Intrabone Infusion of Umbilical Cord Blood Stem Cells
A New Approach to Improve Long-term Hematopoietic Recovery After Allogeneic Umbilical Cord Blood Transplantation in Children - Intrabone Infusion of Umbilical Cord Blood Stem Cells
調査の概要
詳細な説明
Umbilical cord blood transplantation (UCBT) has been increasingly used to treat malignant and non-malignant haematological, immunodeficiency and some metabolic diseases. UCBT offers the advantages of easy procurement, no risk to donors, a reduced risk of transmitting infections, immediate availability of cryopreserved units, and acceptable partial HLA mismatches. However, patients treated with UCBT show delayed hematopoietic and immunological recoveries, have higher rates of infection, and relapse from the original malignant disease, which can all lead to life threatening problems. UCBT can also result in a higher rate of graft failure compared to other hematopoietic stem cell transplantation (HSCT) sources. The problem of a slower hematopoietic recovery post-UCBT has been addressed using a number of different approaches in adult patients.In adults, use of intrabone injection of cord blood results in a faster hematopoietic recovery in a phase II study. However, there is no clinical trial in pediatric patients.
This study is addressed to determine if a change in the cord blood stem cell infusion method can increase and accelerate hematopoietic reconstitution after UCBT in pediatric patients.
研究の種類
入学 (実際)
段階
- フェーズ2
連絡先と場所
研究場所
-
-
Quebec
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Montreal、Quebec、カナダ、H3C1T5
- Centre Hospitalier Universitaire Sainte-Justine
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-
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:
- One to 21 years of age;
- More than 10 kg in weight;
- Diagnosis of hematopoietic disorders (malignant or not) with an indication for hematopoietic stem cell transplantation;
- Absence of an HLA-identical related donor;
- Availability of a single cord blood (CB) with at least 3 x 10^7 nucleated cells (NCs)/kg (if HLA identical or 1 HLA-mismatch) or at least 4 x 10^7 NCs/kg (if a 2 HLA-mismatch) at freezing. Use of two CB units ("double cord transplant") will be allowed provided that: 1) a single CB unit fulfilling the above criteria is not available; 2) a maximum of 2 HLA mismatch is present for each CB unit; and 3) a minimum of 4 x 10^7 NCs/kg (as the sum for both CB units) is present at freezing.
- A myeloablative-conditioning regimen;
- A Lansky (for patients less than 16 years of age) or Karnofsky (for patients more than 16 years of age) score equal to or higher than 70%.
- Adequate organ function as follows:
- Cardiac (ejection fraction > 50%);
- Renal (serum creatinine within the normal range for age, and creatinine clearance or a GFR > 70 ml/min/1.73m2);
- Hepatic (AST or ALT < 5 x upper limit of normal for age);
- Pulmonary (FEV1, FVC, and DLCO ≥ 50% by pulmonary function tests or, in children unable to cooperate, no sign of dyspnea at rest, no exercise intolerance, no supplementary oxygen therapy, and a normal pulmonary radiography or pulmonary scan);
- No sign of uncontrolled systemic bacterial, fungal or viral infection;
- Written informed consent by the patient or his/her legal guardian
Exclusion Criteria:
- Non-myeloablative conditioning;
- Pregnancy or breastfeeding;
- HIV positive serology;
- Bone disease (e.g. osteopetrosis, osteogenesis imperfecta)
- Previous autologous or allogeneic hematopoietic stem cell transplantation performed up to one year before enrolment, except in the case of non-engraftment or early rejection of a previous allogeneic stem cell transplantation.
- Active skin infection at the site of intrabone injection.
- History of intolerance/allergy to sedation medications or local anesthetics.
- Contraindication to sedation
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：Intrabone umbilical cord blood tranplant
Intrabone infusion of umbilical cord blood stem cells
|
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 |
|---|---|---|
|
Platelet recovery rate
時間枠：at 100 days post- transplantation
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First of seven days of untransfused platelet count higher than 20 x 10^9/L
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at 100 days post- transplantation
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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 |
|---|---|---|
|
Neutrophil recovery rate
時間枠：at 60 days post- transplantation
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First of three days of absolute neutrophil count equal or higher than 0.5 x 10^9/L
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at 60 days post- transplantation
|
|
Immunological reconstitution
時間枠：at 30, 60, 100, 180, and 360 days post- transplantation
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Total number of T cells (and subpopulations), B and NK (natural killer) cells in peripheral blood at different time-points
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at 30, 60, 100, 180, and 360 days post- transplantation
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|
Donor chimerism rate
時間枠：at 30, 60,100, and 180 days post-transplantation
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Percentage of donor(s) cells in peripheral blood at different time-points
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at 30, 60,100, and 180 days post-transplantation
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|
Acute GVHD (grade 2-4) rate
時間枠：at 180 days
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Incidence of grade II-IV acute GVHD (Graft versus Host Disease)
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at 180 days
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Infection rate (bacterial, viral, fungal and parasitic)
時間枠：at 180 days post-transplantation
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Clinical and microbiological documented infections will be reported according to anatomic site, date of onset and microorganism
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at 180 days post-transplantation
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Event-free and overall survival
時間枠：at 2 years
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Event-free survival is defined as the time interval between transplantation and relapse, graft rejection, death or last follow-up, whichever occurs first; Overall survival is defined as the time between transplantation and death or last follow-up
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at 2 years
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Adverse infections (grade and frequency)
時間枠：at one month post-transplantation
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Toxicity will be assessed using the Common Terminology Criteria for Adverse Events v4.0
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at one month post-transplantation
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chronic GVHD
時間枠：at 2 years post-transplantation
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Incidence of chronic GVHD (Graft versus Host Disease) will be scored according to NIH consensus on chronic GVHD
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at 2 years post-transplantation
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協力者と研究者
協力者
捜査官
- 主任研究者：Henrique Bittencourt, MD, PhD、St. Justine's Hospital
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
一次修了 (実際)
研究の完了 (実際)
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
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