Clinical trial • Phase III • Musculoskeletal|Immunology

ANIFROLUMAB for Polymyositis | Dermatomyositis

Phase III trial of ANIFROLUMAB for Polymyositis | Dermatomyositis.

Overview

Trial Therapeutic Area
Musculoskeletal|Immunology
Trial Disease
Polymyositis | Dermatomyositis
Trial Stage
Phase III
Drug Modality
Monoclonal antibody
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
22-04-2024
First CTIS Authorization Date
13-08-2024

Trial design

Randomised, open-label, anifrolumab 120 mg sc qw + standard therapy (treatment 1 during double blinded) versus placebo sc qw + standard therapy (treatment 2 during double blinded).-controlled Phase III trial in France, Bulgaria, Spain and others.

Randomised
Yes
Open Label
Yes
Comparator
Anifrolumab 120 mg SC QW + standard therapy (Treatment 1 during double blinded) versus Placebo SC QW + standard therapy (Treatment 2 during double blinded).
Target Sample Size
120

Eligibility

Recruits 120 Vulnerable population selected. Informed consent is obtained via adult Subject Information and Informed Consent Forms (L1 SIS and ICF adults). Separate ICFs are provided for pregnant partners and optional genetic research. Participants are adults (18–75); assent is not applicable..

Pregnancy Exclusion
Lactating, breastfeeding, or pregnant females or females who intend to become pregnant or begin breastfeeding
Vulnerable Population
Vulnerable population selected. Informed consent is obtained via adult Subject Information and Informed Consent Forms (L1 SIS and ICF adults). Separate ICFs are provided for pregnant partners and optional genetic research. Participants are adults (18–75); assent is not applicable.

Inclusion criteria

  • {"criterion_text":"- 18 - 75 years old\n- Body weight 40 kg - ≤ 100 kg\n- Must have “probable” or “definite” diagnosis of PM or DM according to the 2017 ACR/EULAR classification criteria for adult myositis.\n- Moderate or severe disease activity per core set measurements.\n- Currently receiving oral prednisone or other polymyositis or dermatomyositis treatments at a stable dose.\n- No history of active tuberculosis or severe COVID-19.\n- Male and female participants must follow contraception guidelines."}

Exclusion criteria

  • {"criterion_text":"- Participants with documented inclusion body myositis (IBM), immune mediation necrotizing myositis (IMNM), juvenile myositis (if diagnosed within 10 years prior to signing the ICF), drug-induced myositis, cancer associated myositis, amyopathic DM, and non inflammatory myopathies (eg, muscular dystrophies).\n- Recent or concurrent enrollment in another clinical study with an investigational product.\n- Lactating, breastfeeding, or pregnant females or females who intend to become pregnant or begin breastfeeding\n- PM and DM patients at a high risk of malignancy.\n- Participants with rapidly progressive interstitial lung disease.\n- Participants with severe muscle damage or permanent weakness due to non-PM or non-DM conditions (i.e. stroke) as per the investigator's opinion.\n- Any history of severe case of herpes zoster infection\n- History of cancer (except adequately treated basal cell carcinoma or cervical cancer in-situ), immunodeficiency, HIV, HBV, active HCV .\n- Any clinical cytomegalovirus or Epstein-Barr virus infection that has not completely resolved within 12 weeks prior to signing the ICF.\n- Opportunistic infection requiring hospitalization or IV antimicrobial treatment within 3 years prior to randomization.\n- Recent non-opportunistic infection requiring hospitalization or anti-infective treatment."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Participants who have at least moderate improvement in disease activity (TIS ≥ 40) and has not met “confirmed deterioration” criteria at 2 consecutive visits","definition_or_measurement_approach":"Measured by Total Improvement Score (TIS) ≥ 40 and absence of 'confirmed deterioration' at two consecutive visits."}

Secondary endpoints

  • {"endpoint_text":"- MMT-8 (CSM) change from baseline at week 52","definition_or_measurement_approach":"Change from baseline in MMT-8 (manual muscle testing, CSM) at Week 52."}
  • {"endpoint_text":"- Participants who achieve oral corticosteroid dose ≤ 7.5 mg/day at week 52 (yes/no)","definition_or_measurement_approach":"Binary outcome (yes/no) whether participant reaches oral corticosteroid dose ≤ 7.5 mg/day at Week 52."}
  • {"endpoint_text":"- PM Participants who have at least moderate improvement in disease activity (TIS ≥ 40) at week 52 and has not met “confirmed deterioration” criteria at 2 consecutive visits up to and including week 52","definition_or_measurement_approach":"Measured by TIS ≥ 40 and no confirmed deterioration at two consecutive visits through Week 52 (PM subgroup)."}
  • {"endpoint_text":"- DM Participants who have at least moderate improvement in disease activity (TIS ≥ 40) at week 52 and has not met “confirmed deterioration” criteria at 2 consecutive visits up to and including week 52","definition_or_measurement_approach":"Measured by TIS ≥ 40 and no confirmed deterioration at two consecutive visits through Week 52 (DM subgroup)."}
  • {"endpoint_text":"- CDASI-activity change from baseline (DM participants only) at week 8","definition_or_measurement_approach":"Change from baseline in CDASI-activity (Cutaneous Dermatomyositis Disease Area and Severity Index - Activity) at Week 8 for DM participants."}
  • {"endpoint_text":"- \"MMT-8 (CSM) change from baseline at Week 52. Only PM participants will be included in the analysis.\"","definition_or_measurement_approach":"As stated: MMT-8 change from baseline at Week 52; analysis restricted to PM participants."}
  • {"endpoint_text":"- \"MMT-8 (CSM) change from baseline at Week 52 Only DM participants will be included in the analysis.\"","definition_or_measurement_approach":"As stated: MMT-8 change from baseline at Week 52; analysis restricted to DM participants."}
  • {"endpoint_text":"- \"Participants who achieve OCS dose ≤ 7.5 mg/day at Week 52 (yes/no). Only PM participants with baseline OCS dose > 7.5 mg/day at baseline will be included in the analysis.\"","definition_or_measurement_approach":"Binary OCS reduction endpoint at Week 52; analysis restricted to PM participants with baseline OCS > 7.5 mg/day."}
  • {"endpoint_text":"- \"Participants who achieve OCS dose ≤ 7.5 mg/day at Week 52 (yes/no). Only DM participants with baseline OCS dose > 7.5 mg/day at baseline will be included in the analysis.\"","definition_or_measurement_approach":"Binary OCS reduction endpoint at Week 52; analysis restricted to DM participants with baseline OCS > 7.5 mg/day."}

Recruitment

Planned Sample Size
120
Recruitment Window Months
45
Consent Approach
Informed consent is obtained from adult participants via Subject Information and Informed Consent Forms (L1 SIS and ICF adults). Separate consent materials exist for pregnant partners and optional genetic research (dedicated ICFs/addenda). Participant materials and ICFs are provided in country-/language-specific versions (examples in the documents include English, French, Spanish, Dutch, German, Italian, Hungarian, Polish, Bulgarian, Swedish). Assent is not applicable (participants aged 18–75).

Geography

Total Number Of Sites
69
Total Number Of Participants
84

France

Earliest CTIS Part Ii Submission Date
03-06-2024
Latest Decision Or Authorization Date
13-05-2026
Processing Time Days
709
Number Of Sites
11
Number Of Participants
10

Sites

Site Name
Hopital Saint Louis
Department Name
Service de médecine interne
Contact Person Name
Baptiste HERVIER
Contact Person Email
baptiste.hervier@aphp.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
Service de médecine interne et immunologie clinique
Contact Person Name
Grégory PUGNET
Contact Person Email
pugnet.g@chu-toulouse.fr
Site Name
Centre Hospitalier Universitaire D'Angers
Department Name
Service de Médecine interne et Immunologie clinique
Contact Person Name
Christian LAVIGNE
Contact Person Email
chlavigne@chu-angers.fr
Site Name
CHU De Rouen
Department Name
Service de médecine interne
Contact Person Name
Maëlle LE BESNERAIS
Site Name
Centre Hospitalier Universitaire Reims
Department Name
Service de rhumatologie
Contact Person Name
Lois BOLKO
Contact Person Email
lbolko@chu-reims.fr
Site Name
Hospices Civils De Lyon
Department Name
Service de médecine interne
Contact Person Name
Laure GALLAY
Contact Person Email
laure.gallay01@chu-lyon.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Service de médecine interne
Contact Person Name
Olivier BENVENISTE
Contact Person Email
olivier.benveniste@aphp.fr
Site Name
Centre Hospitalier Le Mans
Department Name
Service de rhumatologie-immunologie
Contact Person Name
Paul LEGENDRE
Contact Person Email
plegendre@ch-lemans.fr
Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
Service de médecine interne
Contact Person Name
Alain MEYER
Site Name
Centre Hospitalier Regional Et Universitaire De Brest
Department Name
Service de rhumatologie
Contact Person Name
Dewi GUELLEC
Contact Person Email
dewi.guellec@chu-brest.fr
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Service de médecine interne
Contact Person Name
Eric HACHULLA
Contact Person Email
Eric.HACHULLA@chru-lille.fr

Bulgaria

Earliest CTIS Part Ii Submission Date
12-07-2024
Latest Decision Or Authorization Date
23-01-2026
Processing Time Days
560
Number Of Sites
5
Number Of Participants
8

Sites

Site Name
Medical Center Medtech Services Ltd.
Contact Person Name
Krasimir Kraev
Contact Person Email
dr_k.kraev@abv.bg
Site Name
Medical Center Artmed Ltd.
Contact Person Name
Mariela Geneva-Popova
Contact Person Email
dr.mariela.geneva@gmail.com
Site Name
University Multiprofile Hospital For Active Treatment Kaspela EOOD
Department Name
Clinic of Rheumatology
Contact Person Name
Anastas Batalov
Contact Person Email
abatalov@hotmail.com
Site Name
Diagnostics And Consultation Center Convex Ltd.
Contact Person Name
Vladimira Boyadzhieva
Site Name
University Multiprofile Hospital For Active Treatment Pulmed Ltd.
Department Name
Rheumatology department
Contact Person Name
Lyubomir Sapundzhiev
Contact Person Email
sapoundjiev@abv.bg

Spain

Earliest CTIS Part Ii Submission Date
15-07-2024
Latest Decision Or Authorization Date
13-05-2026
Processing Time Days
667
Number Of Sites
10
Number Of Participants
10

Sites

Site Name
Parc Tauli Hospital Universitari
Department Name
Servicio de Reumatología
Contact Person Name
Carlos Galisteo Lencastre Da Veiga
Contact Person Email
galisteo.parctauli@gmail.com
Site Name
Hospital De La Santa Creu I Sant Pau
Department Name
Servicio de Reumatologia
Contact Person Name
Ivan Castellvi Barranco
Contact Person Email
icastellvi@santpau.cat
Site Name
Hospital Universitario Rio Hortega
Department Name
Servicio de Medicina Interna
Contact Person Name
Julia Barbado Ajo
Contact Person Email
juliabarbado@yahoo.es
Site Name
Hospital Universitario Reina Sofia
Department Name
Servicio de Reumatología
Contact Person Name
Rafaela Ortega Castro
Contact Person Email
orcam84@gmail.com
Site Name
Bellvitge University Hospital
Department Name
Servicio de Reumatología
Contact Person Name
Paola Vidal Montal
Contact Person Email
pvidal@bellvitgehospital.cat
Site Name
Hospital Del Mar
Department Name
Servicio de Neumología
Contact Person Name
Ana Pros Simon
Contact Person Email
apros@parcdesalutmar.cat
Site Name
Hospital Universitario 12 De Octubre
Department Name
Servicio de Reumatología
Contact Person Name
Patricia Esmeralda Carreira Delgado
Contact Person Email
carreira@h12o.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Servicio de Reumatología
Contact Person Name
Ernesto Trallero
Site Name
Complexo Hospitalario Universitario A Coruna
Department Name
Servicio de Reumatología
Contact Person Name
Mercedes Freire González
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Servicio de Reumatología
Contact Person Name
Francisco Javier Bachiller Corral
Contact Person Email
fbachiller@salud.madrid.org

Belgium

Earliest CTIS Part Ii Submission Date
16-05-2024
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
726
Number Of Sites
3
Number Of Participants
4

Sites

Site Name
UZ Leuven
Department Name
Reumatologie
Contact Person Name
Ellen De Langhe
Contact Person Email
ellen.delanghe@uzleuven.be
Site Name
Ziekenhuis Aan De Stroom
Department Name
Reumatologie
Contact Person Name
Alla Ishchenko
Contact Person Email
zna.medsec-reumatologie@zas.be
Site Name
Universitair Ziekenhuis Gent
Department Name
Reumatologie
Contact Person Name
Vanessa Smith
Contact Person Email
vanessa.smith@ugent.be

Denmark

Earliest CTIS Part Ii Submission Date
11-07-2024
Latest Decision Or Authorization Date
11-05-2026
Processing Time Days
669
Number Of Sites
4
Number Of Participants
6

Sites

Site Name
Rigshospitalet
Department Name
Neurology
Contact Person Name
Nanna Witting
Contact Person Email
nanna.witting@regionh.dk
Site Name
Aarhus Universitetshospital
Department Name
Rheumatology
Contact Person Name
Ellen-Margrethe Hauge
Contact Person Email
ellen.hauge@aarhus.rm.dk
Site Name
Rigshospitalet
Department Name
Rheumatology
Contact Person Name
Louise Pyndt Diederichsen
Contact Person Email
louise.diederichsen@regionh.dk
Site Name
Odense University Hospital
Department Name
Rheumatology
Contact Person Name
Kasper Søltoft
Contact Person Email
Kasper.Soeltoft@rsyd.dk

Hungary

Earliest CTIS Part Ii Submission Date
12-06-2024
Latest Decision Or Authorization Date
15-05-2026
Processing Time Days
702
Number Of Sites
4
Number Of Participants
6

Sites

Site Name
Betegapolo Irgalmasrend Budai Irgalmasrendi Korhaz
Department Name
Klinikai Farmakologia
Contact Person Name
Zsuzsanna Barko
Contact Person Email
drbarkozsuzsanna@gmail.com
Site Name
University Of Szeged
Department Name
Szent-Gyorgyi Albert Klinikai Kozpont Reumatologiai es Immunologiai Klinika
Contact Person Name
Laszlo Kovacs
Contact Person Email
kovacs.laszlo@med.u-szeged.hu
Site Name
University Of Debrecen
Department Name
Belgyogyaszati Klinika
Contact Person Name
Zoltan Griger
Contact Person Email
griger.zoltan@med.unideb.hu
Site Name
Semmelweis University
Department Name
Reumatológiai és Immunológiai Klinika Immunológia
Contact Person Name
Kinga Fritsch
Contact Person Email
kflinta@hotmail.com

Netherlands

Earliest CTIS Part Ii Submission Date
24-07-2024
Latest Decision Or Authorization Date
13-05-2026
Processing Time Days
658
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Academisch Medisch Centrum
Department Name
Neurology
Contact Person Name
Anneke van der Kooi
Contact Person Email
a.j.kooi@amc.uva.nl

Czechia

Earliest CTIS Part Ii Submission Date
16-05-2024
Latest Decision Or Authorization Date
11-05-2026
Processing Time Days
695
Number Of Sites
1
Number Of Participants
5

Sites

Site Name
Revmatologicky Ustav
Department Name
Vědeckovýzkumné centrum
Contact Person Name
Jiří Vencovský
Contact Person Email
vencovsky@revma.cz

Italy

Earliest CTIS Part Ii Submission Date
16-05-2024
Latest Decision Or Authorization Date
15-05-2026
Processing Time Days
699
Number Of Sites
12
Number Of Participants
10

Sites

Site Name
Azienda Ospealiero Universitaria Policlinico Umberto I
Department Name
Reumatology Unit
Contact Person Name
Fabrizio Conti
Contact Person Email
fabrizio.conti@uniroma1.it
Site Name
Humanitas Mirasole S.p.A.
Department Name
Division of Rheumatology and Clinical Immunology Laboratory of Autoimmunity and Metabolism
Contact Person Name
Elisa Gremese
Contact Person Email
elisa.gremese@hunimed.eu
Site Name
Azienda Ospedaliera Universitaria Senese
Department Name
UOC Reumatologia
Contact Person Name
Bruno Frediani
Contact Person Email
bruno.frediani@unisi.it
Site Name
Universita' Politecnica Delle Marche
Department Name
Department of Molecular and Clinical Sciences
Contact Person Name
Gianluca Moroncini
Contact Person Email
g.moroncini@staff.univpm.it
Site Name
Fondazione Policlinico Universitario Campus Bio-Medico
Department Name
Immunoreuomatology Unity
Contact Person Name
Roberto Giacomelli
Site Name
Azienda Ospedaliero Universitaria Policlinico G Rodolico San Marco Di Catania
Department Name
Reumatology
Contact Person Name
Rosario Foti
Contact Person Email
rosfoti5@gmail.com
Site Name
University Hospital Consorziale Policlinico
Department Name
Rheumatology Institute
Contact Person Name
Marco Fornaro
Contact Person Email
marco.fornaro@uniba.it
Site Name
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Department Name
PROMISE
Contact Person Name
Giuliana Guggino
Contact Person Email
giuliana.guggino@unipa.it
Site Name
Azienda Ospedaliero-Universitaria Sant Andre
Department Name
Neurology Unit
Contact Person Name
Matteo Garibaldi
Contact Person Email
matteo.garibaldi@uniroma1.it
Site Name
Ospedale San Raffaele S.r.l.
Department Name
Division of Neuroscience
Contact Person Name
Stefano Previtali
Contact Person Email
Previtali.stefano@hsr.it
Site Name
Azienda Ospedale-Universita Padova
Department Name
Department of Medicine
Contact Person Name
Luca Iaccarino
Contact Person Email
luca.iaccarino@unipd.it
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
Rheumatology
Contact Person Name
Lorenzo Cavagna
Contact Person Email
Lorenzo.cavagna@unipv.it

Poland

Earliest CTIS Part Ii Submission Date
18-07-2024
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
663
Number Of Sites
8
Number Of Participants
12

Sites

Site Name
Narodowy Instytut Geriatrii Reumatologii I Rehabilitacji Im Prof. Dr Hab. Med. Eleonory Reicher
Department Name
Narodowy Instytut Geriatrii Reumatologii I Rehabilitacji Im. Prof. Dr Hab. Med. Eleonory Reicher
Contact Person Name
Piotr Szczesny
Contact Person Email
piotr.szczesny@spartanska.pl
Site Name
Uniwersytecki Szpital Kliniczny Nr 2 Uniwersytetu Medycznego W Lodzi SPZOZ
Department Name
Klinika Reumatologii i Immunologii Klinicznej z Pododdziałem Chorób Wewnetrznych
Contact Person Name
Joanna Makowska
Contact Person Email
joanna.makowska@umed.lodz.pl
Site Name
Linden Sp. z o.o. sp.k.
Department Name
CENTRUM MEDYCZNE LINDEN
Contact Person Name
Weronika Kleczynska-Szpakiewicz
Contact Person Email
weronika.kleczynska@gmail.com
Site Name
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
Department Name
Oddział Kliniczny Reumatologii
Contact Person Name
Piotr Wiland
Contact Person Email
piotr.wiland@umw.edu.pl
Site Name
MEDYCZNE CENTRUM HETMANSKA Piotr Leszczynski
Department Name
MEDYCZNE CENTRUM HETMANSKA
Contact Person Name
Piotr Leszczynski
Site Name
Malopolskie Badania Kliniczne Sp. z o.o.
Department Name
Malopolskie Badania Kliniczne
Contact Person Name
Bogdan Batko
Contact Person Email
bpbatko@gmail.com
Site Name
Ortopedyczno-Rehabilitacyjny Szpital Kliniczny Im Wiktora Degi Uniwersytetu Medycznego Im Karola Marcinkowskiego W Poznaniu
Department Name
Oddział Reumatologii, Rehabilitacji i Chorób Wewnetrznych
Contact Person Name
Wlodzimierz Samborski
Contact Person Email
wsamborski@orsk.pl
Site Name
Medicover Integrated Clinical Services Sp. z o.o.
Department Name
MICS Centrum Medyczne Warszawa
Contact Person Name
Malgorzata Gryka-Marton
Contact Person Email
kontakt@medycynakliniczna.pl

Sweden

Earliest CTIS Part Ii Submission Date
19-07-2024
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
662
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Uppsala University Hospital
Department Name
Reumatologkliniken, Dag Hammarskjölds Väg 8, Akademiska Sjukhuset, Uppsala
Contact Person Name
Elisabeth Skoglund

Germany

Earliest CTIS Part Ii Submission Date
19-07-2024
Latest Decision Or Authorization Date
12-05-2026
Processing Time Days
662
Number Of Sites
7
Number Of Participants
6

Sites

Site Name
Klinikum der Universitaet Muenchen AöR
Department Name
Medizinische Klinik und Poliklinik IV
Contact Person Name
Hendrik Schulze-Koops
Site Name
Kerckhoff-Klinik GmbH
Department Name
"Abteilung für Rheumatologie und Klinische Immunologie"
Contact Person Name
Philipp Klemm
Contact Person Email
p.klemm@kerckhoff-klinik.de
Site Name
Medical Center - University Of Freiburg
Department Name
Klinik für Rheumatologie und Klinische Immunologie, Therapiestudienzentrum / Studienambulanz
Contact Person Name
Stephanie Finzel
Site Name
St. Elisabeth Gruppe GmbH Katholische Kliniken Rhein-Ruhr
Department Name
Rheumazentrum Ruhrgebiet
Contact Person Name
Ioana Andreica
Site Name
Universitaetsklinikum Bonn AöR
Department Name
Medizinische Klinik und Poliklinik III
Contact Person Name
Valentin Schäfer
Contact Person Email
valentin.schaefer@ukbonn.de
Site Name
Universitaetsklinikum Tuebingen AöR
Department Name
Innere Medizin II
Contact Person Name
Jörg Henes
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Klinik für Dermatologie, Venerologie und Allergologie
Contact Person Name
Margitta Worm
Contact Person Email
margitta.worm@charite.de

Austria

Earliest CTIS Part Ii Submission Date
05-06-2024
Latest Decision Or Authorization Date
13-05-2026
Processing Time Days
707
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Universitätsklinikum Graz
Department Name
University Clinic for Internal Medicine Clinical Department of Rheumatology and Immunology
Contact Person Name
Jens Thiel
Contact Person Email
jens.thiel@medunigraz.at
Site Name
Medical University Of Vienna
Department Name
Innere Medizin III Abtl. Rheumatologie
Contact Person Name
Helga Lechner-Radner

Sponsor

Primary sponsor

Full Name
AstraZeneca AB
Organisation Type
Pharmaceutical company
Country Of Registered Address
Sweden

Investigational products

Investigational Product Name
Anifrolumab
Active Substance
ANIFROLUMAB
Modality
Monoclonal antibody
Routes Of Administration
SUBCUTANEOUS USE
Route
Subcutaneous
Authorisation Status
Authorised
Orphan Designation
Yes
Starting Dose
120 mg
Dose Levels
120 mg
Frequency
Weekly (QW)
Investigational Product Name
Anifrolumab Placebo
Modality
Other
Route
Subcutaneous
Starting Dose
placebo (matching SC QW schedule)
Frequency
Weekly (QW)
Combination Treatment
Yes

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