Study on the Effectiveness and Safety of Efgartigimod IV for Adults with Primary Immune Thrombocytopenia

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What is this study about?

This clinical trial is focused on studying a condition known as Primary Immune Thrombocytopenia (ITP). ITP is a disorder that can lead to easy or excessive bruising and bleeding, resulting from unusually low levels of platelets, which are cells that help blood clot. The study will evaluate a treatment called efgartigimod, which is given through an intravenous (IV) infusion. Efgartigimod is designed to help manage the symptoms of ITP by potentially increasing platelet counts in the blood.

The purpose of this study is to assess how effective and safe efgartigimod is compared to a placebo. Participants in the study will be randomly assigned to receive either efgartigimod or a placebo, which is a substance with no active medication. The study will begin with a phase where neither the participants nor the researchers know who is receiving the actual treatment or the placebo. This is followed by an open-label phase where all participants will receive efgartigimod. The study will monitor participants over a period to see how well the treatment controls the disease by measuring platelet counts and observing any side effects.

Throughout the study, participants will have regular visits to check their platelet levels and overall health. The goal is to determine if efgartigimod can help maintain platelet counts at a safe level, reducing the risk of bleeding and improving the quality of life for those with ITP. The study will also look at how long it takes for the treatment to start working and how long the effects last. This research aims to provide valuable information on a new potential treatment option for people living with Primary Immune Thrombocytopenia.

1 randomization and initial treatment

Upon joining the study, participants are randomly assigned to receive either efgartigimod or a placebo. This process is double-blinded, meaning neither the participant nor the study team knows which treatment is being administered.

The treatment is administered through an intravenous infusion. The specific product used is Vyvgart 20 mg/mL concentrate for solution for infusion.

2 double-blinded treatment period

During this period, the effectiveness of efgartigimod is compared to the placebo. The main goal is to assess the control of the disease by measuring platelet counts.

Participants receive regular infusions and attend scheduled study visits to monitor their platelet levels and overall health.

3 open-label treatment period

After the double-blinded period, all participants enter an open-label phase where they receive efgartigimod.

This phase allows for further evaluation of the treatment’s long-term safety and effectiveness.

4 monitoring and follow-up

Throughout the study, participants undergo regular health assessments, including blood tests to measure platelet counts and other safety evaluations.

The study aims to track the incidence of any side effects and the overall response to the treatment over time.

Who Can Join the Study?

  • The patient must be at least 18 years old and meet the local legal age for participating in clinical studies when signing the informed consent form.
  • The patient must have a documented average platelet count of less than 30 x 109/L before being randomly assigned to a group in the study. Platelets are small blood cells that help with clotting.
  • The patient must have had primary immune thrombocytopenia (ITP) for more than 12 months by the time they sign the informed consent form. Primary immune thrombocytopenia (ITP) is a condition where the immune system attacks and destroys platelets.
  • The patient must have previously received at least one treatment for ITP, such as corticosteroids, intravenous immunoglobulin (IVIg), anti-D immunoglobulin (for those who have not had their spleen removed and are Rho(D)-positive), thrombopoietin receptor agonists (TPO-RAs), or rituximab.
  • The patient must have shown an insufficient response to a previous ITP treatment with corticosteroids, IVIg, anti-D immunoglobulin (for those who have not had their spleen removed and are Rho(D)-positive), TPO-RAs, or rituximab.
  • The patient must have had a previous response, defined as at least one platelet count of 50 x 109/L or higher, to at least one ITP treatment in the three years before signing the informed consent form. These treatments could include prednisone, dexamethasone, other or unspecified corticosteroids, IVIg, or anti-D immunoglobulin (for those who have not had their spleen removed and are Rho(D)-positive).

Who Cannot Join the Study?

  • Patients who have a different condition than Primary Immune Thrombocytopenia (ITP) cannot participate. ITP is a condition where the immune system attacks and destroys platelets, which are cells that help blood to clot.
  • Patients who are not within the specified age range for the study cannot participate. The age range is not specified here, but it is important for eligibility.
  • Patients who are not part of the specified clinical trial group cannot participate. This refers to specific characteristics or conditions that the study is focusing on.
  • Both male and female patients are eligible, so gender is not an exclusion criterion.
  • Patients who are considered part of a vulnerable population may not be eligible. Vulnerable populations include groups like children, pregnant women, or those unable to give consent.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Other Sites

Site Name City Country Status
Samodzielny Publiczny Szpital Kliniczny Nr 4 W Lublinie Lublin Poland
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University Hospital Ostrava Ostrava Czechia
Semmelweis University Budapest Hungary
Hopital Beaujon Clichy France
Klinikum Wels-Grieskirchen GmbH Wels Austria
Spitalul Clinic Coltea Bucharest Romania
University Multiprofile Hospital For Active Treatment St. Ivan Rilski EAD Sofia Bulgaria
Hniwjcrh Vues dqmogpxq Barcelona Spain
Dcnjnmibylcpegbaemkoqpc clmehj “oqtoyrpprhxmcad Enwx Sofia Bulgaria
Hjqmqxqu Uhqzwrvrixumm dq A Cnkghi A Coruna Galicia Spain
Fbduxnbkwv Ibjga Cu Gewuhj Ocbnbdww Mfzatzrg Pndgnasnfad Milan Italy
Aoqf Gzydjv Opspyqsk Mxdcprnzhdpfd Ngqeonle Milan Italy
Fekyvftw Ngudpunwh Bvqf Brno Czechia
Fcomnrmm Nxpmqjyot Kcujzczlt Vpgwjkzxn Prague Czechia
Aejndpc Otkmujtwnme Uqadogwhmjwmt Ceoirsgzlbgj Djelr Smzmdy E Dosax Sjdqanq Dg Tkjkhd Turin Italy
Kycgnn Uuhwbasrjscmjcmybbaou Gzvy Linz Austria
Aniudyc Oizpywzdbak Ulzspccndbzdn Fmugarag Io Dk Nskvmr Naples Italy
Apafuts Ojsyajvydahfwzvitxeakcwqn Mwwkwmcj Dbixm Cloevg Novara Italy
Cotimj Hivirhjvbrt Uwzcnluvgclid Aeuyhd Picwmzgq Amiens France
Ufnviavsdryfmzsanftyp Ecyeq Asq Essen Germany
Oqqkecxbdewfze Luiy Gkif Linz Austria
Ihtoplbc Cvvmyh Dcsijnzqtduufqsfb L'hospitalet De Llobregat Spain
Wihsapafvyb Wfylbwmscfjrvoyozrec Choxqis Odhxkkopr I Twypddjuzithm Iq Mpjqmcluimv W Laoyx Lodz Poland
Cpot Cnfnhz Cmnisyl Affdxeffc Bxaoz Aqyqkjwgcv Braga Portugal
Acjufia Svmsi Sxbemjvbk Tsfxdgyiofbv Dep Smhct Lqxgg Varese Italy
Pftyb Svdaundm Cxulvrht Ubzfujglgg Hpgdidku Riga Latvia
Upqkojrxze Mjoachbdxoeb Htvfzjkk Frq Agvrak Tjrshrxvi Sedkxrlu Owk Sofia Bulgaria
Ksi Zmfjdr Zagreb Croatia
Samuxel Uglirxnrcnhfu Nx 2 Io Di Joln Bvkmazg W Bznutsapsq Bydgoszcz Poland
Pebuez Hrbiugrmvtk Siw z oxuy Katowice Poland
Aodoqsa Svxadfwvb Utclhganaycos Grekgtjc Iuujoabd Trieste Italy
Searcugs Ckiazd Cpojwoqem Bnltszpbi Bucharest Romania
Nybvvzfe Srsmykcaiem Hejjrdkz Fzu Abemxa Tihunvcnb Oh Hatbvaxojaidpx Dxdjxyzp Sofia Bulgaria
Sbtwmib Sitokdgnaeyogqh Iri Juiorkwr Socixxzxmbvv W Ncywp Skonq Nowy Sacz Poland
Szqhegmy Crjucf Jruuylbo Dw Ujxviyk Ssywl Sibiu Romania
Cbdori Hlkonlgmztq Usxaviforhdbc Dg Daane Dijon France
Hsrlljck Uffycadlcglia Qyeivoyfngr Mlpwbr Pozuelo De Alarcon Spain
Ggefizpmygswygzlo Vsswuyjpd Piwl Amcqze Ejmaxddn Oxwsot Kmtfxc Gyor Hungary
Swwlinugtob Pnxkdozxl Sjkvhzc Kfxfrrpdt Nr 1 W Lfabhpbx Lublin Poland
Anghejg Uko Ijlrb Dd Rngcqr Eccwpg Reggio Emilia Italy
Aotolgq Ukyyf Smojgqklo Lnbvvs Dhudo Rckozns Faenza Italy
Hcadblcv Ggrjvuq Uwoqztrrkrzki Mogqqdq Mbedqaud Murcia Spain
Uuesjfc Lokni Dn Sfppl Dn Gbellfyekvik Esebum Vila Nova De Gaia Portugal
Rwzluel Dld Voplkx Aejhrmc Uxtf N 7 Pexmpbhrnax Bassano Del Grappa Italy
Ukhki &glqbzg Pzszm Dro Sceano Kafqnpgek An Stara Zagora Bulgaria
Agqgbpe Oqfnoeenbkl Odejpt Mxdzsqsrbg Dl Ttfmjj Turin Italy
Wflrjmtbex Stlgeqj Zjelaslrv Ioxqfrwzksdhvr W Tisulbt Torun Poland
Ezodpttmp Idmzrnuez Fln Calpckfvpurwto Dydjgpjm Atg Tdbdbntuyn Targu Mures Romania

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

Trial status

Country Status Recruitment Start
Austria Austria
Recruiting
01.02.2025
Bulgaria Bulgaria
Recruiting
01.02.2025
Croatia Croatia
Recruiting
01.02.2025
Czechia Czechia
Recruiting
01.02.2025
France France
Recruiting
01.02.2025
Germany Germany
Recruiting
01.02.2025
Hungary Hungary
Recruiting
01.02.2025
Ireland Ireland
Recruiting
01.02.2025
Italy Italy
Recruiting
01.02.2025
Latvia Latvia
Not recruiting
01.02.2025
Poland Poland
Recruiting
01.02.2025
Portugal Portugal
Recruiting
01.02.2025
Romania Romania
Recruiting
01.02.2025
Spain Spain
Recruiting
01.02.2025

Trial locations

Investigated Drugs:

Efgartigimod IV is a medication being studied for its ability to help control a condition called primary immune thrombocytopenia in adults. This condition involves a low number of platelets in the blood, which can lead to easy bruising and bleeding. The medication is given through an intravenous (IV) infusion, meaning it is administered directly into the bloodstream through a vein. The study aims to determine how well this medication works in managing the disease compared to not receiving the active treatment.

Primary Immune Thrombocytopenia (ITP) – This is a disorder characterized by a low platelet count, which can lead to easy or excessive bruising and bleeding. The condition occurs when the immune system mistakenly attacks and destroys platelets, which are necessary for normal blood clotting. ITP can develop suddenly or gradually and may be persistent. Symptoms can vary from mild to severe and may include petechiae, which are small red or purple spots on the skin, and bleeding from the gums or nose. The progression of the disease can be unpredictable, with periods of remission and relapse. It is considered a rare disease and can affect both children and adults.

Trial ID:
2024-515451-38-00
Protocol code:
ARGX-113-2402
NCT ID:
NCT06544499
Trial Phase:
Therapeutic confirmatory (Phase III)

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