Netherton Syndrome 환자에서 Dupilumab과 위약의 효능 및 안전성에 대한 파일럿 연구 (NS-DUPI)
Netherton Syndrome 환자에서 Dupilumab과 위약의 효능 및 안전성에 대한 무작위 이중 맹검 파일럿 연구
To date, there are no effective therapy for the management of Netherton Syndrome (NS) Patients use emollients with a limited efficacy on scaling and no efficacy on skin inflammation and pruritus. They may also use topical corticosteroids or calcineurin inhibitors in case of eczematous lesions. The use of therapies targeting skin inflammation has been reported in a few case reports. Their efficacy is very limited and their uses are limited because of the chronicity of the disease, the impaired skin barrier function and the risk for skin infections and skin cancers. Therefore, there is a huge medical need for novel therapies in NS.The expected consequences of this study are that a 16-week course of dupilumab will be more effective than placebo for the treatment of moderate to severe NS Dupilumab could therefore improve skin condition and quality of life.
연구 개요
상세 설명
This is a proof of concept (pilot) double-blind randomized placebo-controlled study evaluating the efficacy and safety of dupilumab for the treatment of NS. Patients will be randomized in a 2:1 ratio to receive dupilumab (2 doses of 300 mg), or placebo, at baseline and then 1 dose of dupilumab 300 mg, or placebo, every 2 weeks until week 14 (total of 8 administrations).Moderate to severe NS were selected in order to be able to measure the improvement of skin condition.
연구 유형
등록 (실제)
단계
- 2 단계
- 3단계
연락처 및 위치
연구 장소
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Paris, 프랑스, 75015
- Dermatologie Necker
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Toulouse, 프랑스
- Dermatology
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
- Adult patients affiliated to a social insurance protection regimen.
- Clinical diagnosis of NS (7) (trichorrhexis invaginata, extensive scaling, skin inflammation, allergic manifestations) and absent or marked reduction of LEKTI staining.
- Moderate to severe forms: NASA (Netherton Area Severity Assessment score) score ≥ 5/12 at inclusion.
- Patients able to understand the study procedures including the ability to complete patient-based self-assessment questionnaires.
- Patients who agree to sign the written informed consent.
Exclusion Criteria:
- Hypersensitivity to dupilumab or its excipients.
- Modification of the usual treatment (emollients and topical corticosteroids used on a regular basis) within 2 weeks before inclusion.
- Treatment with topical calcineurin inhibitors 1 week before inclusion.
- Treatment with oral immunosuppressant (including cyclosporine, methotrexate, azathioprine, mycophenolate mofetil), oral retinoids (acitretin, alitretinoin, isotretinoin) or phototherapy within 4 weeks before inclusion.
- Treatment with immunomodulating biologics (Tumor Necrosis Factor (TNF) inhibitor) 16 weeks before inclusion.
- Treatment with another investigational drug within 8 weeks before inclusion.
- Treatment with a systemic antibiotic within 2 weeks before inclusion.
- Active skin infection requiring the use of a systemic therapy within 2 weeks before the inclusion.
- Any other condition that according to the investigator will impair the ability to evaluate treatment effect.
- Known or suspected history of immunosuppression, including history of invasive opportunistic infections (e.g., tuberculosis, histoplasmosis, listeriosis, coccidioidomycosis, pneumocystis, aspergillosis).
- Current infections including infection with helminthes.
- Pregnant or breastfeeding women, or women planning to become pregnant or breastfeed during the study. Women of childbearing age, potentially sexually active, and unwilling to use adequate birth control methods.
- Mental or physical incapacity to fill in the questionnaires.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 삼루타
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
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실험적: Dupilumab
The patient will receive 2 doses at baseline and then 1 dose every 2 weeks (8 administrations in total) of Dupilumab 300 mg (syringe of 2 mL for subcutaneous administration). |
administration of dupilumab corresponding to dupilumab arm |
|
위약 비교기: Placebo
The patient will receive 2 doses at baseline and then 1 dose every 2 weeks (8 administrations in total) of placebo (syringe of 2 mL for subcutaneous administration).. |
administration of placebo corresponding to placebo arm |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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The severity of the disease of the Netherton Area Severity Assessment score (NASA).
기간: Day 0 and week 16
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NASA score |
Day 0 and week 16
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Clinical efficacy severity of pruritus and pain
기간: Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
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NASA score |
Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
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Presence of infections (adverse event)
기간: Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
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Number of Bacterial or viral Skin infections |
Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
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Quantity of dermocorticosteroids used between each visit will be evaluated by questioning the patient.
기간: Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
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number of tubes multiplied by the weight of one tube |
Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
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QOL score
기간: Day 0, Week 16 and 28
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QOL score |
Day 0, Week 16 and 28
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Skin inflammation
기간: Day 0 and week 16
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number of inflammation markers on biopsies |
Day 0 and week 16
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Protease activity
기간: Day 0 and week 16
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number of protease markers on biopsies |
Day 0 and week 16
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Microbiome qualitative and quantitative analysis
기간: Day 0 and week 16
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number and form of bacteria |
Day 0 and week 16
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Transepidermal water loss (TEWL)
기간: Day 0 and Week 16
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Measured by a Tewameter applied on a standardized area in the anterior aspect of the forearm, |
Day 0 and Week 16
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Safety of dupilumab
기간: Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
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Blood tests performed every month until Week 16 (liver and renal tests, total blood count) |
Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
|
공동 작업자 및 조사자
협력자
수사관
- 수석 연구원: Juliette MAZEREEUW-HAUTIER, Toulouse Hospital
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (실제)
연구 완료 (실제)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- RC31/19/0045
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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