A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
A Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure After Transplantation With TCR Alpha/Beta Grafts Depletion in Patients With Wiskott-Aldrich Syndrome.
調査の概要
状態
詳細な説明
Severe graft dysfunction, such as the degree of donor chimerism predominantly in the myeloid compartment is one of major problem in patients with Wiskott-Aldrich syndrome (WAS), especially after hematopoietic stem cell transplantation (HSCT) from alternative donor. It often leads to the development of severe thrombocytopenia or even transplants rejection. In this study the hypothesis is that the use of plerixafor and G-CSF as additional agents in conditioning regimen would offers advantages due to lowing risk of mixed chimerism after HSCT. This effect is based on the fact that simultaneous use of plerixafor with G-CSF is efficient in inducing stem cell release and opening of bone marrow (BM) niches. Moreover, stem cell release probably leads to liberation of host stem cells from the anti-apoptotic effects of the BM stroma for the more powerful effect of chemotherapy.
In this study, the investigators use TCR alpha/beta grafts depletion of the grafts as basic technology for HSCT from haploidentical and unrelated donors approved in Institution.
Thus, the purpose of this study is to evaluate the safety and efficiency of myeloablative conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation with TCR alpha/beta grafts depletion in patients with Wiskott-Aldrich syndrome.
研究の種類
入学 (予想される)
段階
- フェーズ2
連絡先と場所
研究場所
-
-
-
Moscow、ロシア連邦、117997
- 募集
- Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
-
コンタクト：
- Dmitry Balashov, MD, PhD
- 電話番号：6534 +7(495)287-6570
- メール：bala8@yandex.ru
-
コンタクト：
- Michael Maschan, Professor
- 電話番号：+7(926)287-6570
- メール：mmaschan@yandex.ru
-
副調査官：
- Michael Maschan, Professor
-
副調査官：
- Alexandra Laberko, MD
-
副調査官：
- Svetlana Kozlovskaya, MD
-
副調査官：
- Elena Gutovskaya, MD
-
副調査官：
- Anna Shcherbina, Professor
-
-
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:
- Patients aged ≥ 1 months and < 19 years
- Patients diagnosed with Wiskott-Aldrich syndrome eligible for an allogeneic transplantation and lacking a related HLA-matched donor
- Lansky/Karnofsky score > 40, WHO > 4
- Signed written informed consent
Exclusion Criteria:
- Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 ml / min)
- Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction <40%)
- Serious concurrent uncontrolled medical disorder
- Lack of parents' informed consent.
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：なし
- 介入モデル：単一グループの割り当て
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的：Plerixafor/G-CSF for HSCT conditioning
Myeloablative conditioning regimen with Plerixafor and G-CSF as addition agents before stem cell transplantation in WAS patients.
|
Mobilization of hematopoietic stem (HSC) into circulation
Directed inhibition of CXC chemokine receptor type 4 (CXCR4) for opening enough BM niches for adequate donor HSC engraftment.
|
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 |
|---|---|---|
|
Event free survival (EFS)
時間枠：24 months
|
The EFS probability compared with historical control.
We mean event as patient's death, second transplantation or persistence of severe thrombocytopenia
|
24 months
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Overall survival (OS)
時間枠：24 months
|
The OS probability compared with historical control.
|
24 months
|
|
Percentage of patients with full/mixed donor chimerism
時間枠：12 months
|
Evaluation of the percentage of patients with the full/mixed donor chimerism (whole blood and CD3+ lineage).
In addition, patients will be divided in accordance with % of donors cells: >95%; 50%-95%; 10%-49%; <10%.
All data will be compared with historical control
|
12 months
|
|
Transplant related mortality (TRM)
時間枠：24 months
|
The TRM probability compared with historical control.
|
24 months
|
|
Severe thrombocytopenia (ST)
時間枠：24 months
|
The ST probability after HSCT compared with historical control
|
24 months
|
|
Autoimmune complications (AC)
時間枠：24 months
|
The AC probability after HSCT compared with historical control
|
24 months
|
|
Acute Graft Versus Host Diseases (aGVHD)
時間枠：12 months
|
Cumulative Incidence and severity of aGVHD
|
12 months
|
|
Chronic Graft Versus Host Diseases (cGVHD)
時間枠：24 months
|
Cumulative Incidence and severity of cGVHD
|
24 months
|
|
Plerixafor related complications (PRC)
時間枠：2 week
|
PRC: severity, features, incidence
|
2 week
|
協力者と研究者
捜査官
- スタディチェア：Alexei Maschan, Professor、Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
- 主任研究者：Dmitry Balashov、Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
出版物と役立つリンク
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (実際)
研究の完了 (予想される)
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 (見積もり)
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
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。
G-CSF for Conditioning before HSCT.の臨床試験
-
New Approaches to Neuroblastoma Therapy ConsortiumNational Cancer Institute (NCI)完了
-
City of Hope Medical CenterNational Cancer Institute (NCI)募集急性骨髄性白血病 | ホジキンリンパ腫 | 骨髄線維症 | 急性リンパ芽球性白血病 | 慢性リンパ性白血病 | 非ホジキンリンパ腫 | 骨髄異形成症候群 | 造血器系およびリンパ系の新生物 | 骨髄増殖性腫瘍 | 慢性骨髄性白血病、BCR-ABL1陽性アメリカ
-
Masonic Cancer Center, University of Minnesota完了
-
Ronald LevyNational Institutes of Health (NIH)完了
-
City of Hope Medical CenterNational Cancer Institute (NCI)一時停止
-
M.D. Anderson Cancer CenterNational Cancer Institute (NCI)完了
-
M.D. Anderson Cancer CenterNational Cancer Institute (NCI)終了しました