E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Subacute Cutaneous Lupus Erythematosus Chronic Cutaneous Lupus Erythematosus |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10056509 |
E.1.2 | Term | Cutaneous lupus erythematosus |
E.1.2 | System Organ Class | 10040785 - Skin and subcutaneous tissue disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10057903 |
E.1.2 | Term | Subacute cutaneous lupus erythematosus |
E.1.2 | System Organ Class | 10040785 - Skin and subcutaneous tissue disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10057929 |
E.1.2 | Term | Chronic cutaneous lupus erythematosus |
E.1.2 | System Organ Class | 10040785 - Skin and subcutaneous tissue disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objectives of the study are to evaluate the efficacy of BIIB059 compared with placebo in reducing skin disease activity measured by the Cutaneous Lupus Activity of Physician's Global Assessment-Revised (CLA-IGA-R) score [Parts A and B (US)] and the Cutaneous Lupus Erythematosus Disease Area and Severity Index Activity (CLASI-A) score [Part B (ROW)] in participants with active SCLE and/or CCLE with or without systemic manifestations and refractory and/or intolerant to antimalarials. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of the study are to evaluate the efficacy of BIIB059 in reducing SCLE and/or CCLE disease activity by CLA-IGA-R, CLASI-A; to evaluate additional efficacy parameters of BIIB059 in reducing SCLE and/or CCLE disease activity; safety; tolerability; and immunogenicity of BIIB059 [Parts A and B]. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Key Inclusion Criteria: 1. Histologically confirmed (in the past or during the Screening period) diagnosis of CLE with or without systemic manifestations. 2. Must have active cutaneous manifestations that meet study criteria. 3. Must have a CLASI-A score ≥10. 4. Must have an active CLE lesion despite an adequate trial of antimalarial treatment. NOTE: Other protocol defined Inclusion criteria may apply. |
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E.4 | Principal exclusion criteria |
Key Exclusion Criteria: 1. Any active skin conditions other than CLE that may interfere with the study assessments of CLE. 2. Active severe lupus nephritis. 3. Active neuropsychiatric SLE. 4. Use of intralesional corticosteroids within 1 week prior to Screening and during the study. 5. Use of immunosuppressive or disease-modifying treatments for SLE or CLE [via an oral, intravenous (IV), or SC route] that were initiated less than 12 weeks prior to randomization, have not been at a stable and allowable dose.
NOTE: Other protocol defined Exclusion criteria may apply. |
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E.5 End points |
E.5.1 | Primary end point(s) |
*Parts A (US+ROW) and B (US): Percentage of Participants who Achieve a Cutaneous Lupus Activity of Physician’s Global Assessment-Revised (CLA-IGA-R) Score of 0 or 1 *Part B (ROW): Percentage of Participants who Achieve Cutaneous Lupus Erythematosus Disease Area and Severity Index Activity Score (CLASI-70) Response Response, Defined as ≥ 70% Decrease in CLASI-A Score From Baseline |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Parts A (US+ROW) and B (US): Week 16 Part B (ROW): Baseline to Week 24 |
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E.5.2 | Secondary end point(s) |
*Parts A(US+ROW)&B(US): % of Participants who Achieve a CLA-IGA-R Erythema Score of 0 or 1 *Part B(ROW): % of Participants who Achieve CLASI-70 Response, Defined as ≥ 70% Decrease in CLASI-A Score From Baseline *Part A(US+ROW): % of Participants who Achieve a CLASI-50 Response, Defined as a ≥ 50% Decrease in CLASI-A Score From Baseline *Part A(US+ROW): Change From Baseline in CLASI-D Score *Part B (US+ROW): % of Participants who Achieve a CLA-IGA-R Erythema Score of 0 or 1, at W16&W24, Respectively, for Participants in Full Analysis Set (FAS), who had CLA-IGA-R Erythema Score ≥3 and OMC Score ≥3 at Baseline *Part B(US+ROW): % of Participants who Achieve a CLA-IGA-R OMC Score of 0 or 1, at W16&W24, Respectively, for Participants in FAS, who had CLA-IGA-R Erythema Score ≥3 and OMC Score ≥3 at Baseline *Part B(US+ROW): % of Participants who Achieve at Least 1 Level of Improvement From Baseline in the CLA-IGA-R Erythema Score *Part B(US+ROW): Absolute Change in CLASI-D Score *Part B(US+ROW): Percent Change in CLASI-D Score *Part B(US+ROW): Change From Baseline in CLE-QoL Score *Part B(US+ROW): Change From Baseline in DLQI Score *Parts A(US+ROW)&B(US): % of Participants who Achieve a CLASI-70 Response, Defined as a ≥ 70% Decrease in CLASI-A Score From Baseline *Parts A&B(US+ROW): % of Participants who Achieve a CLA-IGA-R Erythema Score of 0 or 1 *Parts A&B(US+ROW): % of Participants who Achieve a CLA-IGA-R OMC Score of 0 or 1 and at Least 1 Level of Improvement From Baseline *Parts A&B(US+ROW): % of Participants who Achieve a CLA-IGA-R OMC Score of 0 *Parts A&B(US+ROW): % of Participants With at Least 1 Level of Improvement From Baseline in CLA-IGA-R OMC Score *Parts A&B(US+ROW): % of Participants who Achieve a CLA-IGA-R Follicular Activity Score of 0 *Parts A&B(US+ROW): % of Participants who Achieve a CLASI-A Score of 0 or 1 *Parts A&B(US+ROW): % of Participants who Achieve a CLASI-A Score of 0 or 1 *Parts A&B(US+ROW): % of Participants who Achieve a CLASI-A Score of 0 to 3 *Parts A&B(US+ROW): % of Participants who Achieve a 70% Reduction in CLASI-A *Parts A&B(US+ROW): % of Participants who Achieve a 50% Reduction in CLASI-A *Parts A&B(US+ROW): % of Participants who Achieve a 7-Point Reduction From Baseline in CLASI-A Score *Parts A&B(US+ROW): % of Participants With a CLASI-70 Response at W52 Among CLASI-70 Responders at W16 and W24, Respectively, who Were Randomly Assigned to Receive BIIB059 During the DBPC TP *Parts A&B(US+ROW): % of Participants With CLA-IGA-R Erythema Score of 0 or 1 at W52 Among CLA-IGA-R Erythema Responders at W16&W24, Respectively, who Were Randomly Assigned to Receive BIIB059 During the DBPC TP *Parts A&B(US+ROW): % of Participants With CLA-IGA-R OMC Score of 0/1 and at Least 1 Level of Improvement From Baseline at W52 Among CLA-IGA-R OMC Responders at W16&24, Respectively, who Were Assigned to Receive BIIB059 in DBPC TP *Parts A&B(US+ROW): % of Participants With CLA-IGA-R OMC Score of 0 at W52 Among Responders With CLA-IGA-R OMC Score of 0 at W16&W24, Respectively, who Were Randomly Assigned to Receive BIIB059 During the DBPC TP *Parts A&B(US+ROW): % of Participants With CLA-IGA-R Follicular Activity Score of 0 at W52 Among CLA-IGA-R Follicular Activity Responders at W16&W24, Respectively, who Were Randomly Assigned to Receive BIIB059 in DBPC TP *Parts A&B(US+ROW): % of Participants With a CLASI-70 Response at W52 Among CLASI-70 Nonresponders at W16&W24, Respectively, who Were Randomly Assigned to Receive Placebo During the DBPC TP *Parts A&B(US+ROW): % of Participants With a CLA-IGA-R Erythema Score of 0 or 1 at W52 Among CLA-IGA-R Erythema Nonresponders at W16&W24, Respectively, who Were Randomly Assigned to Receive Placebo During the DBPC TP *Parts A,B(US+ROW): % of Participants With CLA-IGA-R OMC Score of 0/1 and at Least 1 Level of Improvement From Baseline at W52 Among CLA-IGA-R OMC Nonresponders at Wks16&24, Respectively, who Were Assigned to Receive Placebo in DBPC TP *Parts A&B(US+ROW): % of Participants With a CLA-IGA-R OMC Score of 0 at W52 Among CLA-IGA-R OMC Nonresponders at W16&W24, Respectively, who Were Randomly Assigned to Receive Placebo During the DBPC TP *Parts A&B(US+ROW): % of Participants who Achieve CLA-IGA-R Follicular Activity Score of 0 at W52 Among CLA-IGA-R Follicular Activity Nonresponders at Wks16&24, Respectively, who Were Randomly Assigned to Receive Placebo in DBPC TP *Parts A&B(US+ROW): Annualized Mild and Moderate Safety of Estrogens in LE National Assessment-SLE Disease Activity Index Flare Index (SFI) Rate and Annualized Severe SFI Rate Through W24 *Parts A&B(US+ROW): Annualized Mild and Moderate SFI Rate and Annualized Severe SFI Rate Through W16 *Parts A&B(US+ROW): Annualized Mild and Moderate SFI Rate and Annualized Severe SFI Rate Through W52 *Parts A&B(US+ROW): Number of Participants Experiencing TEAEs and SAEs *Parts A&B(US+ROW): Number of Participants With Anti-BIIB059 Antibodies in Serum During the Study |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Up to Week 16, Week 16, Up to Week 24, Week 24, Up to Week 52, Week 52, Baseline up to Week 52, Up to Week 76 |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
immunogenicity and tolerability |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
A 2-part seamless design study |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 4 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 7 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 83 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Argentina |
Chile |
Colombia |
Peru |
Philippines |
Switzerland |
Puerto Rico |
Taiwan |
Brazil |
Canada |
China |
Japan |
Korea, Republic of |
Mexico |
Saudi Arabia |
Serbia |
United Kingdom |
United States |
Belgium |
Bulgaria |
France |
Germany |
Hungary |
Italy |
Poland |
Portugal |
Slovakia |
Spain |
Sweden |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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The end of the study period (i.e., DBPC followed by ETP) is Week 52/EOS. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | 29 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 2 |
E.8.9.2 | In all countries concerned by the trial days | 29 |