A Long-Term Extension Study to Learn More About the Safety of Litifilimab (BIIB059) Injections and Whether They Can Improve Symptoms of Adult Participants Who Have Active Cutaneous Lupus Erythematosus (CLE) (AMETHYST LTE)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Biogen Idec Research Limited

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

21 Feb 2024 → ongoing

Decision date (initial)

2026-01-23

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Biogen Idec Research Limited

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Others, Pharmacodynamic, Pharmacokinetic

The primary objective of the study is to evaluate the long-term safety and tolerability BIIB059 (litifilimab) in participants who completed the parent study 230LE301 (NCT05531565) with active subacute CLE and/or chronic CLE with or without systemic manifestations and refractory and/or intolerant to antimalarial therapy.

Secondary objectives 8

1. To evaluate the long-term effect of litifilimab on disease activity in participants with active subacute CLE and/or chronic CLE with or without systemic manifestations and refractory and/or intolerant to antimalarials.
2. To evaluate the effect of litifilimab in preventing disease damage in participants with active subacute CLE and/or chronic CLE with or without systemic manifestations and refractory and/or intolerant to antimalarials
3. To evaluate the long-term effect of litifilimab on preventing lupus flare in participants with CLE with SLE
4. To assess long-term use of oral corticosteroid (OCS) in participants receiving litifilimab treatment
5. To assess the impact of litifilimab on participant-reported health-related quality of life (HRQoL)
6. To evaluate long-term effect of litifilimab on laboratory parameters
7. To evaluate the immunogenicity of litifilimab
8. To evaluate the PK of litifilimab

Conditions and MedDRA coding

1. Lupus Erythematosus, Cutaneous, Subacute 2. Chronic Cutaneous Lupus Erythematosus

Study design 2 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Participants who completed the parent study (230LE301, Part A or Part B) on study treatment (received treatment through Week 48 and attended the last study assessment visit at Week 52)
2. Ability of the participant to understand the purpose and risks of the study, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations

Exclusion criteria 4

1. Early Part A or Part B parent study (230LE301) treatment terminators (participants who discontinued study treatment before Week 48)
2. Early Part A or Part B parent study terminators [participants who withdrew from parent study participation before Week 52 and did not complete the parent study extended treatment period (ETP)]
3. Participants who have developed any other medical diseases, conditions, or abnormalities, rendering their participation in the long-term extension (LTE) study unsuitable in the opinion of the Investigator.
4. Other protocol defined Exclusion criteria may apply

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [Time Frame: Up to 128 weeks]

Secondary endpoints 41

1. Percentage of Participants who Achieve a Cutaneous Lupus Erythematosus Disease Area and Severity Index Activity Score (CLASI)-70 Response, Defined as a 70% Improvement in CLASI-A Score from the Baseline Value (Parent Study) [Time Frame: Up to 128 weeks]
2. Percentage of Participants who Achieve a CLASI-50 Response, Defined as a 50% Improvement in CLASI-A Score from the Baseline Value (Parent Study) [Time Frame: Up to 128 weeks]
3. Percentage of Participants who Achieve a CLASI-90 Response, Defined as a 90% Improvement in CLASI-A Score from the Baseline Value (Parent Study) [Time Frame: Up to 128 weeks]
4. Percentage of Participants who Achieve a Cutaneous Lupus Activity of Physician's Global Assessment-Revised (CLA-IGA-R) Erythema Score of 0 or 1 [Time Frame: Up to 128 weeks]
5. Percentage of Participants who Achieve a CLA-IGA-R Other Morphologic Characteristics (OMC) Score of 0 or 1 [Time Frame: Up to 128 weeks]
6. Cumulative Duration of Sustained CLASI-70 Response, Defined as the Number of Weeks With 70%Improvement in CLASI-A Score from the Baseline Value (Parent Study) [Time Frame: Up to 128 weeks]
7. Cumulative Duration of Sustained CLASI-50 Response, Defined as the Number of Weeks With 50%Improvement in CLASI-A Score from the Baseline Value (Parent Study) [Time Frame: Up to 128 weeks]
8. Cumulative Duration of Sustained CLASI-90 Response, Defined as the Number of Weeks With 90%Improvement in CLASI-A Score From the Baseline Value (Parent Study) [Time Frame: Up to 128 weeks]
9. Cumulative Duration of Sustained Efficacy, Defined as the Number of Weeks With CLA-IGA-R Erythema Score of 0 or 1 [Time Frame: Up to 128 weeks]
10. Cumulative Duration of Sustained Efficacy, Defined as the Number of Weeks With CLA-IGA-R OMC Score of 0 or 1 and Improvement of at Least 1 Point From Baseline Value (Parent Study) [Time Frame: Up to 128 weeks]
11. Cumulative Duration of Sustained Efficacy, Defined as the Number of Weeks With CLA-IGA-R Follicular Activity Score of 0 [Time Frame: Up to 128 weeks]
12. Percentage of Participants With a CLASI-70 Response Among CLASI-70 Responders at Week 52 of the Parent Study [Time Frame: Day 0 (Week 52 of parent study) up to 128 weeks]
13. Percentage of Participants With a CLASI-50 Response Among CLASI-50 Responders at Week 52 of the Parent Study [Time Frame: Day 0 (Week 52 of parent study) up to 128 weeks]
14. Percentage of Participants With a CLASI-90 Response Among CLASI-90 Responders at Week 52 of the Parent Study [Time Frame: Day 0 (Week 52 of parent study) up to 128 weeks]
15. Percentage of Participants With a CLA-IGA-R Erythema Score of 0 or 1 Among Participants With aCLA-IGA-R Erythema Score of 0 or 1 at Week 52 of the Parent Study [Time Frame: Day 0 (Week 52 of parent study) up to 128 weeks]
16. Percentage of Participants With a CLA-IGA-R OMC Score of 0 or 1 and at Least 1 Level of Improvement From Baseline Value (Parent Study) Among Participants With a CLA IGA R OMC Score of 0 or 1 and at Least 1 Level of Improvement From Baseline Value (Parent Study) [Time Frame: Day 0 (Week 52 of parent study) up to 128 weeks]
17. Percentage of Participants With a CLASI-70 Response Among CLASI-50 Responders at Week 52 of the Parent Study [Time Frame: Day 0 (Week 52 of parent study) up to 128 weeks]
18. Percentage of Participants With a CLASI-90 Response Among CLASI-50 Responders at Week 52 of the Parent Study [Time Frame: Day 0 (Week 52 of parent study) up to 128 weeks]
19. Percentage of Participants With a CLASI-90 Response Among CLASI-70 Responders at Week 52 of the Parent Study [Time Frame: Day 0 (Week 52 of parent study) up to 128 weeks]
20. Percentage of Participants With Loss of Response, Defined as an Increase of ≥ 7 Points in CLASI-A Total Score From Baseline [Time Frame: Baseline (Day 0) up to 128 weeks]
21. Percentage of Participants With Loss of Response, Defined as Achieving 2 Points Improvement From Baseline Value (Parent Study) CLA-IGA-R Erythema Score at the Beginning of/ During the LTE Study and Then Relapsing to the CLA-IGA-R Erythema Baseline Value (Parent Study) [Time Frame: Up to 128 weeks]
22. Percentage of Participants With Loss of Response, Defined as Having at Least 2, 3, and 4 Points Worsening in CLA-IGA-R Erythema Score From Their Minimum Score in Parent Study [Time Frame: Up to 128 weeks]
23. Absolute Change in Cutaneous Lupus Erythematosus Disease Area and Severity Index Damage (CLASI-D) Score From the Baseline (Parent Study) to Week 104 [Time Frame: Up to 104 weeks]
24. Percent Change in CLASI-D Score From the Baseline (Parent Study) to Week 104 [Time Frame: Up to 104 weeks]
25. Annualized Mild/Moderate and Severe Safety of Estrogens in Lupus Erythematosus National Assessment-SLE Disease Activity Index Flare Index (SFI) Rates Through Week 52 [Time Frame: Up to 52 weeks]
26. Annualized Mild/Moderate and Severe SFI Rates Through Week 104 [Time Frame: Up to 104 weeks]
27. Percentage of Participants With Oral Corticosteroid (OCS) Dose [Time Frame: Up to 104 weeks]
28. Percentage of Participants With OCS ≤ 7.5 Milligrams per day (mg/day) [Time Frame: Up to 104 weeks]
29. Percentage of Participants With OCS ≤ 5.0 mg/day [Time Frame: Up to 104 weeks]
30. Change From Baseline Value (Parent Study) in Cutaneous Lupus Erythematosus –Quality of Life (CLE-QoL) at Weeks 52 and 104 [Time Frame: Baseline, Weeks 52 and 104]
31. Change From Baseline Value (Parent Study) in European Quality of Life – 5-Dimensions Questionnaire, 3-Level Version (EQ-5D-3L) at Weeks 52 and 104 [Time Frame: Baseline, Weeks 52 and 104]
32. Change From Baseline Value (Parent Study) in 36-Item Short Form Survey (SF-36)(acute version) at Weeks 52 and 104 [Time Frame: Baseline, Weeks 52 and 104]
33. Change From Baseline Value (Parent Study) in Work Productivity and Activity Impairment (WPAI): Lupus at Weeks 52 and 104 [Time Frame: Baseline, Weeks 52 and 104]
34. Change From Baseline Value (Parent Study) in Patient Health Questionnaire-9(PHQ-9) at Weeks 52 and 104 [Time Frame: Baseline, Weeks 52 and 104]
35. Number of Participants With Clinically Relevant Change From Baseline Value (Parent Study) in Standard Laboratory Parameters [Time Frame: Up to 128 weeks]
36. Number of Participants With Clinically Relevant Change From Baseline Value (Parent Study) in ECG Results [Time Frame: Up to 104 weeks]
37. Number of Participants With Anti-BIIB059 (litifilimab) Antibodies in Serum [Time Frame: Up to 128 weeks]
38. Serum Concentration of Litifilimab [Time Frame: Pre-dose up to 128 weeks]
39. Change From Baseline Value (Parent Study) in Subject Global Assessment of Skin - Follow-up (SGA-Skin-FU) at Weeks 52 and 104 [Time Frame: Baseline, Weeks 52 and 104]
40. Change From Baseline Value (Parent Study) in Numerical Rating Scale (NRS) for Pain in Skin Rash at Weeks 52 and 104 [Time Frame: Baseline, Weeks 52 and 104]
41. Change From Baseline Value (Parent Study) in Numerical Rating Scale (NRS) for Itch in Skin Rash at Weeks 52 and 104 [Time Frame: Baseline, Weeks 52 and 104]

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Biogen Idec Research Limited

Sponsor organisation

Biogen Idec Research Limited

Address

Building 5 Foundation Park, Roxborough Way Roxborough Way

City

Maidenhead

Postcode

SL6 3UD

Country

United Kingdom

Scientific contact point

Organisation

Biogen Idec Research Limited

Contact name

Clinical Trials Information Desk

Public contact point

Organisation

Biogen Idec Research Limited

Contact name

Clinical Trials Information Desk

Third parties 13

Locations

11 EU/EEA countries · 62 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 188 files

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

Application history

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