Clinical trial • Phase III • Immunology|Rare Disease

Efgartigimod alfa for Primary immune thrombocytopenia (ITP)

Phase III trial of Efgartigimod alfa for Primary immune thrombocytopenia (ITP).

Overview

Trial Therapeutic Area
Immunology|Rare Disease
Trial Disease
Primary immune thrombocytopenia (ITP)
Trial Stage
Phase III
Drug Modality
Monoclonal antibody
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
27-09-2024
First CTIS Authorization Date
24-01-2025

Trial design

Randomised, open-label, placebo iv (matching placebo to argx-113) — no active dose for placebo specified; investigational arm: efgartigimod iv (argx-113) — product information indicates dosing unit mg/kg, maximum daily dose 10 mg/kg and max total dose amount reported as 1280 (units: mg/kg milligram(s)/kilogram and total), route iv; exact schedule per protocol documents.-controlled Phase III trial in Latvia, Austria, Bulgaria and others.

Randomised
Yes
Open Label
Yes
Comparator
Placebo IV (matching placebo to ARGX-113) — no active dose for placebo specified; investigational arm: Efgartigimod IV (ARGX-113) — product information indicates dosing unit mg/kg, maximum daily dose 10 mg/kg and max total dose amount reported as 1280 (units: mg/kg milligram(s)/kilogram and total), route IV; exact schedule per protocol documents.

Eligibility

Recruits 52 adults.

Inclusion criteria

  • {"criterion_text":"- Is at least 18 years of age and the local legal age of consent for clinical studies when signing the informed consent form (ICF). \n- Has documented baseline mean platelet count of <30 x 10^9/L before randomization\n- Has a documented duration of primary immune thrombocytopenia (ITP) of more than 12 months on the date of informed consent form (ICF) signature\n- Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, intravenous immunoglobulin (IVIg), anti-D immunoglobulin (for participants who are nonsplenectomized and Rho(D)- positive), thrombopoietin receptor agonist (TPO-RAs), or rituximab\n- Has documented insufficient response to a prior ITP treatment with corticosteroids, IVIg, anti-D immunoglobulin (for participants who are nonsplenectomized and Rho(D)-positive), TPO-RAs, rituximab (the specific criteria can be found in the protocol).\n- Has documented prior response defined as 1 platelet count of ≥50 × 10^9/L to at least 1 of the following ITP treatments in the 3 years before the date of ICF signature: prednisone, dexamethasone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin (for participants who are nonsplenectomized and Rho(D)-positive)."}

Exclusion criteria

  • {"criterion_text":"- Other than the indication under study, known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of ITP, confound the results of the study or put the participant at undue risk.\n- Secondary ITP\n- Nonimmune thrombocytopenia\n- Autoimmune hemolytic anemia\n- ITP-associated critical or severe bleeding\n- (additional exclusion criteria may be present in protocol; the above are the principal exclusion items listed)"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Extent of disease control, defined as the number of cumulative weeks during the Double-Blinded Treatment Period with platelet counts of at least 50 × 10^9/L.","definition_or_measurement_approach":"Defined as the number of cumulative weeks during the Double-Blinded Treatment Period (DBTP) with platelet counts ≥ 50 × 10^9/L (measured per the study visit schedule)."}

Secondary endpoints

  • {"endpoint_text":"- Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 4 of the 6 study visits between study weeks 19 and 24 of the DBTP","definition_or_measurement_approach":"Proportion achieving platelet counts ≥50 × 10^9/L at least 4 of 6 visits between weeks 19–24 of DBTP."}
  • {"endpoint_text":"- Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 6 of the 8 study visits between study weeks 17 and 24 of the DBTP","definition_or_measurement_approach":"Proportion achieving platelet counts ≥50 × 10^9/L at least 6 of 8 visits between weeks 17–24 of DBTP."}
  • {"endpoint_text":"- Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 8 of the 12 study visits between weeks 13 and 24 of the DBTP","definition_or_measurement_approach":"Proportion achieving platelet counts ≥50 × 10^9/L at least 8 of 12 visits between weeks 13–24 of DBTP."}
  • {"endpoint_text":"- Proportion of participants achieving a platelet count of at least 50 × 10^9/L on at least 4 occasions at any time until study week 12 during the DBTP","definition_or_measurement_approach":"Proportion with platelet count ≥50 × 10^9/L on ≥4 occasions at any time up to week 12 of DBTP."}
  • {"endpoint_text":"- Time to response, defined as the time to achieve 2 consecutive platelet counts of at least 50 × 10^9/L at any time during the DBTP","definition_or_measurement_approach":"Time from baseline to first of two consecutive platelet counts ≥50 × 10^9/L during DBTP."}
  • {"endpoint_text":"- Proportion of participants with an International Working Group (IWG) response during the DBTP","definition_or_measurement_approach":"Proportion meeting IWG response criteria during DBTP (IWG definitions as per protocol)."}
  • {"endpoint_text":"- Proportion of participants with International Working Group (IWG) complete response during the DBTP","definition_or_measurement_approach":"Proportion meeting IWG complete response criteria during DBTP."}
  • {"endpoint_text":"- Proportion of participants with initial response, defined as a platelet count of at least 30 × 10^9/L and a 2-fold increase from baseline in platelet count at study week 5 of the DBTP","definition_or_measurement_approach":"Proportion with platelet count ≥30 × 10^9/L and ≥2× baseline at week 5 of DBTP."}
  • {"endpoint_text":"- Time to achieve a platelet count of at least 30 × 10^9/L and a 2-fold increase from baseline in platelet count during the DBTP","definition_or_measurement_approach":"Time from baseline to achievement of platelet count ≥30 × 10^9/L and ≥2× baseline during DBTP."}
  • {"endpoint_text":"- Number of cumulative weeks during the DBTP with platelet counts of at least 30 × 10^9/L and ≥20 × 10^9/L above baseline","definition_or_measurement_approach":"Number of cumulative weeks in DBTP with platelet counts ≥30 × 10^9/L and ≥20 × 10^9/L above baseline."}
  • {"endpoint_text":"- Number of cumulative weeks during the DBTP with platelet counts of at least 30 × 10^9/L and at least 20 × 10^9/L above baseline in participants with baseline platelet counts of <15 × 10^9/L","definition_or_measurement_approach":"As above, restricted to participants with baseline platelet <15 × 10^9/L."}
  • {"endpoint_text":"- Extent of disease control, defined as the percentage of weeks with platelet counts of at least 50 × 10^9/L","definition_or_measurement_approach":"Percentage of study weeks with platelet counts ≥50 × 10^9/L during the assessment period."}
  • {"endpoint_text":"- In participants receiving placebo IV in the DBTP, the extent of disease control, defined as the number of cumulative weeks with platelet counts of at least 50 × 10^9/L, between weeks 13 and 24 of the OLTP1","definition_or_measurement_approach":"Cumulative weeks with platelet counts ≥50 × 10^9/L between weeks 13–24 of OLTP1 for participants who received placebo in DBTP."}
  • {"endpoint_text":"- Overall platelet count response, defined as the proportion of participants achieving a platelet count of at least 50 × 10^9/L on at least 4 occasions during the first 52 weeks of treatment with efgartigimod IV.","definition_or_measurement_approach":"Proportion achieving platelet count ≥50 × 10^9/L on ≥4 occasions during first 52 weeks of efgartigimod IV treatment."}
  • {"endpoint_text":"- Mean change from baseline in platelet count at each study visit","definition_or_measurement_approach":"Mean (group) change from baseline in platelet count measured at each scheduled visit."}
  • {"endpoint_text":"- The percentage of weeks with platelet counts of at least 30 × 10^9/L and at least 20 × 10^9/L above baseline","definition_or_measurement_approach":"Percentage of weeks during assessment period where platelet counts meet the stated thresholds above baseline."}
  • {"endpoint_text":"- In participants with baseline platelet counts <15 × 10^9/L, the percentage of weeks with platelet counts of at least 30 × 10^9/L and at least 20 × 10^9/L above baseline","definition_or_measurement_approach":"As above, restricted to participants with baseline platelet <15 × 10^9/L."}
  • {"endpoint_text":"- Proportion of participants who achieve a sustained platelet count response, defined as achieving platelet counts of at least 50 × 10^9/L for at least 4 occasions during 6-week intervals.","definition_or_measurement_approach":"Proportion achieving sustained platelet response: ≥50 × 10^9/L on ≥4 occasions at 6-week intervals."}
  • {"endpoint_text":"- In participants receiving placebo IV in the DBTP, the proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 8 of the 12 study visits between weeks 13 and 24 of the OLTP1","definition_or_measurement_approach":"Proportion (placebo recipients in DBTP) achieving platelet counts ≥50 × 10^9/L at least 8 of 12 visits between weeks 13–24 of OLTP1."}
  • {"endpoint_text":"- In participants receiving placebo IV in the DBTP, the proportion of participants who achieve a sustained platelet count response, defined as achieving platelet counts of at least 50 × 10^9/L for at least 6 of the 8 study visits between weeks 17 and 24 of the OLTP1","definition_or_measurement_approach":"Proportion (placebo recipients) achieving sustained response: ≥50 × 10^9/L in ≥6 of 8 visits between weeks 17–24 of OLTP1."}
  • {"endpoint_text":"- In participants receiving placebo IV in the DBTP, the extent of disease control, defined as the number of cumulative weeks with platelet counts of at least 50 × 10^9/L during the first 24 weeks of treatment with efgartigimod IV","definition_or_measurement_approach":"Cumulative weeks with platelet counts ≥50 × 10^9/L during the first 24 weeks of efgartigimod IV treatment for participants who had previously received placebo."}
  • {"endpoint_text":"- Occurrence rate of rescue ITP therapy","definition_or_measurement_approach":"Incidence/rate of use of rescue ITP therapies as defined in protocol."}
  • {"endpoint_text":"- Proportion of participants with reduction in concurrent ITP therapy during the OLTP1","definition_or_measurement_approach":"Proportion showing reduction in background/concomitant ITP therapy during OLTP1."}
  • {"endpoint_text":"- Incidence and severity of bleeding, assessed by the ITP Bleeding Scale (IBLS)","definition_or_measurement_approach":"Bleeding incidence and severity measured by IBLS."}
  • {"endpoint_text":"- Incidence and severity of adverse events (AEs) (including AEs of clinical interest) and serious AEs.","definition_or_measurement_approach":"Incidence and severity of AEs and SAEs collected per study safety reporting procedures."}
  • {"endpoint_text":"- Vital signs, laboratory safety measurements, physical examination","definition_or_measurement_approach":"Standard safety assessments: vital signs, lab tests, physical exam."}
  • {"endpoint_text":"- Incidence and prevalence of antidrug antibodies (ADA) against efgartigimod in serum over time","definition_or_measurement_approach":"ADA incidence and prevalence measured in serum samples over time."}
  • {"endpoint_text":"- Incidence and prevalence of NAb against efgartigimod in serum over time","definition_or_measurement_approach":"Neutralizing antibody (NAb) incidence and prevalence measured in serum over time."}
  • {"endpoint_text":"- Efgartigimod Cmax and Ctrough over time","definition_or_measurement_approach":"Pharmacokinetic measurement of Cmax and Ctrough for efgartigimod across sampling times."}
  • {"endpoint_text":"- Percent change from baseline in total IgG levels in serum over time","definition_or_measurement_approach":"Percent change from baseline in total serum IgG measured across timepoints."}

Recruitment

Planned Sample Size
52
Recruitment Window Months
39
Consent Approach
Written informed consent to be obtained from each participant; participants must be at least 18 years of age (and meet local legal age of consent) at the time of signing the ICF. Subject information sheets and ICFs are provided (multiple L1_SIS-ICF documents) and are available in English and in local languages as submitted for each country (examples in the CTIS documents: English, Bulgarian, Russian, Latvian, Spanish, French, Polish, Portuguese, Hungarian), with pregnancy/partner information forms provided where applicable.

Methods

  • Site-based recruitment via HCP letters to healthcare professionals (K2_HCP Letter documents)
  • Patient-facing printed materials: Flyers, Posters, Brochures (K2_Flyer, K2_Poster, K2_Brochure documents)
  • Patient letters (K2_Patient Letter documents) sent/available via sites
  • Visit calendars provided to participants (K2_Visit_Calendar documents)
  • Clinic/site contact lists and centre-specific information (Centre-specific Contact List document)
  • Patient identification/emergency ID cards and patient travel/reimbursement support (Clincierge materials and Participant Card documents)

Geography

Total Number Of Sites
54
Total Number Of Participants
52

Latvia

Earliest CTIS Part Ii Submission Date
06-01-2025
Latest Decision Or Authorization Date
30-01-2025
Processing Time Days
24
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Pauls Stradins Clinical University Hospital
Department Name
Oncology Clinic
Principal Investigator Name
Kristīne Bernāte
Principal Investigator Email
kristine.bernate@stradini.lv
Contact Person Name
Kristīne Bernāte
Contact Person Email
kristine.bernate@stradini.lv

Austria

Earliest CTIS Part Ii Submission Date
13-12-2024
Latest Decision Or Authorization Date
29-01-2025
Processing Time Days
47
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
Klinikum Wels-Grieskirchen GmbH
Department Name
Department of Internal Medicine IV (Oncology, Hematology and Nephrology)
Principal Investigator Name
Sonja Heibl
Principal Investigator Email
sonja.heibl@klinikum-wegr-at
Contact Person Name
Sonja Heibl
Contact Person Email
sonja.heibl@klinikum-wegr-at
Site Name
Ordensklinikum Linz GmbH
Department Name
(Hämato)onkologie und Hämostaseologie
Principal Investigator Name
Robert Milanov
Principal Investigator Email
robert.milanov@ordensklinikum.at
Contact Person Name
Robert Milanov
Site Name
Kepler Universitaetsklinikum GmbH
Department Name
Hämatologie und Onkologie
Principal Investigator Name
Clemens Schmitt
Principal Investigator Email
clemens.schmitt@keplerklinkum.at
Contact Person Name
Clemens Schmitt

Bulgaria

Earliest CTIS Part Ii Submission Date
17-01-2025
Latest Decision Or Authorization Date
03-02-2025
Processing Time Days
17
Number Of Sites
5
Number Of Participants
5

Sites

Site Name
University Multiprofile Hospital For Active Treatment St. Ivan Rilski EAD
Department Name
Department of Clinical Hematology
Principal Investigator Name
Atans Radinoff
Principal Investigator Email
aradinoff@hotmail.com
Contact Person Name
Atans Radinoff
Contact Person Email
aradinoff@hotmail.com
Site Name
Alexandrovska University Hospital
Department Name
Clinic of Clinical Hematology
Principal Investigator Name
Evgueniy Hadjiev
Principal Investigator Email
e_hadji58@yahoo.com
Contact Person Name
Evgueniy Hadjiev
Contact Person Email
e_hadji58@yahoo.com
Site Name
Acibadem City Clinic Diagnostic And Consultation Center Tokuda EAD
Department Name
Clinic of Hematology
Principal Investigator Name
Ismail Amine
Principal Investigator Email
dramine66@gmail.com
Contact Person Name
Ismail Amine
Contact Person Email
dramine66@gmail.com
Site Name
University Multiprofile Hospital For Active Treatment Sofiamed OOD
Department Name
Department of Clinical Hematology
Principal Investigator Name
Tzvetan Alaikov
Principal Investigator Email
dr.alaikov@gmail.com
Contact Person Name
Tzvetan Alaikov
Contact Person Email
dr.alaikov@gmail.com
Site Name
Umbal - Prof. D-R Stoyan Kirkovich AD
Department Name
Department of Clinical Hematology
Principal Investigator Name
Mariya Todorova
Principal Investigator Email
dr.maria.dtodorova@gmail.com
Contact Person Name
Mariya Todorova
Contact Person Email
dr.maria.dtodorova@gmail.com

Croatia

Earliest CTIS Part Ii Submission Date
13-01-2025
Latest Decision Or Authorization Date
03-02-2025
Processing Time Days
21
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
University Hospital Centre Zagreb
Department Name
Unit for Hemostasis and Thrombosis and Benign Diseases of the Hematopoietic System
Principal Investigator Name
Dražen Pulanić
Principal Investigator Email
dpulanic@yahoo.com
Contact Person Name
Dražen Pulanić
Contact Person Email
dpulanic@yahoo.com

Czechia

Earliest CTIS Part Ii Submission Date
03-01-2025
Latest Decision Or Authorization Date
28-03-2025
Processing Time Days
84
Number Of Sites
3
Number Of Participants
1

Sites

Site Name
Fakultni Nemocnice Ostrava
Department Name
Klinika hematoonkologie
Principal Investigator Name
Ivo Demel
Principal Investigator Email
ivo.demel@fno.cz
Contact Person Name
Ivo Demel
Contact Person Email
ivo.demel@fno.cz
Site Name
Fakultni Nemocnice Kralovske Vinohrady
Department Name
Interni hematologicka klinika
Principal Investigator Name
Olga Cerna
Principal Investigator Email
olga.cerna@fnkv.cz
Contact Person Name
Olga Cerna
Contact Person Email
olga.cerna@fnkv.cz
Site Name
Fakultni Nemocnice Brno
Department Name
Interni hematologicka a onkologicka klinika
Principal Investigator Name
Jiri Mayer
Principal Investigator Email
mayer.jiri@fnbrno.cz
Contact Person Name
Jiri Mayer
Contact Person Email
mayer.jiri@fnbrno.cz

France

Earliest CTIS Part Ii Submission Date
25-04-2025
Latest Decision Or Authorization Date
07-05-2025
Processing Time Days
12
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
Assistance Publique Hopitaux De Paris
Department Name
Department of Internal Medicine and Clinical Immunology
Principal Investigator Name
Marc Michel
Principal Investigator Email
Marc.michel2@aphp.fr
Contact Person Name
Marc Michel
Contact Person Email
Marc.michel2@aphp.fr
Site Name
Centre Hospitalier Universitaire Amiens Picardie
Department Name
Service Hematologie clique et therapie cellulaire
Principal Investigator Name
Clément Gourguechon
Principal Investigator Email
gourguechon.clement@chu-amiens.fr
Contact Person Name
Clément Gourguechon
Site Name
Centre Hospitalier Universitaire De Dijon
Department Name
Department of Internal Medicine and Clinical Immunology
Principal Investigator Name
Bernard Bonnotte
Principal Investigator Email
bernard.bonnotte@chu-dijon.fr
Contact Person Name
Bernard Bonnotte
Contact Person Email
bernard.bonnotte@chu-dijon.fr

Germany

Earliest CTIS Part Ii Submission Date
07-01-2025
Latest Decision Or Authorization Date
31-01-2025
Processing Time Days
24
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Technische Universitaet Dresden
Department Name
Medizinische Klinik und Poliklinik I
Principal Investigator Name
Karolin Trautmann-Grill
Principal Investigator Email
Karolin.Trautmann@ukdd.de
Contact Person Name
Karolin Trautmann-Grill
Contact Person Email
Karolin.Trautmann@ukdd.de
Site Name
Universitaetsklinikum Essen AöR
Department Name
Klinik für Hämatologie und Stamzelltransplantation
Principal Investigator Name
Alexander Roeth
Principal Investigator Email
studienzentrum-haemaSZT@uk-essen.de
Contact Person Name
Alexander Roeth

Hungary

Earliest CTIS Part Ii Submission Date
28-03-2025
Latest Decision Or Authorization Date
03-06-2025
Processing Time Days
67
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Semmelweis University
Department Name
Belgyógyászati és Hematológiai Klinika
Principal Investigator Name
Zsolt Nagy
Principal Investigator Email
nagy.zsolt@med.semmelweis-univ.hu
Contact Person Name
Zsolt Nagy
Site Name
Gyor-Moson-Sopron Varmegyei Petz Aladar Egyetemi Oktato Korhaz
Department Name
II. Belgyógyászat – Hematológia Osztály
Principal Investigator Name
Zsolt Lázár
Principal Investigator Email
lazarzsolt1982@gmail.com
Contact Person Name
Zsolt Lázár
Contact Person Email
lazarzsolt1982@gmail.com

Ireland

Earliest CTIS Part Ii Submission Date
09-12-2024
Latest Decision Or Authorization Date
04-02-2025
Processing Time Days
57
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Mater Misericordiae University Hospital
Department Name
Haematology
Principal Investigator Name
Anne Fortune
Principal Investigator Email
CTRU@mater.ie
Contact Person Name
Anne Fortune
Contact Person Email
CTRU@mater.ie
Site Name
St James's Hospital
Department Name
Haematology
Principal Investigator Name
Catherine Flynn
Principal Investigator Email
cancerclinicaltrials@stjames.ie
Contact Person Name
Catherine Flynn

Italy

Earliest CTIS Part Ii Submission Date
02-01-2025
Latest Decision Or Authorization Date
03-04-2025
Processing Time Days
91
Number Of Sites
13
Number Of Participants
13

Sites

Site Name
Azienda Unita Sanitaria Locale Della Romagna
Department Name
Ospedale S. Maria delle Croci ,UOC Ematologia, Viale Randi 5, 48121, Ravenna
Principal Investigator Name
Claudia Cellini
Principal Investigator Email
claudia.cellini@auslromagna.it
Contact Person Name
Claudia Cellini
Contact Person Email
claudia.cellini@auslromagna.it
Site Name
Regione Del Veneto Azienda ULSS N 7 Pedemontana
Department Name
Ospedale San Bassiano UOC Oncoematologia
Principal Investigator Name
Eros Di Bona
Principal Investigator Email
eros.dibona@aulss7.veneto.it
Contact Person Name
Eros Di Bona
Contact Person Email
eros.dibona@aulss7.veneto.it
Site Name
Azienda USL IRCCS Di Reggio Emilia
Department Name
Arcispedale Santa Maria Nuova, Ematologia,Viale Risorgimento 80 Reggio Emilia,42123,Italy
Principal Investigator Name
Barbara Gamberi
Principal Investigator Email
Barbara.Gamberi@ausl.re.it
Contact Person Name
Barbara Gamberi
Contact Person Email
Barbara.Gamberi@ausl.re.it
Site Name
Azienda Socio Sanitaria Territoriale Dei Sette Laghi
Department Name
Ospedale di Circolo e Fondazione Macchi -SC Ematologia
Principal Investigator Name
Domenica Caramazza
Principal Investigator Email
domenica.caramazza@asst-settelaghi.it
Contact Person Name
Domenica Caramazza
Site Name
ASST Grande Ospedale Metropolitano Niguarda
Department Name
Dipartimento Ematologia, Oncologia e Medicina Molecolare
Principal Investigator Name
Monica Carpenedo
Principal Investigator Email
Monica.carpenedo@ospedaleniguarda.it
Contact Person Name
Monica Carpenedo
Site Name
Careggi University Hospital
Department Name
Azienda Ospedaliero Universitaria Careggi, SOD Ematologia, Largo Brambilla 3, 5134 Firenze,Italy
Principal Investigator Name
Valentina Carrai
Principal Investigator Email
carraiv@aou-careggi.toscana.it
Contact Person Name
Valentina Carrai
Contact Person Email
carraiv@aou-careggi.toscana.it
Site Name
Azienda Ospedaliero-Universitaria Maggiore Della Carita
Department Name
SCDU Ematologia
Principal Investigator Name
Andrea Patriarca
Principal Investigator Email
andrea.patriarca@uniupo.it
Contact Person Name
Andrea Patriarca
Contact Person Email
andrea.patriarca@uniupo.it
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
SC Ematologia, Via Francesco Sforza 35, 20122 Milano , Italy
Principal Investigator Name
Bruno Fattizzo
Principal Investigator Email
bruno.fattizzo@policlinico.mi.it
Contact Person Name
Bruno Fattizzo
Site Name
Azienda Ospedaliera Ordine Mauriziano Di Torino
Department Name
SCDU Ematologia, Largo Filippo Turati 62, 10128,Turin, Italy
Principal Investigator Name
Piera Sivera
Principal Investigator Email
psivera@mauriziano.it
Contact Person Name
Piera Sivera
Contact Person Email
psivera@mauriziano.it
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
SC Medicina – Emostasi e Trombosi, Via Francesco Sforza 35, 20122 Milano , Italy
Principal Investigator Name
Andrea Artoni
Principal Investigator Email
Andrea.artoni@policlinico.mi.it
Contact Person Name
Andrea Artoni
Site Name
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Department Name
Ospedale Molinette, SC Ematologia U,Via Genova 3,10126,Torino,Italy
Principal Investigator Name
Alessandra Borchiellini
Principal Investigator Email
aborchiellini@cittadellasalute.to.it
Contact Person Name
Alessandra Borchiellini
Site Name
Azienda Sanitaria Universitaria Giuliano Isontina
Department Name
Ospedale Maggiore Department Oncology SC UCO Ematologia, Piazza dell’Ospitale 1,34125 Trieste,Italy
Principal Investigator Name
Francesco Zaja
Principal Investigator Email
francesco.zaja@asugi.sanita.fvg.it
Contact Person Name
Francesco Zaja
Site Name
Azienda Ospedaliera Universitaria Federico II Di Napoli
Department Name
UOC Ematologia
Principal Investigator Name
Fabrizio Pane
Principal Investigator Email
fabrizio.pane@unina.it
Contact Person Name
Fabrizio Pane
Contact Person Email
fabrizio.pane@unina.it

Poland

Earliest CTIS Part Ii Submission Date
06-01-2025
Latest Decision Or Authorization Date
29-01-2025
Processing Time Days
23
Number Of Sites
6
Number Of Participants
6

Sites

Site Name
Wojewodzkie Wielospecjalistyczne Centrum Onkologii I Traumatologii Im M.Kopernika W Lodzi
Department Name
Oddział Hematologii Ogólnej i Chorób Wewnętrznych
Principal Investigator Name
Jacek Treliński
Principal Investigator Email
Sekretariat.hemat@kopernik.lodz.pl
Contact Person Name
Jacek Treliński
Site Name
Pratia Hematologia Sp. z o.o.
Department Name
Pratia Onkologia Katowice
Principal Investigator Name
Sebastian Grosicki
Principal Investigator Email
kontakt.onkologia.katowice@pratia.com
Contact Person Name
Sebastian Grosicki
Site Name
Szpital Uniwersytecki Nr 2 Im Dr Jana Biziela W Bydgoszczy
Department Name
Klinika Hematologii
Principal Investigator Name
Marta Sobas
Principal Investigator Email
marta.sobas@cm.umk.pl
Contact Person Name
Marta Sobas
Contact Person Email
marta.sobas@cm.umk.pl
Site Name
Szpital Specjalistyczny Im. Jedrzeja Sniadeckiego W Nowym Saczu SPZOZ
Department Name
Oddział Hematologiczny
Principal Investigator Name
Szymon Fornagiel
Principal Investigator Email
szf@wp.pl
Contact Person Name
Szymon Fornagiel
Contact Person Email
szf@wp.pl
Site Name
Uniwersytecki Szpital Kliniczny Nr 4 W Lublinie
Department Name
Szpital, Oddział Wieloprofilowy Zachowawczy
Principal Investigator Name
Marta Morawska
Principal Investigator Email
mmorawska79@gmail.com
Contact Person Name
Marta Morawska
Contact Person Email
mmorawska79@gmail.com
Site Name
Niepubliczny Zaklad Opieki Zdrowotnej Neuromed M. I M. Nastaj. sp.p.
Department Name
Not applicable/Nie dotyczy
Principal Investigator Name
Tomasz Gromek
Principal Investigator Email
gromek.neuromed@gmail.com
Contact Person Name
Tomasz Gromek
Contact Person Email
gromek.neuromed@gmail.com

Portugal

Earliest CTIS Part Ii Submission Date
21-02-2025
Latest Decision Or Authorization Date
08-04-2025
Processing Time Days
46
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
CCAB Centro Clinico Academico Braga Associacao
Department Name
Internal Medicine
Principal Investigator Name
Carlos Capela
Principal Investigator Email
Carloscapela@med.uminho.pt
Contact Person Name
Carlos Capela
Contact Person Email
Carloscapela@med.uminho.pt
Site Name
Unidade Local De Saude De Gaia/Espinho E.P.E.
Department Name
Hematology
Principal Investigator Name
Teresa Melo
Principal Investigator Email
teresa.lencastre@ulsge.min-saude.pt
Contact Person Name
Teresa Melo

Romania

Earliest CTIS Part Ii Submission Date
10-01-2025
Latest Decision Or Authorization Date
03-02-2025
Processing Time Days
24
Number Of Sites
4
Number Of Participants
4

Sites

Site Name
Spitalul Clinic Judetean De Urgenta Sibiu
Department Name
Hematology
Principal Investigator Name
Alina Catana
Principal Investigator Email
secretariat@scjus.ro
Contact Person Name
Alina Catana
Contact Person Email
secretariat@scjus.ro
Site Name
Spitalul Clinic Coltea
Department Name
Hematology
Principal Investigator Name
Gabriela Borsaru
Principal Investigator Email
secretariat@coltea.ro
Contact Person Name
Gabriela Borsaru
Contact Person Email
secretariat@coltea.ro
Site Name
Spitalul Clinic Colentina Bucuresti
Department Name
Hematology
Principal Investigator Name
Daniela Georgescu
Principal Investigator Email
secretariat@spitalulcolentina.ro
Contact Person Name
Daniela Georgescu
Site Name
Spitalul Clinic Judetean De Urgenta Targu Mures
Department Name
Hematology
Principal Investigator Name
Ioan Macarie
Principal Investigator Email
secretariat@spitalmures.ro
Contact Person Name
Ioan Macarie
Contact Person Email
secretariat@spitalmures.ro

Spain

Earliest CTIS Part Ii Submission Date
29-11-2024
Latest Decision Or Authorization Date
24-01-2025
Processing Time Days
56
Number Of Sites
7
Number Of Participants
7

Sites

Site Name
Complexo Hospitalario Universitario A Coruna
Department Name
Hematology
Principal Investigator Name
Michael Calviño Suárez
Principal Investigator Email
Michael.Calvino.Suarez@sergas.es
Contact Person Name
Michael Calviño Suárez
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Hematology
Principal Investigator Name
Javier Lopez Jimenez
Principal Investigator Email
jljimenez@salud.madrid.org
Contact Person Name
Javier Lopez Jimenez
Contact Person Email
jljimenez@salud.madrid.org
Site Name
Hospital General Universitario Morales Meseguer
Department Name
Hematology
Principal Investigator Name
Maria Luisa Lozano Almela
Principal Investigator Email
mllozano@um.es
Contact Person Name
Maria Luisa Lozano Almela
Contact Person Email
mllozano@um.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Hematology
Principal Investigator Name
David Valcarcel Ferreiras
Principal Investigator Email
dvalcarcel@vhio.net
Contact Person Name
David Valcarcel Ferreiras
Contact Person Email
dvalcarcel@vhio.net
Site Name
Hospital Universitario Quironsalud Madrid
Department Name
Hematology
Principal Investigator Name
Maria Aranzazu Alonso Alonso
Principal Investigator Email
aranzazu.alonso@quironsalud.es
Contact Person Name
Maria Aranzazu Alonso Alonso
Contact Person Email
aranzazu.alonso@quironsalud.es
Site Name
Institut Catala D'oncologia
Department Name
Hematology
Principal Investigator Name
Silvana Novelli Canales
Principal Investigator Email
snovelli@iconcologia.net
Contact Person Name
Silvana Novelli Canales
Contact Person Email
snovelli@iconcologia.net
Site Name
Hospital Clinico Universitario De Valencia
Department Name
Hematology
Principal Investigator Name
Maria Luisa Calabuig Muñoz
Principal Investigator Email
marisacalabuig@yahoo.es
Contact Person Name
Maria Luisa Calabuig Muñoz
Contact Person Email
marisacalabuig@yahoo.es

Sponsor

Primary sponsor

Full Name
Argenx
Organisation Type
Pharmaceutical company
Country Of Registered Address
Belgium

Contract research organisations

Name
Icon Clinical Research Limited
Responsibilities
Clinical Operations, Site Management, Start-up, Clinical Outcome, Technology Management, Translations, Patient Materials, Investigator recruitment, Project management, Safety reporting
Name
PPD International Holdings LLC
Responsibilities
Central Lab analyses including Safety analysis
Name
IQVIA Limited
Name
Suvoda LLC

Third parties

  • {"country":"Germany","full_name":"Azenta Germany GmbH","duties_or_roles":"Lab facilities for long-term storage of samples.","organisation_type":"Pharmaceutical company"}
  • {"country":"France","full_name":"Mapi Research Trust","duties_or_roles":"Clinical Outcome Assessment Strategies/ Licensing;","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"code 3 (responsibility code present; specific duty text not provided)","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"The Doctors Laboratory Limited","duties_or_roles":"code 4 (responsibility code present; specific duty text not provided)","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Belgium","full_name":"SGS Belgium","duties_or_roles":"code 6 (responsibility code present; specific duty text not provided)","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Accellacare Limited","duties_or_roles":"In-home health care services, nursing","organisation_type":"Pharmaceutical company"}
  • {"country":"Netherlands","full_name":"Central Diagnostic Laboratory","duties_or_roles":"code 4 (responsibility code present; specific duty text not provided)","organisation_type":"Health care"}
  • {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"Central Lab analyses including Safety analysis","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Gray Consulting Inc.","duties_or_roles":"Patient Travel and Reimbursement","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Drug Development Solutions Limited","duties_or_roles":"code 4 (responsibility code present; specific duty text not provided)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Central eCOA/ePRO provision","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"code 8 (responsibility code present; specific duty text not provided)","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Medical Equipment Supplies And Management Limited","duties_or_roles":"Equipment Provision","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Clinical Operations, Site Management, Start-up, Clinical Outcome, Technology Management, Translations, Patient Materials, Investigator recruitment, Project management, Safety reporting","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Arup Laboratories Inc.","duties_or_roles":"code 4 (responsibility code present; specific duty text not provided)","organisation_type":"Laboratory/Research/Testing facility"}

Investigational products

Investigational Product Name
ARGX-113
Active Substance
Efgartigimod alfa
Modality
Monoclonal antibody
Routes Of Administration
Intravenous
Route
Intravenous
Authorisation Status
Investigational (no marketing authorisation specified in record)
Orphan Designation
Yes
Maximum Dose
10 mg/kg (max daily dose amount 10 mg/kg reported)
Investigational Product Name
Vyvgart 20 mg/mL concentrate for solution for infusion
Active Substance
Efgartigimod alfa
Modality
Monoclonal antibody
Routes Of Administration
Intravenous
Route
Intravenous
Authorisation Status
Marketing authorisation: EU/1/22/1674/001 (product has marketing authorisation details in record)
Orphan Designation
Yes
Maximum Dose
10 mg/kg (max daily dose amount 10 mg/Kg reported)
Investigational Product Name
Placebo for efgartigimod
Modality
Other

Related trials

Other published trials that may interest you.