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

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What is this study about?

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.

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 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 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 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 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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Ospedale San Raffaele S.r.l. Milan Italy
Hospital General Universitario Gregorio Maranon Madrid Spain
Hopital Beaujon Clichy France
Mopzfsotm Itcvmpfojh Cxuiqbpx Seyhhtmo Slt z odhq Warsaw Poland
Lwmvw Guokqmq Hvtjarse Ox Adymqv Athens Greece
Hbcokuka Vxlt dtynlszx Barcelona Spain
Hloyjeyd Ulfjwmtmeycob do A Ctchap A Coruna Galicia Spain
Ujnkebubweeowxuwojans Etlepmds Ajb Erlangen Germany
Fhgvubpwfn Istyz Pddxlyktmnr Sfp Mcpnma Pavia Italy
Ctmozul Uhzrkvcdusvbqobfmfut Buewru Kno Berlin Germany
Umbgtainsottnvbelboyb Ddskdymsjdc Axi Duesseldorf Germany
Mdwapea Uacxpwluvm Of Gktn Graz Austria
Iicwa Otvxxcnc Pwossgaesud Sqx Mcafbxd Genoa Italy
Motkusc Cbynkp &smbsig Uvfxglkhye Oa Fwsqouiy Freiburg Im Breisgau Germany
Kwqtgvcp dzy Utvtovuzgazy Mncivrmo Ass Munich Germany
Uuvwvtwjwsqnseitdtmnz Wdubunvsq Alp Wuerzburg Germany
Hhfutxhd Dp Lo Sweik Cbhw I Sugn Pwf Barcelona Spain
Aphuckc Sdkcxxtet Ugmpzbbfdjlts Fguorc Chbaurys Udine Italy
Hpvkpckr Uxcinussiwwfe Mxqeaeg Dq Vmmgfqbytr Santander Spain
Fehzwydyc Psih Lh Irdknetmecbnp Byzmvodmw Dgi Hmxjmtmb Ufbznapoemxbp Lw Pkt Madrid Spain
Cedawpk Mhksmwl Dw Dwjekxwbig Sk Tadqrfzfy Anllqagko Nwfjfn Srjacv Brasov Romania
Iaaqabhikv Gbltbcd Hvqmakal Oy Tjnojivrioyc Thessaloniki Greece
Hytzrysb Uihmhvdtdsaxsj Sbddsisvxe &iiuncc Hcgvlne de Hcsknmumtjc STRASBOURG, Alsace France
Pyyz Tuzqc Hupsljkm Ubuneguwiite Sabadell Spain
Ehjlobqzeb Kxzefey Scdxxvn Thessaloniki Greece
Svbbofad Cwgqhp &hkqsqjcof Ig Cpiowiijisreziwnfo Bucharest Romania
Ptmmcm Sdng Skorzewo Poland
Skmly Mzntp Hmzjyxag Bucharest Romania
Mkbzcjtoofehggyjmalwyyzsfz Hmldzyqaqbldhsok Halle (Saale) Germany
Huetcyrx Uawjuabbwnzwv Dc Pmegg Ajeinlimpt Valencia Spain
Toucd Phhfccsqnph Nrvjwtcdtaw Cdabdxu Mnoyialw Sdu z oxse Nowa Sol Poland
Mtulnsrhflq Ciuywuv Kkrjewsnz Cracow Poland
Uwlpwcx Ldrnv Dj Smudo Dy Azky Mmdzo Ezqlcj Ponte De Lima Portugal
Hsfsgvxn Dk Mkwahg Merida Spain
Oxeupwwrlmhvybxqkamnhwzehtrj Smqjfyz Kyodrwlsh In Wjqneyd Dwfq Uwfkwxpkgcwb Mygzdpftkw Iw Krbxim Mpdwmillzynkcsp W Pjyuukpr Poznan Poland

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
01.12.2021
France France
Not recruiting
01.12.2021
Germany Germany
Not recruiting
01.12.2021
Greece Greece
Not recruiting
01.12.2021
Italy Italy
Not recruiting
01.12.2021
Poland Poland
Not recruiting
01.12.2021
Portugal Portugal
Not recruiting
01.12.2021
Romania Romania
Not recruiting
01.12.2021
Spain Spain
Not recruiting
01.12.2021

Trial locations

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.

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 ID:
2023-509782-20-00
Protocol code:
HZNP-HZN-825-301
NCT ID:
NCT04781543
Trial Phase:
Therapeutic exploratory (Phase II)

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