Active Idiopathic… · Phase III (2022-502739-20-00)

Start 16 Nov 2023 · Status Authorised, recruiting · 10 EU/EEA countries · 30 sites · Protocol C0251006

Overview

Sponsor-declared trial summary

Phase Therapeutic confirmatory (Phase III)

Status Authorised, recruiting

Participants planned 318

Countries 10

Sites 30

Active Idiopathic Inflammatory Myopathies (Including Dermatomyositis or Polymyositis)

To evaluate the efficacy of dazukibart compared with placebo in reducing muscle symptoms in adult participants with active DM and adult participants with active PM.

Key facts

Sponsor: Pfizer Inc.
Participant type: Patients
Age range: 18-64 years, 65+ years
Gender: Male and Female
Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trial duration: 16 Nov 2023 → ongoing
Decision date (initial): 2023-10-16
Transition trial: No
Low-intervention: No
Rare-disease indication: Yes
Vulnerable population: Yes
Funding sources: Pfizer, Inc.

External identifiers

EU CT number: 2022-502739-20-00
ClinicalTrials.gov: NCT05895786

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To evaluate the efficacy of dazukibart compared with placebo in reducing muscle symptoms in adult participants with active DM and adult participants with active PM.

Secondary objectives 1

To evaluate efficacy of dazukibart compared with placebo in reducing skin (Cohort 1) and muscle signs and symptoms (Cohorts 1 and 2) and patient health status in adult participants with active DM and adult participants with active PM.

Conditions and MedDRA coding

Active Idiopathic Inflammatory Myopathies (Including Dermatomyositis or Polymyositis)

Version	Level	Code	Term	System organ class
20.0	PT	10012503	Dermatomyositis	100000004858
20.0	PT	10036102	Polymyositis	100000004859

Regulatory references

Scientific advice from competent authorities: Pharmaceuticals And Medical Devices Agency, Food And Drug Administration, European Medicines Agency
Plan to share IPD: Yes
IPD plan description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

Adult (aged ≥ 18 years old or minimum legal adult age as defined per local regulation, whichever is greater.
Definite or probable IIM as per ACR/EULAR Classification criteria of IIM with probability ≥55%, with confirmation of IIM subtypes: DM based on age at onset of first symptoms (DM ≥18 years) AND two of the following: Gottron’s papules; Gottron’s sign; Heliotrope eruption; Serology with at least 1 positive of the following TIF1-ƴ/P155, NXP2/P140, Mi2, MDA5, SAE 1 and/or 2, JO-1, PL-12, PL-7, EJ, or OJ. PM, age of onset of first symptoms ≥18 years AND the following: Absence of pathognomonic skin manifestations characteristic of DM (Gottron’s papules, Gottron’s sign, and Heliotrope rash), Muscle weakness pattern characteristic of myositis (eg, symmetric muscle weakness of the proximal upper/lower extremities; or neck flexors are relatively weaker than neck extensors; or in the legs proximal muscles are weaker than distal muscles), With EITHER of the following: Serology with at least 1 positive anti-synthetase autoantibodies (JO-1, PL-12, PL-7, EJ, OJ), OR Evidence of muscle biopsy confirming PM diagnosis.
Activity disease that fulfills the following criteria: 1. MMT-8 score ≤141 (out of 150 total possible). 2. At least 2 of the following abnormal CSM as a numerical scale (derived from VAS, where applicable) and/or objective measures of active muscle disease: • Patient global activity ≥2-points; • Physician’s global disease activity ≥2-points; • Extra-muscular activity (MDAAT) ≥2-points; • HAQ-DI ≥0.25. 3. At least 1 muscle enzyme >1.3 ×ULN, a magnetic resonance imaging (MRI) report within 12 weeks prior to Screening confirming active muscle disease (eg, findings of edema in skeletal muscle suggested by increased signal on T2 and short tau inversion recovery (STIR) sequences or by gadolinium enhancement), OR a muscle biopsy report within 12 weeks prior to Screening indicating inflammatory cell infiltration or elevated expression of inflammatory proteins, including but not limited to, human myxovirus resistance protein 1 (MxA) and major histocompatibility complex class I (MHC I), due to underlying DM or PM and the absence of rimmed vacuoles or necrotic fibers which may be pathognomonic of inclusion body myositis (IBM) and immune-mediated necrotizing myositis (IMNM).
Must be receiving a stable dose of SOC background medications at the time of enrollment, defined as a stable dose of: (1) 1 oral corticosteroid, or (2) 1 immunosuppressant, or (3) a combination of 1 oral corticosteroid and 1 immunosuppressant as background therapy. For example, a participant who may be receiving only 1 immunosuppressant may have a contraindication or intolerance, or has had an inadequate response to corticosteroids prescribed to control disease.

Exclusion criteria 1

Medical conditions pertaining to DM or PM: • Myositis due to non-IIM. • Existing diagnosis of IBM. • IMNM, including presence of positive anti-SRP and anti-HMGCR antibody confirmed by medical history. • Myositis with end-stage organ involvement at Screening or Visit 1. • Inability to walk or bound to a wheelchair. Requiring oxygen supplementation at the time of screening.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

Cohort 1 (DM) - Global (except US): Moderate improvement in TIS at Week 24; Cohort 2 (PM) - Global (except US): Moderate improvement in TIS at Week 24

Secondary endpoints 3

Cohort 1 (DM): Global (except US) Change from baseline in MMT-8 score at Week 24. Change from baseline in Cutaneous Dermatomyositis Disease Area and Severity Index Activity Score (CDASI-A) at Week 24 for participants with baseline CDASI-A score ≥14. Normalized area under the dose-time curve (AUC) of corticosteroid dose over 52 weeks. Moderate improvement in TIS at Week 52.
Cohort 1 (DM): Global (except US): Change from baseline in PROMIS-PF at week 24. Change from baseline in 5-D Itch Scale Score at Week 24 for participants with baseline CDASI-A score ≥14.  Change from baseline in FACIT-F score at Week 24.
Cohort 2 (PM): Global (except US) Change from baseline in MMT-8 score at Week 24. Normalized AUC of corticosteroid dose over 52 weeks. Moderate improvement in TIS at Week 52. Change from baseline in PROMIS-PF at Week 24. Change from baseline in FACIT-F score at Week 24.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Dazukibart

PRD11222618 · Product

Active substance: Dazukibart
Substance synonyms: PF-06823859, Humanised IgG1K monoclonal antibody against interferon beta
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS ADMINISTRATION
Max daily dose: 600 mg milligram(s)
Max total dose: 7800 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Not Authorised
MA holder: PFIZER INC.
Paediatric formulation: No
Orphan designation: Yes
Orphan designation number: EU/3/20/2392

PF-06823859

PRD10183352 · Product

Active substance: Humanised IGG1K Monoclonal Antibody Against Interferon Beta
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS ADMINISTRATION
Max daily dose: 600 mg milligram(s)
Max total dose: 7800 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Not Authorised
MA holder: PFIZER INC.
Paediatric formulation: No
Orphan designation: Yes
Orphan designation number: EU/3/20/2392

Placebo 2

Placebo for PF-06823859 solution for injection

N/A · Product

Other product name: N/A
Pharmaceutical form: N/A
ATC code: N/A — N/A
Marketing authorisation: N/A
MA holder: N/A
MA country: Iceland
Paediatric formulation: No

Placebo for dazukibart 60mg/ml solution for injection

N/A · Product

Other product name: N/A
Pharmaceutical form: N/A
ATC code: N/A — N/A
Marketing authorisation: N/A
MA holder: N/A
MA country: Iceland
Paediatric formulation: No

Auxiliary 18

Betamethasone

SUB05797MIG · Substance

Active substance: Betamethasone
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 7.2 mg milligram(s)
Max total dose: 7.2 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Mycophenolate Mofetil

SUB03360MIG · Substance

Active substance: Mycophenolate Mofetil
Pharmaceutical form: CAPSULES
Route of administration: ORAL
Max daily dose: 3000 mg milligram(s)
Max total dose: 3000 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Hydrocortisone

SUB08065MIG · Substance

Active substance: Hydrocortisone
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 240 mg milligram(s)
Max total dose: 240 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Methotrexate

SUB08856MIG · Substance

Active substance: Methotrexate
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 3.5 mg milligram(s)
Max total dose: 3.5 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Hydroxychloroquine Sulfate

SCP134762 · ATC

Active substance: Hydroxychloroquine Sulfate
Substance synonyms: 2-[4-[(7-CHLOROQUINOLIN-4-YL)AMINO]PENTYL-ETHYL-AMINO]ETHANOL, SULFURIC ACID, HYDROXYCHLOROQUINE SULPHATE
Route of administration: ORAL
Max daily dose: 400 mg milligram(s)
Max total dose: 89600 mg milligram(s)
Max treatment duration: 32 Week(s)
Authorisation status: Authorised
ATC code: P01BA02 — HYDROXYCHLOROQUINE
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Chloroquine Phosphate

SCP134358 · ATC

Active substance: Chloroquine Phosphate
Route of administration: ORAL
Max daily dose: 250 mg milligram(s)
Max total dose: 56000 mg/g milligram(s)/gram
Max treatment duration: 32 Week(s)
Authorisation status: Authorised
ATC code: P01BA01 — CHLOROQUINE
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Deflazacort

SUB06943MIG · Substance

Active substance: Deflazacort
Pharmaceutical form: TABLETS
Route of administration: ORAL
Max daily dose: 72 mg milligram(s)
Max total dose: 72 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Betamethasone

SUB05797MIG · Substance

Active substance: Betamethasone
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS ADMINISTRATION
Max daily dose: 7.2 mg milligram(s)
Max total dose: 7.2 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Methylprednisolone

SUB08872MIG · Substance

Active substance: Methylprednisolone
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 48 mg milligram(s)
Max total dose: 48 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Prednisone

SUB10020MIG · Substance

Active substance: Prednisone
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 60 mg milligram(s)
Max total dose: 60 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Azathioprine

SUB05647MIG · Substance

Active substance: Azathioprine
Pharmaceutical form: ORAL SUSPENSION
Route of administration: ORAL
Max daily dose: 150 mg milligram(s)
Max total dose: 150 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Triamcinolone Acetonide

SUB04936MIG · Substance

Active substance: Triamcinolone Acetonide
Pharmaceutical form: SUSPENSION FOR INJECTION
Route of administration: INTRADERMAL
Max daily dose: 48 mg milligram(s)
Max total dose: 48 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Leflunomide

SUB08424MIG · Substance

Active substance: Leflunomide
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 20 mg milligram(s)
Max total dose: 20 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Dexamethasone

SUB07017MIG · Substance

Active substance: Dexamethasone
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS INJECTION
Max daily dose: 9 mg milligram(s)
Max total dose: 9 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Methotrexate

SUB08856MIG · Substance

Active substance: Methotrexate
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS INJECTION
Max daily dose: 3.5 mg milligram(s)
Max total dose: 3.5 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Budesonide

SUB05955MIG · Substance

Active substance: Budesonide
Pharmaceutical form: PROLONGED-RELEASE CAPSULE, HARD
Route of administration: ORAL
Max daily dose: 18 mg milligram(s)
Max total dose: 18 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Prednisolone

SUB10018MIG · Substance

Active substance: Prednisolone
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 60 mg milligram(s)
Max total dose: 60 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Methylprednisolone

SUB08872MIG · Substance

Active substance: Methylprednisolone
Pharmaceutical form: SOLUTION FOR INJECTION/INFUSION
Route of administration: INTRAMUSCULAR
Max daily dose: 48 mg milligram(s)
Max total dose: 48 mg milligram(s)
Max treatment duration: 52 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Pfizer Inc.

Sponsor organisation: Pfizer Inc.
Address: 235 East 42nd Street
City: New York
Postcode: 10017-5703
Country: United States

Scientific contact point

Organisation: Pfizer Inc.
Contact name: Clinical Medical Lead

Public contact point

Organisation: Pfizer Inc.
Contact name: Clinical Medical Lead

Third parties 16

Organisation	City, country	Duties
Labcorp Central Laboratory Services LP ORG-100032236	Indianapolis, United States	Other, Laboratory analysis
TecEx ORL-000006567	Virginia Beach, United States	Other
Premier Research ORL-000003568	Morrisville, United States	Other
Innovative Trials Limited ORG-100044081	Letchworth Garden City, United Kingdom	Other
WCG Clinical Inc. ORG-100040730	Princeton, United States	Other
Clario ORL-000001443	United States	Other
Ppd Inc. ORG-100018960	Wilmington, United States	Other
Canfield Scientific Inc. ORG-100042834	Parsippany, United States	Other
Signant Health Global LLC ORG-100040604	Blue Bell, United States	Other
Scout Clinical ORG-100042228	Dallas, United States	Other
Q2 Solutions LLC ORG-100017000	Ithaca, United States	Other
Clariness GmbH ORG-100045306	Hamburg, Germany	Other
QPS LLC ORG-100012847	Newark, United States	Other
Syneos Health Inc. ORG-100008382	Morrisville, United States	On site monitoring, Code 12, Other, Code 5
Dxterity Diagnostics Inc. ORG-100044632	Rancho Dominguez, United States	Other
Medpace Inc. ORG-100026760	Cincinnati, United States	Other

Locations

10 EU/EEA countries · 30 investigational sites

By country

Country	MS status	Planned subjects	Sites
Belgium	Ended	2	1
Bulgaria	Ongoing, recruiting	10	2
France	Ongoing, recruiting	6	4
Germany	Ongoing, recruiting	5	3
Hungary	Ongoing, recruiting	1	2
Italy	Ongoing, recruiting	18	5
Poland	Ongoing, recruiting	20	7
Slovakia	Ongoing, recruiting	3	1
Spain	Ongoing, recruiting	13	4
Sweden	Ongoing, recruiting	1	1
Rest of world Mexico, Korea, Republic of, Israel, Japan, United Kingdom, India, Taiwan, China, United States, Turkey, Argentina	—	239	—

Investigational sites

Belgium

1 site · Ended

UZ Leuven

Neurology, Herestraat 49, 3000, Leuven

Bulgaria

2 sites · Ongoing, recruiting

Medical Center Artmed Ltd.

N/A, Ulitsa Mladost 8, 4002, Plovdiv

Dkc Fokus-5 Lzip OOD

N/A, Ulitsa Hristo Stanchev 15, 1463, Sofiya

France

4 sites · Ongoing, recruiting

Hopitaux Universitaires Pitie Salpetriere

Internal Medicine, 47 To 83 Boulevard De L Hopital, 75013, Paris

Les Hopitaux Universitaires De Strasbourg

Department of Physiology and Functional Explorations, Muscle Function Exploration, 1 Place De L Hopital, Cs 80426, Strasbourg Cedex

Hospital Edouard Herriot

Internal Medicine, 5 Place D Arsonval, 69003, Lyon

Centre Hospitalier Universitaire Reims

Rheumatology, Rue Du General Koenig, 51092, Reims Cedex

Germany

3 sites · Ongoing, recruiting

Klinikum der Universitaet Muenchen AöR

Medizinische Klinik und Poliklinik IV, Pettenkoferstrasse 8a, Ludwigsvorstadt-Isarvorstadt, Munich

Universitaetsklinikum Ulm AöR

Klinik für Neurologie, Oberer Eselsberg 45, Eselsberg, Ulm

Universitaetsklinikum Erlangen AöR

Hautklinik, Ulmenweg 18, Innenstadt, Erlangen

Hungary

2 sites · Ongoing, recruiting

University Of Pecs

Reumatológiai és Immunológiai Klinika, Akac Utca 1, 7632, Pecs

University Of Debrecen

Belgyogyaszati Klinika, Klinikai Immunologiai Tanszek, Moricz Zsigmond Korut 22, 4032, Debrecen

Italy

5 sites · Ongoing, recruiting

Humanitas Research Hospital

Rheumatology and Clinical Immunology Unit, Via Alessandro Manzoni 56, 20089, Rozzano

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Science of aging, orthopedic and rheumatology, Largo Francesco Vito 1, 00168, Rome

Careggi University Hospital

Rheumatology unit, Largo Giovanni Alessandro Brambilla 3, 50134, Florence

Azienda Ospedaliero Universitaria Policlinico G Rodolico San Marco Di Catania

Rheumatology Unit, Via Santa Sofia 78, 95123, Catania

Ospedale San Raffaele S.r.l.

UO Immunologia, Reumatologia, Allergologia e Mala, Via Olgettina 60, 20132, Milan

Poland

7 sites · Ongoing, recruiting

Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie

Oddzial Kliniczny Reumatologii, Immunologii i Chorob Wewnetrznych, Ul. Macieja Jakubowskiego 2, 30-688, Cracow

Malopolskie Centrum Kliniczne

N/A, Ul. Balicka 12a/5b, 30-149, Cracow

Twoja Przychodnia Poznanskie Centrum Medyczne Sp. z o.o.

N/A, Ul. Marcelinska 92, 60-324, Poznan

Narodowy Instytut Geriatrii Reumatologii I Rehabilitacji Im Prof. Dr Hab. Med. Eleonory Reicher

Centrum Wsparcia Badan Klinicznych, Ul. Spartanska 1, 02-637, Warsaw

Nova Reuma Domyslawska I Rusilowicz Lekarza Reumatologa I Fizjoterapeuty sp.p.

N/A, Ul Prowiantowa 15/4, 15-707, Bialystok

INTER CLINIC Piotr Adrian Klimiuk

N/A, ul. Warszawska 52, 15-077, Bialystok

Centrum Medyczne Plejady Sp. z o.o. S.K.

N/A, U2 U4 U5, Ul. Tadeusza Szafrana 5d, Cracow

Slovakia

1 site · Ongoing, recruiting

Narodny Ustav Reumatickych Chorob

Reumatologická ambulancia, Nabrezie Ivana Krasku 4782/4, 921 01, Piestany

Spain

4 sites · Ongoing, recruiting

Hospital Universitario Infanta Leonor

Rheumatology Department, Avenida Gran Via Del Este 80, 28031, Madrid

Hospital Universitario 12 De Octubre

Rheumatology Department, Bloque D, Avenida De Cordoba S/n, Madrid

Hospital General Universitario Gregorio Maranon

Rheumatology Department, Calle Del Doctor Esquerdo 46, 28009, Madrid

University Hospital Of Canary Islands

Rheumatology Department, Carretera De La Cuesta Taco S/n, Cuesta La, San Cristobal De La Laguna

Sweden