代謝機能障害関連脂肪肝疾患または代謝機能障害関連脂肪性肝炎(MASLD/MASH)を伴う成人参加者のALN-CIDEBを評価する研究
A Phase 1/2a, Randomized, Double-Blind, Placebo-Controlled, Two-Part Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of a Single Dose of ALN-CIDEB in Adult Participants With Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD) and Two Doses of ALN-CIDEB in Adult Participants With Metabolic Dysfunction-Associated Steatohepatitis (MASH)
This study is researching an experimental drug called ALN-CIDEB, also referred to as "study drug". The study is focused on participants with metabolic dysfunction-associated steatotic liver disease (MASLD) (Part A) and metabolic dysfunction-associated steatohepatitis (MASH) (Part B). MASLD and MASH are long-lasting liver conditions caused by having too much fat in the liver.
The aim of the study is to see how safe and tolerable the study drug is.
The study is looking at several other research questions, including:
- What side effects may happen from taking the study drug
- How the study drug works to change liver fat content
- How much study drug and study drug metabolites (byproducts of the body breaking down the study drug) are in the blood at different times
調査の概要
研究の種類
入学 (実際)
段階
- フェーズ2
- フェーズ 1
連絡先と場所
参加基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
Key Inclusion Criteria:
- Part A: 18 to 55 years at Screening Visit 1 with MASLD, at Screening Visit 1 Part B: 18 to 65 years at Screening Visit 1 with a diagnosis of MASH, at Screening Visit 1
- Body Mass Index (BMI) ≥30 kg/m2 and ≤40 kg/m2 at Screening Visit 1
- Controlled-Attenuation Parameter (CAP) ≥285 dB/m by FibroScan during screening as described in the protocol
- Liver fat content ≥8.5% by MRI-PDFF during screening
- If on anti-hypertensive and/or lipid lowering medications and/or glucose lowering medications, must be on generally stable dose(s) for at least 12 weeks prior to screening and no changes to the dose(s) are anticipated during the study
- Part B: A diagnosis of MASH documented in the participant's medical history, or a clinical suspicion of MASH based on non-invasive biomarkers (eg, evidence of fatty liver on imaging and elevated liver enzymes) and clinical risk factors, including having a history of 2 or more elements of metabolic syndrome, as defined in the protocol
- Part B: Screening percutaneous liver biopsy NAFLD Activity Score (NAS) ≥3 and fibrosis stage, as defined in the protocol
Key Exclusion Criteria:
- Known historical or current diagnosis of portal hypertension or cirrhosis based on clinical assessment, imaging, and/or liver biopsy
- Known historical or current diagnosis of other forms of chronic liver disease, as defined in the protocol
- Prior or current suspected or known drug-induced liver injury within 1 year prior to screening
- History of liver transplant, current placement on a liver transplant list, or Model for End-stage Liver Disease (MELD) score >12
- Contraindication to MRI examinations, such as persons with cardiac pacemaker and implants made of metal, severe claustrophobia, size restrictions, or other contraindications for MRI
- Liver stiffness measurement, laboratory parameter assessment, estimated Glomerular Filtration Rate (GFR), and evidence of uncontrolled hypertension, as defined in the protocol
- Evidence of Human Immunodeficiency Virus (HIV) infection, Hepatitis B Virus (HBV) infection, or Hepatitis C Virus (HCV) infection during screening, as described in the protocol
- History of Type 1 Diabetes
- Bariatric surgery, including any procedures to revise, reverse, or remove any previous bariatric surgery interventions, within approximately 5 years prior to randomization or planned during the study period
NOTE: Other protocol-defined inclusion/exclusion criteria apply.
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:順次割り当て
- マスキング:ダブル
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的:Part A
Randomized per the protocol |
Administered per the protocol Administered per the protocol |
|
実験的:Part B
Randomized per the protocol |
Administered per the protocol Administered per the protocol |
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 |
時間枠 |
|---|---|
|
茶の重症度
時間枠:最大48週間
|
最大48週間
|
|
治療関連有害事象(TEAE)の発生率
時間枠:最大48週間
|
最大48週間
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
血漿中のAln-Cidebの総濃度
時間枠:最大48週間
|
最大48週間
|
|
|
血漿中の潜在的な主要代謝産物の総濃度
時間枠:最大48週間
|
最大48週間
|
|
|
磁気共鳴画像法-プロトン密度脂肪分画(MRI-PDFF)による肝臓脂肪分画の変化
時間枠:ベースラインから48週間まで
|
ベースラインから48週間まで
|
|
|
アスパラギン酸アミノトランスフェラーゼ(AST)の変化
時間枠:ベースラインから48週間まで
|
ベースラインから48週間まで
|
|
|
アラニンアミノトランスフェラーゼ (ALT) の変化
時間枠:ベースラインから48週まで
|
ベースラインから48週まで
|
|
|
Urinary recovery of ALN-CIDEB as a proportion of the dose
時間枠:Up to 36 Weeks
|
Part A |
Up to 36 Weeks
|
|
Urinary recovery of potential major metabolite(s) as a proportion of the dose
時間枠:Up to 36 Weeks
|
Part A |
Up to 36 Weeks
|
|
Change in hepatic Cell death-Inducing DNA fragmentation factor alpha-like Effector B (CIDEB) messenger RiboNucleic Acid (mRNA) level
時間枠:Baseline up to 36 Weeks
|
Part B |
Baseline up to 36 Weeks
|
協力者と研究者
捜査官
- スタディディレクター:Clinical Trial Management、Regeneron Pharmaceuticals
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
その他の研究ID番号
- ALN-CIDEB-NASH-2486
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
IPD プランの説明
All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
IPD 共有時間枠
When Regeneron has:
- received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development
- made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry)
- the legal authority to share the data, and
- ensured the ability to protect participant privacy
IPD 共有アクセス基準
Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf
IPD 共有サポート情報タイプ
- STUDY_PROTOCOL
- SAP
- ICF
- ANALYTIC_CODE
- CSR
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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