An open-label extension study with dazukibart for participants with idiopathic inflammatory myopathies (including DM or PM) who have completed the treatment period of a qualifying parent study.

Start 14 Mar 2025 · Status Ongoing, recruiting · 6 EU/EEA countries · 11 sites · Protocol C0251010

Overview

Sponsor-declared trial summary

Phase Therapeutic confirmatory (Phase III)

Status Ongoing, recruiting

Participants planned 184

Countries 6

Sites 11

Idiopathic Inflammatory Myopathy (including dermatomyositis or polymyositis)

To evaluate the long-term safety of dazukibart in participants with DM or PM

Key facts

Sponsor: Pfizer Inc.
Participant type: Patients
Age range: 18-64 years, 65+ years
Gender: Male and Female
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05], Diseases [C] - Immune System Diseases [C20]
Trial duration: 14 Mar 2025 → ongoing
Decision date (initial): 2025-01-22
Transition trial: No
Low-intervention: No
Rare-disease indication: Yes
Vulnerable population: No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Pharmacogenetic, Pharmacogenomic, Therapy

To evaluate the long-term safety of dazukibart in participants with DM or PM

Secondary objectives 6

To evaluate the efficacy of dazukibart in reducing muscle (Cohort 1 and Cohort 2) and skin (Cohort 1) signs and symptoms in participants with DM or PM treated with dazukibart in this study.
To evaluate the health impact of dazukibart on patient reported outcomes in participants with DM or PM treated with dazukibart in this study.
To evaluate the change in background medication use in participants with DM or PM treated with dazukibart in this study.
To evaluate the rescue therapy-averting effects of dazukibart in participants with DM or PM treated with dazukibart in this study.
To evaluate the long-term safety of dazukibart on myositis specific autoantibodies in participants with DM or PM treated with dazukibart in this study.
To monitor for immunogenicity in participants with DM or PM treated with dazukibart in this study who had positive ADAs prior to entry into this study.

Conditions and MedDRA coding

Idiopathic Inflammatory Myopathy (including dermatomyositis or polymyositis)

Version	Level	Code	Term	System organ class
24.1	PT	10085970	Idiopathic inflammatory myopathy	100000004859
20.0	PT	10036102	Polymyositis	100000004859
20.0	PT	10012503	Dermatomyositis	100000004858

Regulatory references

Scientific advice from competent authorities: Pharmaceuticals And Medical Devices Agency, European Medicines Agency, Food And Drug Administration, Center For Drug Evaluation
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 1

Participants who have completed the treatment period of a qualifying study and did not discontinue study treatment early will be eligible to receive study drug (OL Treatment Groups). From C0251006 study, participants must have completed the 52-week treatment period of the study to receive study drug in this study.

Exclusion criteria 5

Any medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator’s judgment, make the participant inappropriate for the study.
Previous administration with an investigational product (drug or vaccine) other than dazukibart in a qualifying study within 30 days (or as determined by the local requirement) or 5 half-lives preceding baseline in this study (whichever is longer).
Current use of any prohibited concomitant medication(s).
Active bacterial, viral, fungal, mycobacterial or other infections (including but not limited to tuberculosis (TB) and atypical mycobacterial disease).
Participants with an ongoing adverse event in a qualifying study which, in the opinion of the investigator or sponsor, is an ongoing safety concern and makes the participant inappropriate for the study, OR the participant has met safety monitoring criteria in a qualifying study that have not resolved.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

TEAEs, SAEs, AESIs, and AEs leading to treatment discontinuation; Clinically significant abnormalities in laboratory tests, ECG measurements, and vital signs; Change from baseline in FVC/DLCO; Absolute values and change from baseline in C-SSRS at all scheduled timepoints.

Secondary endpoints 7

Cohort 1 (DM) and Cohort 2 (PM): • Change from baseline* in MMT-8 score at all scheduled timepoints. • Change from baseline* in PhGA at all scheduled timepoints. • Change from baseline* in extramuscular activity or disease activity score at all scheduled timepoints. • Change from baseline* in results in muscle enzymes at all scheduled timepoints. • Minimal, Moderate, and Major improvement in TIS at all scheduled timepoints. • TIS (continuous) at all scheduled timepoints.
Cohort 1 (DM) and Cohort 2 (PM): Change from baseline at all scheduled timepoints in: PROMIS-PF, PtGA, HAQ-DI, FACIT-F, EQ-5D-5L and EQ-VAS, HRU questionnaire; Cohort 1 (DM) only: Change from baseline at all scheduled timepoints in: 5D-Itch Scale
Change from baseline in corticosteroid dose over time; Response over time based on participants achieving: Corticosteroid dose ≤5 mg/day, Corticosteroid free; Change from baseline in non-steroid immunosuppressant/immunomodulator and antimalarial dose over time. Response over time based on participants achieving: Non-steroid immunosuppressants/immunomodulator and antimalarial free.
Receive rescue therapy (yes or no) during the study; Number/cycles of rescue therapy received during the study.
Auto antibodies (eg. TIF1-γ/P155, NXP2/P140, SAE, JO-1 and MDA-5).
Baseline* and post-treatment ADAs and Nabs.
To be continued - secondary endpoint for secondary objective nr.1: Cohort 1 (DM) Only: Percent change from baseline and change from baseline in CDASI-A score at all scheduled timepoints; Change from baseline in CDASI-D score at all scheduled timepoints.

Investigational products

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

Test 1

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: SOLUTION FOR INJECTION
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

Auxiliary 18

Deflazacort

SUB06943MIG · Substance

Active substance: Deflazacort
Pharmaceutical form: TABLET
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

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

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

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

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

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

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

Hydroxychloroquine

SUB08077MIG · Substance

Active substance: Hydroxychloroquine
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 400 mg milligram(s)
Max total dose: 400 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

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

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

Triamcinolone Acetonide

SUB04936MIG · Substance

Active substance: Triamcinolone Acetonide
Pharmaceutical form: SUSPENSION FOR INJECTION
Route of administration: INJECTION
Max daily dose: 48 mg milligram(s)
Max total dose: 48 mg milligram(s)
Max treatment duration: 42 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

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

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

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

Chloroquine Phosphate

SUB01238MIG · Substance

Active substance: Chloroquine Phosphate
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 250 mg milligram(s)
Max total dose: 250 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: 66 Hudson Boulevard East
City: New York
Postcode: 10001-2189
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 5

Organisation	City, country	Duties
Premier Research ORL-000003568	Morrisville, United States	Other
Ppd Inc. ORG-100018960	Wilmington, United States	Other
Signant Health Global Solutions Limited ORG-100047290	Dublin 2, Ireland	Other
Medpace Inc. ORG-100026760	Cincinnati, United States	Other
Labcorp Central Laboratory Services LP ORG-100032236	Indianapolis, United States	Other

Locations

6 EU/EEA countries · 11 investigational sites

By country

Country	MS status	Planned subjects	Sites
Bulgaria	Ongoing, recruiting	5	2
Hungary	Ongoing, recruiting	4	1
Italy	Authorised, recruitment pending	1	1
Poland	Ongoing, recruiting	9	4
Spain	Authorised, recruitment pending	2	2
Sweden	Authorised, recruitment pending	2	1
Rest of world Argentina, Mexico, India, Korea, Republic of, Japan, Taiwan, Israel, China, United Kingdom, Turkey, United States	—	161	—

Investigational sites

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

Hungary

1 site · Ongoing, recruiting

University Of Debrecen

Belgyógyászati Klinika, Klinikai Immunológiai Tanszék, Moricz Zsigmond Korut 22, 4032, Debrecen

Italy

1 site · Authorised, recruitment pending

Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania

Rheumatology, Viale Azeglio Ciampi Snc, 95121, Catania

Poland

4 sites · Ongoing, recruiting

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

N/A, 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

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

Spain

2 sites · Authorised, recruitment pending

Hospital Universitario 12 De Octubre

Rheumatology, Avenida De Cordoba Sn, 28041, Madrid

Hospital General Universitario Gregorio Maranon

Rheumatology, Calle Del Doctor Esquerdo 46, 28007, Madrid

Sweden