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

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
IRCCS Humanitas Research Hospital	Rozzano	Italy
CENTRUM MEDYCZNE REUMA PARK	Warsaw	Poland
Medical University Of Vienna	Vienna	Austria
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Centre Hospitalier Universitaire De Lille	Lille	France
Centro Hospitalar Universitario Sao Joao E.P.E.	Porto	Portugal
Futuremeds Sp. z o.o.	Wroclaw	Poland
Oncopole Claudius Regaud	Toulouse	France
Technische Universitaet Dresden	Dresden	Germany
Unidade Local De Saúde De Santa Maria, E.P.E.	Lisbon	Portugal

Other Sites

Site Name	City	Country
Saint Maria Hospital	Bucharest	Romania
Azienda Sanitaria Universitaria Friuli Centrale	Udine	Italy
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Hospital Universitario Dr Peset Aleixandre	Valencia	Spain
Twoja Przychodnia Nowosolskie Centrum Medyczne Sp. z o.o.	Nowa Sol	Poland
Unidade Local De Saude Do Alto Minho E.P.E.	Ponte De Lima	Portugal
Ippokratio General Hospital Of Thessaloniki	Thessaloniki	Greece
Hospital De Merida	Merida	Spain
Malopolskie Centrum Kliniczne	Cracow	Poland
Universitaetsklinikum Erlangen AöR	Erlangen	Germany
Ospedale San Raffaele S.r.l.	Milan	Italy
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
IRCCS Ospedale Policlinico San Martino	Genoa	Italy
Pratia S.A.	Skorzewo	Poland
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Hopital Beaujon	Clichy	France
Ortopedyczno-Rehabilitacyjny Szpital Kliniczny Im Wiktora Degi Uniwersytetu Medycznego Im Karola Marcinkowskiego W Poznaniu	Poznan	Poland
Mfmwvopwf Ipkdupoksh Czqqjpuw Sqwzysvi Svg z oibb	Warsaw	Poland
Hpewkbuv Utbbmxhqfqagc Mijlway Dg Vizdolrdes	Santander	Spain
Cdzjsoo Mfllaza Dr Dhafggsjdb Sl Tmsfsznhb Achfdibkh Niwkml Ssfgvu	Brasov	Romania
Ehthnwyhuf Knrcfmg Sycrjtf	Thessaloniki	Greece
Pzzk Tzdrw Hqjffwom Utjyxsgrxsgv	Sabadell	Spain
Lakma Gfedjiq Hxwbkxzg Or Amkmro	Athens	Greece
Sgdjepal Clgxec &puxqpbnui Ic Cwuycnvcvwysodbrxz	Bucharest	Romania
Ugubmsjjpbxcdjakgfsha Dcppfvmwdyk Aqg	Duesseldorf	Germany
Mjbibbq Uzqarxvoig Ob Goip	Graz	Austria
Kywqipcp ddt Ubivubyjuilr Mmwaguxz Aao	Munich	Germany
Uipycbyjibczgnqobculn Wmuoqgznr Ant	Wuerzburg	Germany
Hidkxrcz Dp La Sbnyv Cofl I Smhp Pdd	Barcelona	Spain
Mpivcvjjgwoweyupsiverirstr Hfwtpwombkaaibtp	Halle (Saale)	Germany
Fahwudmei Pevd Le Inhywxvesvtqz Bbllcyprv Dnc Hjofnhqw Uuhfomboewgis Lr Pjw	Madrid	Spain
Hpwdailn Vfpo djhunwvo	Barcelona	Spain
Hdfsiapx Usddefbippjfp dz A Cmymfj	A Coruna Galicia	Spain
Htrhtdjy Uatglfzpngzzng Sjfdugusgi &pmrnmm Hruibhq dt Hssdsodgwpc	STRASBOURG, Alsace	France

Trial status

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

Trial locations

Investigated drugs:

Fipaxalparant

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.

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

What is this study about?

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