Clinical trial • Phase III • Haematology

IANALUMAB for Primary immune thrombocytopenia

Phase III trial of IANALUMAB for Primary immune thrombocytopenia.

Overview

Trial Therapeutic Area
Haematology
Trial Disease
Primary immune thrombocytopenia
Trial Stage
Phase III
Drug Modality
Monoclonal antibody|Small molecule
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
10-07-2024
First CTIS Authorization Date
08-08-2024

Trial design

Randomised, placebo to ianalumab in addition to eltrombopag (control arm: placebo + eltrombopag). dosing/schedule for eltrombopag in the protocol record is not specified in the ctis json (eltrombopag product info indicates oral formulation; max daily dose listed as 75 mg in product data). Phase III trial in Hungary, Norway, Spain and others.

Randomised
Yes
Comparator
Placebo to ianalumab in addition to eltrombopag (control arm: placebo + eltrombopag). Dosing/schedule for eltrombopag in the protocol record is not specified in the CTIS JSON (eltrombopag product info indicates oral formulation; max daily dose listed as 75 mg in product data).
Target Sample Size
99
Trial Duration For Participant
168

Eligibility

Recruits 99 Vulnerable population flag selected in CTIS. Participation restricted to adults ("Male or female patients aged 18 years and older"); a signed informed consent must be obtained prior to participation. Child assent documentation is present among submitted documents (e.g. "L1_ICF - Child Assent_1_FR_French_NonRed"), and country-specific ICFs and follow-up pregnancy ICFs are provided, indicating assent/consent procedures and materials for different populations/languages where applicable..

Pregnancy Exclusion
Nursing (breast feeding) or pregnant women.
Vulnerable Population
Vulnerable population flag selected in CTIS. Participation restricted to adults ("Male or female patients aged 18 years and older"); a signed informed consent must be obtained prior to participation. Child assent documentation is present among submitted documents (e.g. "L1_ICF - Child Assent_1_FR_French_NonRed"), and country-specific ICFs and follow-up pregnancy ICFs are provided, indicating assent/consent procedures and materials for different populations/languages where applicable.

Inclusion criteria

  • {"criterion_text":"- Male or female patients aged 18 years and older on the day of signing the informed consent.\n- A signed informed consent must be obtained prior to participation in the study\n- A diagnosis of primary ITP, with insufficient response to, or relapse after a first-line corticosteroid therapy ± IVIG.\n- Patients with platelet count <30 G/L for whom eltrombopag is clinically indicated as per physician’s discretion and with no contraindication to receive eltrombopag."}

Exclusion criteria

  • {"criterion_text":"- ITP patients who received second-line ITP treatments (other than corticosteroid therapy ± IVIG) including splenectomy. However, patients exposed to thrombopoietin receptor agonist (TPO-RAs) for a limited time (max. one week) before screening are eligible.\n- Patients with key lab abnormalities and patients with Evans syndrome or any other cytopenia (patients with low grade anemia related to bleeding or iron deficiency are eligible).\n- Patients with history of clinically significant hematological disorders, or with marked altered hematologic parameters.\n- Patients with current or history of life-treating bleeding.\n- Patients that are Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B surface Antigen (HBsAg)/ Hepatitis B core antibody (HBcAb)- positive.\n- Patients with known active or uncontrolled infection requiring systemic treatment during screening period.\n- Patients with hepatic impairment with Child-Pugh score >5\n- Patients with concurrent coagulation disorder and/or receiving anti-platelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid (≤150 mg daily).\n- Nursing (breast feeding) or pregnant women."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Time to treatment failure (TTF) defined as the time from randomization until: -\tPlatelet counts <30 G/L or need for rescue treatment later than 8 weeks from randomization, -\tStart of new ITP treatment -\tIneligibility to taper or inability to discontinue eltrombopag or -\tDeath. TTF will be assessed in each treatment group and each of the 2 doses of ianalumab (ianalumab+ eltrombopag) will be compared to the control arm (placebo+ eltrombopag).","definition_or_measurement_approach":"TTF is defined as time from randomization to first occurrence of any of: platelet count <30 G/L or need for rescue treatment later than 8 weeks from randomization; start of a new ITP treatment; ineligibility to taper or inability to discontinue eltrombopag; or death. TTF will be assessed in each treatment arm and each ianalumab dose compared with control (placebo + eltrombopag)."}

Secondary endpoints

  • {"endpoint_text":"- At each time point: -\tComplete Response (CR) rate (proportion of participants with any platelet count ≤100 G/L without rescue or new ITP treatment).Response rate (R) (proportion of participants with any platelet count ≤50 G/L without rescue or new ITP treatment). Best response rate over all time points(proportion of participants with a best response of either R or CR). Time from randomization to R & CR. Duration of R & CR. Stable response at 6 months & at 1 year.\n- Probability to be treatment failure-free (as defined for the primary efficacy endpoint) at the end of the planned treatment period (end of Week 24)\n- Frequency of adverse events and other safety parameters. Number of severe infections and proportion of participants with severe infection.\n- Proportion of participants with bleeding events according to World Health Organization (WHO)Bleeding Scale.\n- Number and proportion of participants receiving rescue treatment.\n- Change from baseline on total score of the Patient- Reported Outcomes Measurement Information System (PROMIS)Short Form (SF) v1.0 Fatigue 13a. Change from baseline in ITP Patient Assessment Questionnaire (PAQ) domain scores of Symptoms, Fatigue, Bother, Activity.\n- B-cell levels: - Change from baseline in the frequency (% within the CD45) and absolute number of CD19+ B-cell counts - Time to first occurrence of B-cell recovery, defined as ≥80% of baseline or ≥50 cells/µL Immunoglobulins: -\tChange from baseline in immunoglobulin level\n- Ianalumab concentration in serum and PK parameters after the first and last dose in a subset of participants\n- Incidence and titer of anti-ianalumab antibodies in serum (anti-drug antibodies (ADA) assay) over time","definition_or_measurement_approach":"Secondary endpoints include response rates (CR and R) defined by platelet thresholds without rescue or new ITP treatment, time to and duration of responses, probability of being treatment-failure-free at Week 24, safety parameters including adverse event frequency and severe infections, bleeding events per WHO Bleeding Scale, rescue treatment counts, PRO changes (PROMIS Fatigue SF v1.0 and ITP-PAQ domains), B-cell and immunoglobulin measures (changes from baseline and time to B-cell recovery ≥80% baseline or ≥50 cells/µL), ianalumab PK concentrations and parameters after first and last dose, and immunogenicity (ADA incidence and titers over time). Specific measurement definitions are provided in the endpoint text (e.g., platelet thresholds, Week 24 timepoint, B-cell recovery thresholds)."}

Recruitment

Planned Sample Size
99
Recruitment Window Months
61
Consent Approach
A signed informed consent must be obtained prior to participation. Country-specific subject information and informed consent forms (L1_ICF) and procedure documents (L2_ICF Procedure) were submitted in multiple languages (examples: Hungarian, Norwegian, Spanish, German, Dutch, Italian, French, Romanian, English). Separate data protection consent documents and follow-up ICFs for pregnant participants are provided in some countries; child assent documentation is present in submitted materials (e.g. French child assent document).

Methods

  • Country-specific recruitment arrangements documents (K1_Recruitment Arrangements) were submitted for each participating country (documents listed per country: e.g., Hungary, Norway, Spain, Germany, Netherlands, Italy, Belgium, Czechia, Romania, France).
  • Country-specific advertisements and recruitment materials (K2_Advertisements) in multiple languages for participating countries (documents present for DE, ES, HU, NO, IT, BE, NL, FR, RO and others).

Geography

Total Number Of Sites
41
Total Number Of Participants
48

Hungary

Earliest CTIS Part Ii Submission Date
25-07-2024
Latest Decision Or Authorization Date
14-07-2025
Processing Time Days
354
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
University Of Debrecen
Department Name
#3551: Hematologia Osztaly
Contact Person Name
Arpad Illes
Contact Person Email
illesarpaddr@gmail.com
Site Name
Semmelweis University
Department Name
#3552: Belgyogyaszati es Hematologiai Klin
Contact Person Name
Zsolt Nagy

Norway

Earliest CTIS Part Ii Submission Date
25-07-2024
Latest Decision Or Authorization Date
11-07-2025
Processing Time Days
351
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Sykehuset Oestfold HF Kalnes
Department Name
#3150 - Kreft og hematologisk avdeling, trombose poliklinikk
Contact Person Name
Waleed Ghanima
Contact Person Email
Waleed.Ghanima@so-hf.no

Spain

Earliest CTIS Part Ii Submission Date
01-07-2024
Latest Decision Or Authorization Date
16-07-2025
Processing Time Days
380
Number Of Sites
5
Number Of Participants
5

Sites

Site Name
Hospital Del Mar
Department Name
#3850: Hematología
Contact Person Name
Blanca Sanchez Gonzalez
Contact Person Email
bsanchezgonzalez@psmar.cat
Site Name
Hospital Universitario De Salamanca
Department Name
#3855: Hematología
Contact Person Name
Jose Ramon Gonzalez Porras
Contact Person Email
jrgp@usal.es
Site Name
Hospital General Universitario Morales Meseguer
Department Name
#3854: Hematología
Contact Person Name
Maria Luisa Lozano Almela
Contact Person Email
mllozano@um.es
Site Name
Complexo Hospitalario Universitario De Santiago
Department Name
#3852: Hematología
Contact Person Name
Adrián Mosquera Orgueira
Site Name
Hospital Universitario 12 De Octubre
Department Name
#3851: Hematología
Contact Person Name
Maria Poza Santaella
Contact Person Email
mariapoza93@hotmail.com

Germany

Earliest CTIS Part Ii Submission Date
25-07-2024
Latest Decision Or Authorization Date
15-07-2025
Processing Time Days
355
Number Of Sites
13
Number Of Participants
13

Sites

Site Name
Gemeinschaftspraxis Haematologie Onkologie
Department Name
#3450: Private Practice
Contact Person Name
Thomas Illmer
Contact Person Email
illmer@onkologie-dresden.net
Site Name
Universitaetsklinikum Bonn AöR
Department Name
3457_CIO_B:Medizinische Klinik III
Contact Person Name
Thomas Zander
Contact Person Email
Thomas.zander@uk-koeln.de
Site Name
Universitaetsklinikum Aachen AöR
Department Name
3457_CiO_A:Uniklinik RWTH Aachen Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzellt
Contact Person Name
Thomas Zander
Contact Person Email
Thomas.zander@uk-koeln.de
Site Name
Universitaetsklinikum Jena KöR
Department Name
3453:Klinik für Innere Medizin II   Hämatologie und internistische Onkologie
Contact Person Name
Thomas Stauch
Contact Person Email
Thomas.Stauch@med.uni-jena.de
Site Name
Universitaetsklinikum Duesseldorf AöR
Department Name
3457_CIO_D:Klinik für Hämatologie, Onkologie und klinische Immunologie
Contact Person Name
Thomas Zander
Contact Person Email
Thomas.zander@uk-koeln.de
Site Name
University Hospital Cologne AöR
Department Name
#3457: Department I of Internal Medicine
Contact Person Name
Thomas Zander
Contact Person Email
Thomas.zander@uk-koeln.de
Site Name
Universitaetsklinikum Carl Gustav Carus Dresden an der Technischen Universitaet Dresden AöR
Department Name
#3456: Medizinische Klinik und Poliklinik 1
Contact Person Name
Karolin Trautmann-Grill
Site Name
Universitätsklinikum (additional listed sites)
Site Name
Universitätsklinikum (additional listed sites)
Site Name
Universitätsklinikum (additional listed sites)
Site Name
Universitätsklinikum (additional listed sites)
Site Name
Universitätsklinikum (additional listed sites)

Netherlands

Earliest CTIS Part Ii Submission Date
25-07-2024
Latest Decision Or Authorization Date
11-07-2025
Processing Time Days
351
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Universitair Medisch Centrum Utrecht
Department Name
#3750: Hematology
Contact Person Name
Roger Schutgens
Contact Person Email
r.schutgens@umcutrecht.nl
Site Name
Leids Universitair Medisch Centrum (LUMC)
Department Name
#3751: Hematology
Contact Person Name
Jaap Zwaginga
Contact Person Email
j.j.zwaginga@lumc.nl

Italy

Earliest CTIS Part Ii Submission Date
25-07-2024
Latest Decision Or Authorization Date
11-07-2025
Processing Time Days
351
Number Of Sites
6
Number Of Participants
6

Sites

Site Name
Azienda Unita Locale Socio Sanitaria N 8 Berica
Department Name
#3653 - Presidio Ospedaliero San Bortolo
Contact Person Name
GiusepApe Carli
Contact Person Email
beppe_carli@yahoo.it
Site Name
Azienda Unita Sanitaria Locale Di Bologna
Department Name
#3655 - U.O.C. Ematologia
Contact Person Name
Francesca Palandri
Contact Person Email
francesca.palandri@unibo.it
Site Name
Azienda Ospedaliera Policlinico Universitario Tor Vergata
Department Name
#3652 - U.O.S.D. Patologie Mieloproliferative Dipartimento Oncoematologia
Contact Person Name
Francesco Buccisano
Site Name
Azienda Sanitaria Universitaria Giuliano Isontina
Department Name
#3651 - U.C.O. Ematologia - Dipartimento di Oncologia
Contact Person Name
Francesco Zaja
Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
#3650 - U.O.C. Servizio e DH di Ematologia
Contact Person Name
Elena Rossi
Contact Person Email
elena.rossi@unicatt.it
Site Name
Additional listed Italian site

Belgium

Earliest CTIS Part Ii Submission Date
25-07-2024
Latest Decision Or Authorization Date
11-07-2025
Processing Time Days
351
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Algemeen Ziekenhuis Delta
Department Name
#3101: Hematology
Contact Person Name
Lien Deleu
Contact Person Email
lien.deleu@azdelta.be
Site Name
Centre Hospitalier Universitaire Dinant Godinne Sainte-Elisabeth-UCL-Namur
Department Name
#3102: Hematology
Contact Person Name
Julien Devreux

Austria

Earliest CTIS Part Ii Submission Date
25-07-2024
Latest Decision Or Authorization Date
21-07-2025
Processing Time Days
361
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Medical University Of Vienna
Department Name
3050: Department of Hematology
Contact Person Name
Johanna Gebhart
Site Name
Klinikum Wels-Grieskirchen GmbH
Department Name
3052: Department of Hematology
Contact Person Name
Sonja Heibl
Contact Person Email
sonja.heibl@klinikum-wegr.at

Czechia

Earliest CTIS Part Ii Submission Date
25-07-2024
Latest Decision Or Authorization Date
14-07-2025
Processing Time Days
354
Number Of Sites
3
Number Of Participants
4

Sites

Site Name
Fakultni Nemocnice Brno
Department Name
#3250: Interni hematologicka a onkologicka klinika
Contact Person Name
Jiri Mayer
Contact Person Email
mayer.jiri@fnbrno.cz
Site Name
Fakultni Nemocnice Kralovske Vinohrady
Department Name
#3251: Hematologicka klinika
Contact Person Name
Olga Cerna
Contact Person Email
olga.cerna@fnkv.cz
Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
#3252: Hematologie
Contact Person Name
Marek Trneny
Contact Person Email
trneny@cesnet.cz

Romania

Earliest CTIS Part Ii Submission Date
14-08-2024
Latest Decision Or Authorization Date
16-07-2025
Processing Time Days
336
Number Of Sites
2
Number Of Participants
5

Sites

Site Name
Spitalul Clinic Municipal De Urgenta Timisoara
Department Name
#7055 - Hematology
Contact Person Name
Ioana Ionita
Contact Person Email
mdioanaionita@yahoo.com
Site Name
Spitalul Clinic Municipal Filantropia Craiova
Department Name
#7051 - Hematology
Contact Person Name
Luminita Ocroteala
Contact Person Email
diaconu_luminita@yahoo.com

France

Earliest CTIS Part Ii Submission Date
25-07-2024
Latest Decision Or Authorization Date
24-10-2025
Processing Time Days
456
Number Of Sites
3
Number Of Participants
1

Sites

Site Name
Clinique Victor Hugo (Centre De Cancerologie De La Sarthe)
Department Name
#3350: Hématologie
Contact Person Name
Kamel Laribi
Contact Person Email
klaribi@ch-lemans.fr
Site Name
Centre Hospitalier Blois Simone Veil
Department Name
#3354: Médecine interne
Contact Person Name
Bertrand Lioger
Contact Person Email
liogerb@ch-blois.fr
Site Name
CHRU De Nancy
Department Name
#3352: Médecine interne
Contact Person Name
Thomas Moulinet
Contact Person Email
t.moulinet@chru-nancy.fr

Sponsor

Primary sponsor

Full Name
Novartis Pharma AG
Organisation Type
Pharmaceutical company
Country Of Registered Address
Switzerland

Contract research organisations

Name
Syneos Health Inc.
Responsibilities
[{"code":"1","id":905954}]
Name
Icon Clinical Research Limited
Responsibilities
[{"code":"1","id":905959},{"code":"2","id":905960},{"code":"7","id":905961}]
Name
IQVIA Limited / Iqvia Laboratories Limited / Iqvia Rds Inc.
Responsibilities
[{"code":"1","id":905944},{"code":"4","id":905967},{"code":"15","id":905962}]
Name
Parexel International (IRL) Limited
Responsibilities
[{"code":"12","id":905951}]
Name
Labcorp (various entities)
Responsibilities
[{"code":"4","id":905947},{"code":"4","id":905956}]

Third parties

  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"[{\"code\":\"1\",\"id\":905954}]","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"[{\"code\":\"1\",\"id\":905959},{\"code\":\"2\",\"id\":905960},{\"code\":\"7\",\"id\":905961}]","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Iqvia Laboratories Limited","duties_or_roles":"[{\"code\":\"4\",\"id\":905967}]","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Icon Laboratory Services Inc.","duties_or_roles":"[{\"code\":\"4\",\"id\":905957}]","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United Kingdom","full_name":"Labcorp Early Development Laboratories Limited","duties_or_roles":"[{\"code\":\"4\",\"id\":905956}]","organisation_type":"Pharmaceutical company"}
  • {"country":"China","full_name":"Labcorp Pharmaceutical Research And Development (Shanghai) Co. Ltd.","duties_or_roles":"[{\"code\":\"4\",\"id\":905947}]","organisation_type":"Pharmaceutical company"}
  • {"country":"India","full_name":"Veeda Clinical Research Limited","duties_or_roles":"[{\"code\":\"4\",\"id\":905955}]","organisation_type":"Pharmaceutical company"}
  • {"country":"Netherlands","full_name":"ICON Bioanalytical Laboratory","duties_or_roles":"[{\"code\":\"13\",\"id\":905964},{\"code\":\"4\",\"id\":905965}]","organisation_type":"Industry"}
  • {"country":"United Kingdom","full_name":"Illingworth Research Group Limited","duties_or_roles":"[{\"code\":\"15\",\"id\":905952,\"value\":\"Off site research nursing implementation and management\"}]","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"Austria","full_name":"Mag. Andreas Raffeiner GmbH","duties_or_roles":"[{\"code\":\"8\",\"id\":905943}]","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"[{\"code\":\"15\",\"id\":905945,\"value\":\"Licensing, translations and PRO formatting\"}]","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Reify Health Inc.","duties_or_roles":"[{\"code\":\"13\",\"id\":905942}]","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"[{\"code\":\"12\",\"id\":905951}]","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Iqvia Rds Inc.","duties_or_roles":"[{\"code\":\"15\",\"id\":905962,\"value\":\"Technical management of ePROs\"},{\"code\":\"3\",\"id\":905963}]","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"[{\"code\":\"1\",\"id\":905944}]","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Laboratory Corporation Of America","duties_or_roles":"[{\"code\":\"4\",\"id\":905953}]","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Czechia","full_name":"Movianto Ceska republika s.r.o.","duties_or_roles":"[{\"code\":\"15\",\"id\":905946,\"value\":\"IMP storage, destruction of unused and returned IMP\"}]","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Jumo Health USA Inc.","duties_or_roles":"[{\"code\":\"15\",\"id\":905950,\"value\":\"Preparation and translation of patient engagement materials\"}]","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"Germany","full_name":"DATAMAP-Gesellschaft fuer Datenmanagement Datenanalyse und Datenpraesentation mbH","duties_or_roles":"[{\"code\":\"10\",\"id\":905948},{\"code\":\"15\",\"id\":905949,\"value\":\"Independent statistician, creation of (un)blinded outputs for Data Monitoring Committee Meetings\"}]","organisation_type":"Non-Pharmaceutical company"}

Investigational products

Investigational Product Name
VAY736
Active Substance
IANALUMAB
Modality
Monoclonal antibody
Routes Of Administration
INTRAVENOUS
Route
Intravenous
Authorisation Status
Product listing present in CTIS (prodAuthStatus=1); orphan designation EU/3/25/3036 (ITP)
Orphan Designation
Yes
Starting Dose
3 mg/kg
Dose Levels
3 mg/kg; 9 mg/kg
Maximum Dose
9 mg/kg
Investigational Product Name
ELTROMBOPAG
Active Substance
ELTROMBOPAG
Modality
Small molecule
Routes Of Administration
ORAL
Route
Oral
Authorisation Status
Approved/marketed in multiple Member States (product is approved in the MSCs).
Maximum Dose
75 mg (max daily dose listed for product in CTIS data)
Investigational Product Name
Placebo to ianalumab 150 mg/1 mL concentrate for solution for infusion
Modality
Other
Combination Treatment
Yes

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