Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis

• Sponsor: Alexion Pharmaceuticals (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04999020
• Collaborator: (none)
• Enrollment: 180
• Locations: 36
• Arms: 2
• Anticipated Duration (Months): 33
• Patients Per Site: 5
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

This is a Phase 2/3, double-blind, randomized, placebo-controlled, parallel group, multicenter study to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ravulizumab in adult participants with dermatomyositis (DM).

Condition or Disease	Intervention/Treatment	Phase
Dermatomyositis	Drug: Ravulizumab; Drug: Placebo	Phase 2/Phase 3

Detailed Description

The study will be conducted in 2 parts: Part A (Phase 2) and Part B (Phase 3). There will be 3 periods in both Part A and Part B of this study: Screening Period, Randomized Controlled Period, and Open-Label Extension Period.

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 180 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants With Dermatomyositis
• Actual Study Start Date: Dec 2, 2021
• Anticipated Primary Completion Date: Aug 31, 2022
• Anticipated Study Completion Date: Aug 31, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Ravulizumab Participants will receive ravulizumab in both Parts A and B.	Drug: Ravulizumab; Intravenous dosing will consist of a loading dose followed by maintenance doses administered every 8 weeks (q8w). The maintenance dosing will be initiated 2 weeks after the loading dose is administered.; Other Names: ALXN1210 Ultomiris
Placebo Comparator: Placebo Participants will receive placebo in both Parts A and B.	Drug: Placebo; Intravenous dosing will consist of a loading dose followed by maintenance doses administered q8w. The maintenance dosing will be initiated 2 weeks after the loading dose is administered.

Outcome Measures

Primary Outcome Measures

1. Part A: Proportion Of Participants With A ≥ 20-point Improvement Response On International Myositis Assessment And Clinical Studies-Total Improvement Score (IMACS-TIS) (TIS20) At Week 26 Of The Randomized Controlled Period [Week 26]

2. Part B: Proportion Of Participants With A ≥ 20-point Improvement Response On TIS20 At Week 50 [Week 50]

Secondary Outcome Measures

1. Part A: Mean TIS At Week 26 [Week 26]

2. Part A: Mean Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score At Week 26 [Baseline, Week 26]

3. Part A: Change From Baseline Of Each Of The IMACS Core Set Measures At Week 26 [Baseline, Week 26]

4. Part A: Time To First CDASI Activity Score Improvement [Baseline through Week 26]

   Minimally clinically important differences (MCID) = 7-point improvement.

5. Part A: Proportion Of Participants With CDASI MCID Improvement At Week 26 [Week 26]

6. Part A: Change In Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) At Week 26 [Baseline, Week 26]

7. Part A: Proportion Of Participants With TIS20 At Each Visit [Baseline through Week 26]

8. Part A: Proportion Of Participants With A ≥ 40-point Improvement Response On IMACS-TIS (TIS40) At Each Visit [Baseline through Week 26]

9. Part A: Proportion Of Participants With A ≥ 60-point Improvement Response On IMACS-TIS (TIS60) At Each Visit [Baseline through Week 26]

10. Part A: Time To First Response Of TIS20, TIS40, Or TIS60 [Baseline through Week 26]

11. Part A: Time To First IMACS Myositis Core Set Measure Improvements [Baseline through Week 26]

12. Part B: Mean TIS At Week 50 [Week 50]

13. Part B: Mean Change From Baseline In Manual Muscle Testing Subset Of 8 Muscles (MMT-8) At Week 50 [Baseline, Week 50]

14. Part B: Mean Change From Baseline In Extra-muscular Disease Activity Based On Myositis Disease Activity Assessment Tool (MDAAT) At Week 50 [Baseline, Week 50]

15. Part B: Mean Change From Baseline In CDASI Activity Score At Week 50 [Baseline, Week 50]

16. Part B: Mean Change From Baseline In Patient Global Activity Assessment At Week 50 [Baseline, Week 50]

17. Part B: Mean Change From Baseline In Physician Global Activity Assessment At Week 50 [Baseline, Week 50]

18. Part B: Mean Change From Baseline In Health Assessment Questionnaire (HAQ) At Week 50 [Baseline, Week 50]

19. Part B: Mean Change From Baseline In Muscle Enzyme Values At Week 50 [Baseline, Week 50]

20. Part B: Mean TIS At Each Visit From Week 2 Through Week 50 [Week 2 through Week 50]

21. Part B: Proportion Of Participants With TIS20 At Each Visit [Baseline through Week 50]

22. Part B: Proportion Of Participants With TIS40 At Each Visit [Baseline through Week 50]

23. Part B: Proportion Of Participants With TIS60 At Each Visit [Baseline through Week 50]

24. Part B: Time To First Response Of TIS20, TIS40, Or TIS60 [Baseline through Week 50]

25. Part B: Time To First IMACS Myositis Core Set Measure Improvements [Baseline through Week 50]

26. Part B: Time To First CDASI Activity Score Improvement [Baseline through Week 50]

    MCID = 7-point improvement

27. Part B: Proportion Of Participants With CDASI MCID Improvement At Week 50 [Week 50]

28. Part B: Change In CDA-IGA At Week 50 [Week 50]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Key Inclusion Criteria:

• 18 years of age or older at the time of signing the informed consent.

• Body weight ≥ 30 kilograms at the time of Screening.

• Male or female.

• Diagnosis: Meet American College of Rheumatology/European League Against Rheumatism classification criteria for definite or probable DM.

• Participants who have an inadequate response (that is, continued impairment by medical doctor report) or are intolerant to 2 or more DM treatments, including systemic corticosteroids or immunosuppressive/immunomodulatory therapies (for example, azathioprine, methotrexate, rituximab, intravenous immunoglobulin), either in combination or as monotherapy.

• Vaccinated against Neisseria meningitidis within 3 years prior to initiating ravulizumab as per national and local guidelines. Participants must receive the vaccination at least 2 weeks before first study intervention. The sponsor recommends that national and local guidelines for prophylactic antibiotics should also be followed.

• Female participants of childbearing potential and male participants must follow specified contraception guidance as described in the protocol.

Key Exclusion Criteria:

• Cancer-associated myositis, defined as the diagnosis of myositis within 3 years of the diagnosis of cancer (except basal or squamous cell skin cancer or carcinoma in situ of the cervix that has been excised and cured and at least 3 months before Screening).

• Evidence of active malignant disease or malignancies diagnosed within the previous 3 years including hematological malignancies and solid tumors.

• Participants with other forms of myositis.

• Participants with significant muscle damage (for example, severe muscle atrophy, end stage muscle disease) as per investigator opinion.

• History of Neisseria meningitidis infection.

• Human immunodeficiency virus (HIV) infection (evidenced by HIV Type 1 or Type 2 antibody titer).

• Active systemic bacterial, viral, or fungal infection within 14 days prior to ravulizumab administration.

• Presence of fever ≥ 38°Celsius (100.4°Fahrenheit) within 7 days prior to study drug administration on Day 1.

• History of hypersensitivity to murine proteins or to 1 of the excipients of ravulizumab.

• Pregnant, breastfeeding, or intending to conceive during the course of the study.

• Inability or unwillingness to adhere to the protocol requirements.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Clinical Trial Site	Orange	California	United States	92868
2	Clinical Trial Site	Tampa	Florida	United States	33612
3	Clinical Trial Site	Kansas City	Kansas	United States	66160
4	Clinical Trial Site	Baltimore	Maryland	United States	21224
5	Clinical Trial Site	New York	New York	United States	10021
6	Clinical Trial Site	Cleveland	Ohio	United States	44195
7	Clinical Trial Site	Pittsburgh	Pennsylvania	United States	15213
8	Clinical Trial Site	Lille Cedex		France	59037
9	Clinical Trial Site	Paris		France	75010
10	Clinical Trial Site	Strasbourg		France	67098
11	Clinical Trial Site	Toulouse Cedex 9		France	31059
12	Clinical Trial Site	Dusseldorf		Germany	40225
13	Clinical Trial Site	Erlangen		Germany	91054
14	Clinical Trial Site	Essen		Germany	45147
15	Clinical Trial Site	Freiburg		Germany	79106
16	Clinical Trial Site	Gottingen		Germany	37075
17	Clinical Trial Site	Bari	BA	Italy	70124
18	Clinical Trial Site	Brescia	BS	Italy	25123
19	Clinical Trial Site	Pisa	PI	Italy	56126
20	Clinical Trial Site	Pavia	PV	Italy	27100
21	Clinical Trial Site	Roma		Italy	00168
22	Clinical Trial Site	Bunkyo-ku	Tokyo	Japan	113-8655
23	Clinical Trial Site	Iruma-Gun		Japan	350-0495
24	Clinical Trial Site	Suita-Shi		Japan	5650871
25	Clinical Trial Site	Incheon		Korea, Republic of	22332
26	Clinical Trial Site	Seoul		Korea, Republic of	03080
27	Clinical Trial Site	Seoul		Korea, Republic of	04763
28	Clinical Trial Site	Seoul		Korea, Republic of	06591
29	Clinical Trial Site	L'Hospitalet de Llobregat	Barcelona	Spain	8907
30	Clinical Trial Site	Bilbao	Vizcaya	Spain	48013
31	Clinical Trial Site	Barcelona		Spain	08035
32	Clinical Trial Site	Barcelona		Spain	08036
33	Clinical Trial Site	Madrid		Spain	28034
34	Clinical Trial Site	Madrid		Spain	28041
35	Clinical Trial Site	Liverpool		United Kingdom	L9 7AL
36	Clinical Trial Site	Salford		United Kingdom	M55AP

Sponsors and Collaborators

• Alexion Pharmaceuticals

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Alexion Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT04999020
• Other Study ID Numbers: ALXN1210-DM-310; 2021-001200-15
• First Posted: Aug 10, 2021
• Last Update Posted: Aug 18, 2022
• Last Verified: Aug 1, 2022

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Alexion Pharmaceuticals: Ravulizumab; ALXN1210; Ultomiris; Pharmacokinetics; Pharmacodynamics; Efficacy; DM
• Additional relevant MeSH terms: Dermatomyositis; Ravulizumab

Study Results

No Results Posted as of Aug 18, 2022