A study to evaluate the efficacy, safety, and pharmacokinetics of IgPro20 in adults with dermatomyositis (DM)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

CSL Behring LLC

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

Trial duration

20 Feb 2020 → 2 Dec 2024

Decision date (initial)

2024-04-18

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2023-508293-28-00

EudraCT number

2018-003171-35

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Efficacy, Therapy, Safety

The primary objective of this study is to assess the efficacy of IgPro20 SC doses in comparison to placebo in adult subjects with DM, as measured by responder status based on the Total Improvement Score (TIS) assessments at Weeks 17, 21, and 25.

Secondary objectives 1

1. The secondary objectives of the study are to assess the efficacy, with additional clinical outcome measures, of IgPro20 in comparison to placebo, the safety of IgPro20 in comparison to placebo, safety and efficacy at Week 53, and safety after Week 53 to end of study participation of IgPro20

Conditions and MedDRA coding

Dermatomyositis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. male or female subjects ≥ 18 years of age with diagnosis of at least probable idiopathic inflammatory myopathies per European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) Classification Criteria which includes confirmation of dermatomyositis (DM) rash/manifestation, disease activity defined by presence of DM rash / manifestation or an objective disease activity measure, and disease severity defined by Physician global visual analog scale (VAS) with a minimum value of 2.0 cm on a 10 cm scale and MMT-8 ≤ 142 or CDASI total activity score ≥ 14.

Exclusion criteria 1

1. Cancer-associated myositis, evidence of active malignant disease or malignancies diagnosed within the previous 5 years, Physician Global Damage ≥ 3, or clinically relevant improvement between Screening Visit and Baseline

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Responder rate: A responder is defined as a subject with a total improvement score(TIS) ≥ 20 points at Week 25 and at least 1 of the previous scheduled visits (Week 17 or Week 21), who completes 24 weeks of randomized IMP treatment without the use of rescue corticosteroid treatment. The TIS is a sum response criterion which incorporates 6 weighted IMACS core set measures (CSMs).

Secondary endpoints 40

1. Mean Total Improvement Score (TIS) - Up to Week 25
2. Point estimates and 95% CI for mean difference (IgPro20 – placebo) in TIS - Up to Week 25
3. Mean changes from Baseline in Manual Muscle Testing (MMT-8) - Up to Week 25
4. Point estimates and 95% CI for mean change difference (IgPro20 – placebo) in MMT-8 - Up to Week 25
5. Mean changes from Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) total activity score - Up to Week 25
6. Point estimates and 95% CI for mean change difference (IgPro20 – placebo) - Up to Week 25
7. Number of subjects who are able to reduce the oral corticosteroid dose by ≥ 25% - Up to Week 25
8. Percentage and 95% CI of subjects who are able to reduce the oral corticosteroid dose by ≥ 25% - Up to Week 25
9. Point estimates and 95% CI for the odds ratio (IgPro20:Placebo) of subjects who are able to reduce the oral corticosteroid dose by ≥ 25% - Up to Week 25
10. Mean TIS - Week 5 up to Week 53
11. Percentage of subjects achieving TIS ≥ 20, ≥ 40, and ≥ 60 points - Week 5 up to Week 53
12. Time to first achieving TIS ≥ 20, ≥ 40, and ≥ 60 points on the TIS - Week 5 up to Week 53
13. Percentage of subjects achieving TIS ≥ 20 points at the end of study period 2 - Up to Week 53
14. Mean changes in individual CSMs (except muscle enzymes) and CDASI from Baseline - Between Week 5 and Week 25
15. Mean changes in individual CSMs (except muscle enzymes) and CDASI from Week 25 - Week 29 to Week 53
16. Number of subjects meeting Definition of Worsening (DOW) at least once, twice, or > twice - Baseline up to Week 53
17. Percentage of subjects meeting DOW at least once, twice, or > twice - Baseline up to Week 53
18. Time to meeting DOW for the first time - Baseline up to Week 53
19. Number of subjects meeting DOW and receiving rescue corticosteroid treatment - Baseline up to Week 53
20. Percentage of subjects meeting DOW and receiving rescue corticosteroid treatment - Baseline up to Week 53
21. Number of subjects who start oral corticosteroid dose taper - Baseline up to Week 53
22. Percentage of subjects who start oral corticosteroid dose taper - Baseline up to Week 53
23. Number of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%, ≥ 50%, ≥ 75% - Baseline up to Week 25
24. Number of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%, ≥ 50%, ≥ 75% - Baseline up to Week 53
25. Percentage of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%, ≥ 50%, ≥ 75% - Baseline up to Week 25
26. Percentage of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%, ≥ 50%, ≥ 75% - Baseline up to Week 53
27. Percentage of subjects receiving rescue corticosteroid treatment - Baseline up to Week 25
28. Percentage of subjects whose rescue corticosteroid treatment is tapered - Baseline up to Week 25
29. Time to first intake of rescue corticosteroid treatment - Baseline up to Week 25
30. Number of subjects having at least 1 level, 2 levels, and more than 2 levels of improvement from Baseline in mobility, self-care, and usual activities domains of EQ-5D-5L - Baseline up to Week 53
31. Percentage of subjects having at least 1 level, 2 levels, and more than 2 levels of improvement from Baseline in mobility, self-care, and usual activities domains of EQ-5D-5L - Baseline up to Week 53
32. Number of subjects having no reduction in levels, at least 1 level, 2 levels, and more than 2 levels of improvement in mobility, self-care, and usual activities domains of EQ-5D-5L - Week 25 up to Week 53
33. Percentage of subjects having no reduction in levels, at least 1 level, 2 levels, and more than 2 levels of improvement in mobility, self-care, and usual activities domains of EQ-5D-5L - Week 25 up to Week 53
34. Percentage of subjects with Treatment Emergent Adverse Events (TEAEs) - Up to 8 years
35. Percentage of subjects with related TEAEs - Up to 8 years
36. Percentage of subjects with serious TEAEs - Up to 8 years
37. Rate of TEAEs per days with infusion - Up to 8 years
38. Rate of TEAEs per days with infusion, by severity - Up to 8 years
39. Rate of related TEAEs per days with infusion - Up to 8 years
40. Rate of serious TEAEs per days with infusion - Up to 8 years

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

CSL Behring LLC

Sponsor organisation

CSL Behring LLC

Address

1020 1st Avenue

City

King Of Prussia

Postcode

19406-1310

Country

United States

Scientific contact point

Organisation

CSL Behring LLC

Contact name

Trial Registration Coordinator

Public contact point

Organisation

CSL Behring LLC

Contact name

Trial Registration Coordinator

Third parties 10

Locations

7 EU/EEA countries · 30 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

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 14 files

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

Application history

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