproductive system issues. TEPEZZA is already approved for treating Thyroid Eye Disease (TED), providing some safety information from its use in that condition.

While these studies offer insights, this trial aims to evaluate TEPEZZA specifically for diffuse cutaneous systemic sclerosis. Always consult a doctor to understand the risks and benefits for your situation. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for diffuse cutaneous systemic sclerosis (dcSSc) focus on managing symptoms and slowing disease progression through immunosuppressive drugs like methotrexate and mycophenolate mofetil. But TEPEZZA stands out because it targets the insulin-like growth factor-1 receptor (IGF-1R), which is thought to play a role in the fibrotic process of dcSSc. This mechanism of action is different from current options, potentially offering a more direct approach to addressing the underlying fibrosis. Researchers are excited about TEPEZZA because it could offer a new pathway to effectively reduce skin thickening and improve quality of life for patients with dcSSc.

What evidence suggests that TEPEZZA might be an effective treatment for diffuse cutaneous systemic sclerosis?

Research has shown that TEPEZZA (teprotumumab-trbw) may alleviate inflammation and other symptoms in conditions like diffuse cutaneous systemic sclerosis (dcSSc). In studies involving individuals with thyroid eye disease, nearly 90% experienced improvements in inflammation and eye symptoms. TEPEZZA blocks a specific receptor involved in inflammation and tissue changes. Although these results pertain to a different condition, they suggest that TEPEZZA might also reduce inflammation and tissue thickening in dcSSc. However, further research is necessary to confirm its effectiveness for dcSSc. Participants in this trial will receive either TEPEZZA or a placebo to assess its potential benefits for dcSSc. ^{6 7 8 9 10}

Who Is on the Research Team?

MD

Principal Investigator

Amgen

Are You a Good Fit for This Trial?

Inclusion Criteria

Written informed consent.

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

Skin thickening from dcSSc in the forearm suitable for repeat biopsy.

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 for a Trial Participant

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)

What Are the Treatments Tested in This Trial?

Interventions

• TEPEZZA

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

Citations

1. tepezzahcp.com tepezzahcp.com/about-tepezza/tepezza-efficacy

TEPEZZA® for HCPs - TEPEZZA® (teprotumumab-trbw) Efficacy DIPLOPIA · 2X more patients had complete diplopia resolution vs placebo · INFLAMMATION · Almost 3X more patients experienced reduction in inflammation · MRI · In 6 ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38824618/

Long-Term Efficacy of Teprotumumab in Thyroid Eye Disease Inflammatory and ophthalmic composite outcome improvements were seen in 90% of patients with nearly 70% reporting improvement in diplopia and ...

3. nejm.org nejm.org/doi/full/10.1056/NEJMoa1910434

Teprotumumab for the Treatment of Active Thyroid Eye ... Teprotumumab resulted in better outcomes with respect to proptosis, Clinical Activity Score, diplopia, and quality of life than placebo; serious adverse events ...

4. academic.oup.com academic.oup.com/jcem/article/109/1/25/7334391

Efficacy and Safety of Teprotumumab in Patients With Thyroid ... These data demonstrate that teprotumumab significantly reduced proptosis from baseline with a higher rate of proptosis response compared with ...

5. liebertpub.com liebertpub.com/doi/10.1089/thy.2023.0656

Long-Term Efficacy of Teprotumumab in Thyroid Eye Disease Inflammatory and ophthalmic composite outcome improvements were seen in 90% of patients with nearly 70% reporting improvement in diplopia and proptosis. Further ...

6. tepezzahcp.com tepezzahcp.com/about-tepezza/safety-and-adverse-reactions

Safety & Adverse Reactions - TEPEZZA (teprotumumab-trbw) Safety Profile of TEPEZZA ; Fatigue · 10 (12%), 6 (7%) ; Hyperglycemia · 8 (10%), 1 (1%) ; Hearing impairment · 8 (10%), 0 ; Dysgeusia (taste disturbance), 7 (8%), 0.

7. msds.amgen.com msds.amgen.com/tepezza-safety-datasheet

TEPEZZA® Wash hands, face and other potentially exposed areas immediately after handling this material. Page 3 / 8. Page 4. TEPEZZA®. Safety Data Sheet.

8. tepezza.com tepezza.com/about-tepezza/side-effects-and-safety

TEPEZZA Side Effects and Safety Information The most common side effects of TEPEZZA include muscle cramps or spasms, nausea, hair loss, diarrhea, feeling tired, high blood sugar, hearing problems, taste ...

9. academic.oup.com academic.oup.com/jcem/article/110/1/159/7693917

Postmarketing Safety Concerns of Teprotumumab: A Real ... This study identified ear and labyrinth disorders, and reproductive system and breast disorders, as specific safety signals of teprotumumab.

10. file.medchemexpress.com file.medchemexpress.com/batch_PDF/HY-P99165/Teprotumumab-SDS-MedChemExpress.pdf

Safety Data Sheet Provide accessible safety shower and eye wash station. Personal protective equipment. Eye protection. Safety goggles with side-shields. Hand ...