A Study to Investigate the Efficacy and Safety of Anifrolumab Administered as Subcutaneous Injection and Added to Standard of Care Compared with Placebo Added to Standard of Care in Adult Participants with Idiopathic Inflammatory Myopathies (Polymyositis and Dermatomyositis)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

AstraZeneca AB

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Phenomena and Processes [G] - Immune System Phenomena [G13], Diseases [C] - Immune System Diseases [C20], Phenomena and Processes [G] - Musculoskeletal and Neural Physiological Phenomena [G11], Diseases [C] - Musculoskeletal Diseases [C05], Phenomena and Processes [G] - Immune system processes [G12]

Trial duration

13 Dec 2024 → ongoing

Decision date (initial)

2024-08-19

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

AstraZeneca AB

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To demonstrate the superiority of anifrolumab to placebo on moderate improvement in disease activity at Week 52

Secondary objectives 9

1. To demonstrate the superiority of anifrolumab to placebo on muscle improvement at Week 52
2. To demonstrate the superiority of anifrolumab to placebo on oral corticosteroid dose reduction at Week 52
3. To demonstrate the superiority of anifrolumab to placebo on moderate improvement in disease activity in PM participants at Week 52
4. To demonstrate the superiority of anifrolumab to placebo on moderate improvement in disease activity in DM participants at Week 52
5. To demonstrate the superiority of anifrolumab to placebo on improving skin activity at Week 8
6. To assess the efficacy of anifrolumab compared with placebo on muscle improvement in PM participants at Week 52
7. To assess the efficacy of anifrolumab compared with placebo on muscle improvement in DM participants at Week 52
8. To assess the efficacy of anifrolumab compared with placebo on OCS dose reduction in PM participants at Week 52
9. To assess the efficacy of anifrolumab compared with placebo on OCS dose reduction in DM participants at Week 52

Conditions and MedDRA coding

Polymyositis or dermatomyositis according to 2017 EULAR / ACR classification criteria

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

IPD plan description

NA

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. 18 - 75 years old
2. Body weight 40 kg - ≤ 100 kg
3. Must have “probable” or “definite” diagnosis of PM or DM according to the 2017 ACR/EULAR classification criteria for adult myositis.
4. Moderate or severe disease activity per core set measurements.
5. Currently receiving oral prednisone or other polymyositis or dermatomyositis treatments at a stable dose.
6. No history of active tuberculosis or severe COVID-19.
7. Male and female participants must follow contraception guidelines.

Exclusion criteria 11

1. 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).
2. PM and DM patients at a high risk of malignancy.
3. Participants with rapidly progressive interstitial lung disease.
4. 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.
5. Any history of severe case of herpes zoster infection
6. History of cancer (except adequately treated basal cell carcinoma or cervical cancer in-situ), immunodeficiency, HIV, HBV, active HCV .
7. Any clinical cytomegalovirus or Epstein-Barr virus infection that has not completely resolved within 12 weeks prior to signing the ICF.
8. Opportunistic infection requiring hospitalization or IV antimicrobial treatment within 3 years prior to randomization.
9. Recent non-opportunistic infection requiring hospitalization or anti-infective treatment.
10. Recent or concurrent enrollment in another clinical study with an investigational product.
11. Lactating, breastfeeding, or pregnant females or females who intend to become pregnant or begin breastfeeding

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Participants who have at least moderate improvement in disease activity (TIS ≥ 40) and has not met “confirmed deterioration” criteria at 2 consecutive visits

Secondary endpoints 9

1. MMT-8 (CSM) change from baseline at week 52
2. Participants who achieve oral corticosteroid dose ≤ 7.5 mg/day at week 52 (yes/no)
3. 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
4. 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
5. CDASI-activity change from baseline (DM participants only) at week 8
6. "MMT-8 (CSM) change from baseline at Week 52. Only PM participants will be included in the analysis."
7. "MMT-8 (CSM) change from baseline at Week 52 Only DM participants will be included in the analysis."
8. "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."
9. "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."

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

AstraZeneca AB

Sponsor organisation

AstraZeneca AB

Address

Astraallen Gartuna, Karlebyhus Byggnad 674 Karlebyhus Byggnad 674

City

Sodertalje

Postcode

151 85

Country

Sweden

Scientific contact point

Organisation

AstraZeneca AB

Contact name

AstraZeneca Clinical Study Information Center

Public contact point

Organisation

AstraZeneca AB

Contact name

AstraZeneca Clinical Study Information Center

Locations

13 EU/EEA countries · 70 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 139 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

15 submissions — initial application plus substantial / non-substantial modifications