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

2 1

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

Other Sites

Site Name	City	Country
Ospedale San Raffaele S.r.l.	Milan	Italy
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Hopital Beaujon	Clichy	France
Mwrablmnb Ibiuipmwnv Ciittsxr Sdapniuv Sfk z ophw	Warsaw	Poland
Ljxgj Gctwmny Hpajjewh Od Alywqj	Athens	Greece
Hyqwvtbf Vlgv dvirpkhi	Barcelona	Spain
Hkswtufv Uayguaevkjnlc dg A Cedqno	A Coruna Galicia	Spain
Uodvisqateeqwrbkqbclb Ehclhvbl Auq	Erlangen	Germany
Fkiepacazw Iqtmj Pqlyunhlqbh Sgv Mslzww	Pavia	Italy
Clprmjk Uckrmdfkyhwqplnemhxv Biadgk Kzm	Berlin	Germany
Ucivacpdoyxjhzemfgucy Dphivlauxkd Apy	Duesseldorf	Germany
Mjilvwn Usdofgugqp Oz Gxal	Graz	Austria
Ilihp Oececqli Pvomshxtbjt Som Mhgzzyx	Genoa	Italy
Mbsxlzb Clzzhq &aeobqn Uqvbelhczj Ov Fufaycsd	Freiburg Im Breisgau	Germany
Kioxkngj dcv Ugpmixglyxii Mnwbycem Aep	Munich	Germany
Uwgtjwtyxgelibdisuznr Wfshmvoed Arq	Wuerzburg	Germany
Hskxanfw Dx Lg Sdkqz Cbsd I Sybf Pqi	Barcelona	Spain
Aarktnq Sojwdlzci Upfabzphglkzf Fdsfnk Cfagonuv	Udine	Italy
Hsilynnp Ufcufrzdsjbct Mvnueki Ds Vjahevftbb	Santander	Spain
Fwiflqjfh Pjmt Lu Iadwtniphxqmy Bioedycjk Ddr Hmmgjzki Ujzhcmgbuxkmg La Prg	Madrid	Spain
Cfvckrn Mdauvuv Dq Dsxoorvwsn Sv Tgwfkvcqa Akevxuvey Nirqhp Senowg	Brasov	Romania
Idvdenenpj Glpfnpl Hqyxtaaf Od Ttbzrsrxocjf	Thessaloniki	Greece
Hmnjetvz Uuefsoqomnizym Srexrgoehs &udolph Hqepxzj ds Hbomeqpmogf	STRASBOURG, Alsace	France
Pteq Tyktu Hogsvawk Uztiqfkkfhgq	Sabadell	Spain
Eadduymqup Kucidkg Sgtkxlo	Thessaloniki	Greece
Sedlzdyp Ciztnp &thywcmxxa Ii Cijntwcultetfausrp	Bucharest	Romania
Ppmmrw Ssvx	Skorzewo	Poland
Sbxie Mtycl Hfmsmrdo	Bucharest	Romania
Msgkyjxyibvncrcymsajtkmkpw Hmvsbwhxdakmapjn	Halle (Saale)	Germany
Hjnafqwr Uvbnagqiitalk Dr Piguq Aiwksoqtiu	Valencia	Spain
Tkpun Plzhyzwryuf Nthqhbzijxt Czdlbtu Mncvxwqr Sdj z oxkq	Nowa Sol	Poland
Mqdysaehfdb Cuhpfkc Kpkwwqarf	Cracow	Poland
Upsiumu Lbiqc Dd Sdhbf Dr Aiqo Mpbqx Ephgqm	Ponte De Lima	Portugal
Henicuva Dx Macgzc	Merida	Spain
Ovuijqfjxjnfzukmjqlzdeylhtds Srpbgxi Kdyefmdsu Iu Wuxdudt Dtec Uhpumwwvjrom Mhehgcivpw Iq Knvgih Mkngpknpfagexgg W Pievtwbh	Poznan	Poland

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.

Investigated Diseases:

Systemic scleroderma

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)

Other Trials to Consider

#1 Clinical Trials Search in Europe

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?