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Study on the Effects of Fipaxalparant in Patients with Diffuse Cutaneous Systemic Sclerosis

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Diffuse Cutaneous Systemic Sclerosis, a rare disease that affects the skin and internal organs by causing them to harden and tighten. The study will evaluate a treatment called HZN-825, also known by its code name Fipaxalparant. This medication is taken in the form of a tablet and is being tested to see if it can help improve the symptoms of this disease.

The purpose of the study is to assess how effective and safe HZN-825 is for patients with this condition. Participants in the study will receive either the medication or a placebo, and their progress will be monitored over a period of 52 weeks. The main focus will be on changes in lung function, specifically looking at something called forced vital capacity (FVC), which measures how much air a person can exhale after taking a deep breath.

Throughout the study, participants will have regular check-ups to monitor their health and any changes in their condition. The study aims to provide valuable information on whether HZN-825 can be a beneficial treatment option for people living with Diffuse Cutaneous Systemic Sclerosis.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the patient provides written informed consent. Eligibility is confirmed based on specific criteria, including age, diagnosis, and disease characteristics.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to establish baseline measurements. This includes evaluating lung function through a test called forced vital capacity (FVC) and assessing skin involvement.

  3. Step 3

    Treatment phase

    The patient receives the study medication, HZN-825, or a placebo. The medication is administered in the form of a film-coated tablet, taken orally. The treatment duration is 52 weeks.

    The dosage and frequency of administration are determined by the study protocol, which may involve one or two dose regimens.

  4. Step 4

    Ongoing assessments

    Throughout the treatment phase, regular assessments are conducted to monitor the patient's health and response to the medication. These assessments include lung function tests, skin evaluations, and other health-related quality of life measures.

  5. Step 5

    Final evaluation

    At the end of the 52-week treatment period, a final evaluation is performed. This includes a comparison of the change in FVC from the beginning of the study to the end, as well as other health assessments.

Who can join the trial?

9 criteria

  • Provide written informed consent, which means you agree to participate after understanding the study details.
  • Be a male or female between the ages of 18 and 75 years at the time of screening.
  • Meet the 2013 classification criteria for **Systemic Sclerosis (SSc)**, with a total score of 9 or more. **Systemic Sclerosis** is a condition that affects the skin and other organs.
  • Have skin involvement near the elbow and/or knee, which is a specific type of **Systemic Sclerosis** called diffuse cutaneous SSc.
  • Have had the first signs of **Systemic Sclerosis** (other than **Raynaud's phenomenon**, which is a condition causing cold fingers and toes) for 6 years or less at the time of enrollment.
  • Have skin on the forearm that is suitable for a repeat biopsy, which is a small sample of skin taken for testing. This is only for the first 110 participants who will have a biopsy.
  • Have a **modified Rodnan skin score (mRSS)** of 15 or more at screening. This score measures skin thickness.
  • Have a **forced vital capacity (FVC)** of 45% or more at screening, as determined by spirometry. **Forced vital capacity** is a measure of lung function.
  • Be willing and able to follow the treatment plan and attend evaluations for the entire duration of the trial.

Who cannot join the trial?

10 criteria

  • Patients who have a different condition than Diffuse Cutaneous Systemic Sclerosis cannot participate.
  • Patients who are not within the specified age range cannot participate.
  • Patients who are not able to follow the study procedures cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have another serious illness that might interfere with the study cannot participate.
  • Patients who have had a recent infection or surgery that might affect the study results cannot participate.
  • Patients who are taking certain medications that might interfere with the study cannot participate.
  • Patients who have a history of drug or alcohol abuse cannot participate.
  • Patients who have participated in another clinical trial recently cannot participate.
  • Patients who have allergies to the study medication cannot participate.
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Investigated drugs

HZN-825 is a medication being studied for its potential to help patients with diffuse cutaneous systemic sclerosis, a condition that affects the skin and internal organs. The trial aims to see if HZN-825 can improve lung function over a period of 52 weeks. The medication is being tested in different dose regimens to determine its effectiveness and safety for patients with this condition.

What is already known about the treatment

HZN-825 – This medication is administered orally and is currently being studied in clinical trials for its effectiveness in treating diffuse cutaneous systemic sclerosis. It is not yet widely recognized in medical literature as a standard treatment, as it is still under investigation. The main therapeutic indication for HZN-825 is to improve lung function in patients with this condition, as measured by changes in forced vital capacity. At the molecular level, HZN-825 is believed to work by modulating specific pathways involved in fibrosis, although the exact mechanism is still being researched. It is classified pharmacologically as an anti-fibrotic agent.

Investigated diseases

Diffuse Cutaneous Systemic Sclerosis – This is a rare autoimmune disease characterized by the hardening and tightening of the skin and connective tissues. It primarily affects the skin but can also involve internal organs such as the lungs, heart, and kidneys. The disease progresses with the overproduction of collagen, leading to thickened skin and potential organ dysfunction. Symptoms may include skin thickening, joint pain, and reduced mobility. As the disease advances, it can cause significant changes in skin texture and flexibility. The progression and severity of symptoms can vary widely among individuals.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-509782-20-00Protocol codeHZNP-HZN-825-301Estimated enrolment300 patientsSponsorHorizon Therapeutics Ireland Designated Activity Company

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