Phase 2 and 3 Placebo Controlled & Long-term Extension Study of Rilzabrutinib in Adults With iCVID to Evaluate Efficacy, Safety, and Tolerability
Phase 2, Randomized, Double-blind, Placebo-controlled Study (Part A) Followed by a Phase 3, Randomized, Double-blind, Placebo Controlled Study (Part B) With an Open Label Extension (Part C) and Long-term Extension (Part D) to Evaluate the Efficacy, Safety and Tolerability of Rilzabrutinib in Adult Participants With Inflammatory Common Variable Immunodeficiency (iCVID)
This is a 4-part study where both Part A and Part B, which starts after the completion of Part A, are randomized (2:1), double-blinded, placebo-controlled studies, followed by an open label (Part C) and long-term extension period (Part D), phase 2 and 3 study, designed to evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of rilzabrutinib in adult patients with iCVID.
In Part A, all participants will have iCVID associated ILD and at least one additional inflammatory/ autoimmune complication (cytopenia [thrombocytopenia, anemia] and/or lymphoproliferative disease [splenomegaly, lymphoid hyperplasia]). At the completion of 24 weeks in Part A, participants will be assessed to the predefined No Response Citeria and may elect to discontinue the DB period (Part A) and enter the OL period (Part C).
Alternatively, for Part B, all participants will have CVID associated with at least one of the following: ILD, other inflammatory/ autoimmune complications (cytopenia, lymphoproliferative disease).
Part A: 32-week DB treatment of rilzabrutinib or placebo. Part B: 32-week DB treatment of rilzabrutinib or placebo. Part C: 32-week OL treatment of rilzabrutinib. Part D: OL treatment of rilzabrutinib until the last-patient-in (LPI- Part D) completes 52 weeks of treatment.
調査の概要
研究の種類
入学 (推定)
段階
- フェーズ2
- フェーズ 3
連絡先と場所
研究連絡先
- 名前：Trial Transparency email recommended (Toll free for US & Canada)
- 電話番号：option 6 800-633-1610
- メール：Contact-US@sanofi.com
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
Inclusion criteria
- Participants who have previously been diagnosed with CVID based on the following criteria A) Documented low serum IgG compared to age-adjusted reference ranges B) Documented low IgA and/or IgM compared to age-adjusted reference range C) Immunophenotypic findings consistent with CVID D) Absence of any other defined immunodeficiency state
- Receiving immunoglobulin IV or SC replacement therapy
- Participants who have been diagnosed with one or more of the following inflammatory complications (iCVID) A) Interstitial lung disease (ILD) B) CVID-related cytopenia for >3 months since diagnosis - thrombocytopenia or anemia C) Lymphoproliferative disease on historical CT imaging - splenomegaly or lymphoid hyperplasia
- Eastern Cooperative Oncology Group performance status Grade 2 or lower, at screening
- Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
Exclusion criteria
- Active pulmonary infection at screening that could account for the HRCT abnormalities
- Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator (e.g. hepatobiliary conditions causing hepatic impairment)
- Refractory nausea or vomiting
- Chronic uncontrolled infectious diseases as determined by the Investigator
- Laboratory abnormalities at the screening visit (e.g. elevated liver enzymes)
- Recent history of substance, alcohol abuse or mental illness
- Malabsorption syndromes, active moderate-to-severe enteropathy
- Concurrent treatment with immunosuppressive or immunomodulatory drugs other than corticosteroids The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
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 |
|---|---|
|
プラセボコンパレーター：placebo
placebo |
Oral tablet |
|
実験的：rilzabrutinib
rilzabrutinib |
Oral tablet |
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 |
|---|---|---|
|
Proportion of participants with iCVID who meet any responder definitions
時間枠：At Week 32
|
In participants with ILD at baseline, a responder is defined as achieving a clinically meaningful response at Week 32. In participants with cytopenia (thrombocytopenia, anemia) at baseline, a responder is defined as achieving therapeutic efficacy at Week 32, defined as Durable platelet response or Durable hemoglobin response (DHR) between W16 and W32, and no rescue medication for thrombocytopenia is taken at any time. In participants with lymphoproliferative disease (splenomegaly or lymphoid hyperplasia) at baseline, a responder is defined as achieving therapeutic efficacy by Week 32. |
At Week 32
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Change from baseline in FACIT-Fatigue total score at Week 24 in Part A and Week 32 in Part B
時間枠：At Week 24 in Part A and Week 32 in Part B
|
At Week 24 in Part A and Week 32 in Part B
|
|
|
Percent change from baseline in FVC% predicted at Week 32
時間枠：At Week 32
|
At Week 32
|
|
|
Percent change from baseline in diffusing capacity of the lung for carbon monoxide at Week 32
時間枠：At Week 32
|
At Week 32
|
|
|
Change from baseline in FACIT-dyspnea severity score at Week 24 in Part A and Week 32 in Part B
時間枠：At Week 24 in Part A and Week 32 in Part B
|
At Week 24 in Part A and Week 32 in Part B
|
|
|
Change from baseline in time spend in moderate to vigorous physical activity measured with a wearable device
時間枠：At Week 32
|
At Week 32
|
|
|
Incidence of treatment-emergent adverse events (TEAE), serious AEs (SAE), TEAE leading to permanent discontinuation, adverse events of special interest (AESI)
時間枠：Until Week 116 of LPI for Part D
|
including clinical laboratory evaluations, vital sign measurements, and electrocardiograms |
Until Week 116 of LPI for Part D
|
協力者と研究者
スポンサー
研究記録日
主要日程の研究
研究開始 (推定)
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
追加の関連 MeSH 用語
その他の研究ID番号
- EFC20516
- 2026-525354-10 (レジストリ識別子：CTIS)
- U1111-1325-2663 (レジストリ識別子：WHO UTN)
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
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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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