A Study of Quinacrine in Participants With Cutaneous Lupus Erythematosus
A Randomized, Double-blind, Placebo-controlled Study of Quinacrine (QC) in Participants With Active Cutaneous Lupus Erythematosus (CLE), Including Subacute CLE (SCLE) and/or Discoid LE (DLE), With or Without Concurrent Systemic Manifestations
The research study is being conducted to learn more about how patients with cutaneous lupus erythematosus (CLE) respond to the use of quinacrine. Quinacrine is a medication that was originally developed and used starting in the 1930's to treat malaria. It has been used for decades to help reduce inflammation in the body. Doctors have observed that quinacrine may also help improve skin symptoms in patients with CLE, a condition in which the immune system mistakenly attacks the skin, causing rashes, sores, and other skin problems. Although some doctors have prescribed quinacrine for CLE based on these observations, it has not yet been formally approved by the U.S. Food and Drug Administration (FDA) for this use. The purpose of this clinical trial is to carefully study how safe and effective quinacrine is for treating CLE, as well as how it works in the body. This can help researchers better understand whether it should become a standard treatment option.
Participation will last for about 28 weeks in total. This study is a randomized, double-blind, placebo-controlled study. "Placebo-controlled" means that participants may receive quinacrine or participants may receive a placebo for the first 12 weeks of the study. A placebo looks like the study drug but contains no active medication. It is used to help find out if the results of the study are due to the study drug or due to something else.
Randomized means participants will be put into the study drug group or the placebo group by chance. Participants have a 1:1 chance of receiving the study drug. This means for every 2 people in the study, 1 will receive the study drug and 1 will receive the placebo. Double-blind means that neither participants nor the study team will know which study group participants have been put in. For the next 12 weeks of the study (Week 12-Week 28), all participants will receive the study drug.
調査の概要
研究の種類
入学 (推定)
段階
- フェーズ2
連絡先と場所
研究連絡先
- 名前:Victoria P Werth, MD
- 電話番号:2156152940
- メール:penndermautoimmune@pennmedicine.upenn.edu
研究場所
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Pennsylvania
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Philadelphia、Pennsylvania、アメリカ、19104
- 募集
- Hospital of the University of Pennsylvania, Department of Dermatology
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コンタクト:
- Victoria P Werth, MD
- 電話番号:215-615-2940
- メール:werth@pennmedicine.upenn.edu
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コンタクト:
- Robert Haskins
- 電話番号:215-615-2940
- メール:Robert.haskins@pennmedicine.upenn.edu
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参加基準
適格基準
就学可能な年齢
- 大人
- 高齢者
健康ボランティアの受け入れ
説明
Inclusion Criteria:
- ≥ 18 years of age at the time of signing the informed consent form.
- Willing and capable of giving written informed consent, which includes being able to comply with all aspects of the study treatment and assessments schedule, including the ability to receive or self-administer study treatment at home or outside of the study site clinic.
Must have diagnosis of SCLE or DLE that has been histologically confirmed (in the past or at Screening), with or without systemic LE manifestations. For participants without historical biopsy data, a skin biopsy must be performed at Screening to confirm CLE diagnosis prior to randomization.
All participants must also have active skin manifestations that fulfill the following:
- CLASI-A ≥8 at Screening and randomization
- Must have active CLE despite an adequate trial of conventional therapies (defined topical corticosteroids and HCQ used for at least 12 weeks prior to Screening) OR previously documented failure to respond to these agents when used for at least 12 weeks OR the requirement to discontinue these agents due to side effects or poor tolerability.
- If patients are using HCQ during screening, the same dose should be continued until the end of the study.
Exclusion Criteria:
- Have any medical condition or laboratory abnormality during the Screening Period that, in the opinion of the Investigator, is clinically significant and could interfere with the participant's ability to be included in the study.
- Have undergone phlebotomy with removal of ≥ 500 mL of blood within 30 days prior to the Screening Visit.
- Have received a transfusion of any blood or blood products within 60 days or donated plasma within 7 days prior to the Screening Visit.
- Have participated in any other study involving an investigational product within the last 30 days or 5 half-lives, whichever is greater, prior to the Screening Visit.
- Subjects receiving treatment with primaquine or any concomitant medication that is a substrate of CYP2D6 at screening or during the study.
- Subjects receiving concomitant medications that are classified as moderate or strong inhibitors of CYP3A4/5 at screening or during the study.
Have any of the following laboratory abnormalities at the Screening Visit (as per the central laboratory)
- Aspartate aminotransferase (AST) ≥ 1.5 x~ upper limit of normal (ULN).
- Alanine aminotransferase (ALT) ≥ 1.5 x~ ULN.
- Total bilirubin ≥ 1.5 ULN. Note: A participant with elevated fasting unconjugated serum bilirubin with documented Gilbert syndrome may be enrolled at the Investigator's discretion.
- Subjects with eGFR < 45 at the time of screening..
- Subjects with G6PD deficiency defined as <30% of normal enzyme activity at the time of screening.
- Have had a cardiovascular event (e.g., acute myocardial infarction, stroke) or revascularization procedure (e.g., percutaneous coronary intervention, coronary artery bypass graft) within 6 months of the Screening Visit.
- Have evidence of prolonged QT (QTcF > 450 msec for males and > 470 msec for females) on electrocardiogram (ECG) at the Screening Visit.
- Have a recent serious infection requiring injectable antimicrobial therapy or hospitalization within the 4 weeks prior to Screening Visit or any ongoing febrile illness or infection requiring oral antimicrobial therapy within 1 week of the Screening Visit.
- Have had any surgical procedure (except for minor procedures) within 4 weeks prior to the Screening Visit.
- Have known active nephritis or neuropsychiatric SLE.
- Have current inflammatory skin disease other than SCLE/DLE that, in the opinion of the Investigator, could interfere with the inflammatory skin assessments or confound the disease activity assessments.
- Use of immunosuppressive or disease-modifying treatments for SLE that were initiated less than 3 months prior to Screening, have not been at a stable dose for at least 1 month prior to Screening.
Have received/used any of the following prior medications or undergone any of the following therapeutic procedures:
- Use of high-potency topical corticosteroid and/or topical agents (immunosuppressant) for skin lesions within 7 days prior to randomization.
- Use of high-potency intralesional corticosteroid within 4 weeks prior to randomization.
- JAK or TYK2 inhibitors within 1 month before the visit 1.
- IFN1/IFN1R inhibitors within 3 months before the Screening visit.
研究計画
研究はどのように設計されていますか?
デザインの詳細
- 主な目的:処理
- 割り当て:ランダム化
- 介入モデル:並列代入
- マスキング:4倍
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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実験的:Quinacrine (Experimental)
Quinacrine 100mg Daily for 12 Weeks
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Quinacrine Hydrochloride 100mg Daily
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プラセボコンパレーター:Placebo
Placebo daily for 12 weeks
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一致したプラセボ
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実験的:Open Label Extension
Quinacrine 100 mg daily for 12 weeks
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Quinacrine Hydrochloride 100mg Daily
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この研究は何を測定していますか?
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Change in Cutaneous Lupus Erythematosus Disease Area and Severity Index - Activity (CLASI-A) score
時間枠:12 weeks
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Differences in the change in score from baseline to Week 12 between the Quinacrine arm versus placebo arm.
Scores range from 0 to 70 with higher scores representing more severe, active disease.
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12 weeks
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Binary responder endpoints based on Cutaneous Lupus Erythematosus Disease Area and Severity Index - Activity (CLASI-A) improvement
時間枠:24 Weeks
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24 Weeks
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Low Cutaneous Lupus Activity - Investigator's Global Assessment (CLA-IGA) scores
時間枠:24 weeks
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Low activity defined as scores of 0-1 on a scale of 0 (Clear) to 4 (Severe Activity)
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24 weeks
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Change From Baseline in Cutaneous Lupus Erythematosus-Quality of Life (CLE-QoL) Score
時間枠:24 Weeks
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Scores range from 0-100 where lower scores indicate a poorer quality of life
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24 Weeks
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Change From Baseline in Physician Global Assessment (PGA) Score
時間枠:24 Weeks
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Scores range from 0 (Clear / no signs of disease) to 5 (Very severe disease activity)
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24 Weeks
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Change From Baseline in Patient Global Assessment (PtGA) Score
時間枠:24 Weeks
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Scores range from 0 (Clear / no signs of disease) to 5 (Very severe disease activity)
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24 Weeks
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Change from Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Item Bank 2.0 - Cognitive Function - Short Form 8a
時間枠:24 Weeks
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Standardized measure of cognitive functioning where scores range from 0 - 100.
Scores lower than 50 represent impairment, while scores above 50 represent better than average functioning.
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24 Weeks
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協力者と研究者
スポンサー
捜査官
- 主任研究者:Victoria P Werth, MD、University of Pennsylvania
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
キーワード
追加の関連 MeSH 用語
その他の研究ID番号
- AISU0001
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
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