Multi-modular Chimeric Antigen Receptor T Cells tarGeting B7-H3 in Children, Teenage & Young Adult Sarcoma (MIGHTY)
MIGHTY is a multicentre, open label, phase 1 clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in children, teenagers and young adults aged 1-24 with relapsed or refractory (r/r) rhabdomyosarcoma (RMS), Ewing sarcoma (ES) or desmoplastic small round cell tumour (DSRCT).
The study will assess the feasibility of generating the ATIMP and administering hBRCA84D CAR T cells to patients with r/r RMS, ES or DSRCT.
調査の概要
詳細な説明
MIGHTY is a multicentre, open label, phase 1 clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in children, teenagers and young adults aged 1-24 with relapsed or refractory (r/r) rhabdomyosarcoma (RMS), Ewing sarcoma (ES) or desmoplastic small round cell tumour (DSRCT).
The ATIMP for this study (hBRCA84D CAR T cells) are autologous T cells targeting B7-H3 in patients with r/r RMS, ES, or DSRCT.
Patients will undergo an unstimulated leucapheresis for the generation of the ATIMP which will take approximately 15 days to generate.
Patients will receive lymphodepleting (LD) chemotherapy with fludarabine administered over 4 days (Day -6 to Day -3) and cyclophosphamide administered over 2 days (Day -4 and Day-3).
Patients will be treated at one of three dose levels following LD chemotherapy as described above.
The study will evaluate the feasibility of generating the ATIMP, the safety of administering ATIMP, the tolerability of the ATIMP and how effectively the CAR T cells engraft, expand and persist following administration in patients with r/r RMS, ES or DSRCT.
Following infusion of the CAR T cells, patients will be monitored for between 2-4 weeks as an inpatient. Following discharge, patients will enter the interventional follow up phase and be followed up for 1 year. Patients will be seen at 6 weeks post infusion then 3 monthly until 1 year post CAR T cells infusion.
If patients relapse within the first-year post CAR T cell infusion, they will come off the interventional follow up and will be followed up annually until 15 years after the CAR T infusion.
After completing the 1-year interventional phase of the study, all patients, irrespective of whether they progressed or responded to treatment, will enter long term follow up until 15 years post CAR T infusion.
研究の種類
入学 (推定)
段階
- フェーズ 1
連絡先と場所
研究連絡先
- 名前:Karin Straathof
研究連絡先のバックアップ
- 名前:MIGHTY Trial Coordinator
- 電話番号:+4420 7679 9852
- メール:ctc.mighty@ucl.ac.uk
研究場所
-
-
-
London、イギリス
- 募集
- Great Ormond Street Hospital
-
コンタクト:
- Olga Slater
-
London、イギリス
- 募集
- University College London Hospital
-
コンタクト:
- Sandra Strauss
-
-
参加基準
適格基準
就学可能な年齢
- 子
- 大人
健康ボランティアの受け入れ
説明
Inclusion Criteria:
- Age ≥ 1 and ≤ 24 years.
- Tissue diagnosis of RMS, ES or DSRCT
- Expression of B7-H3 in the tumour
- Relapsed or refractory disease after one or multiple lines of previous treatment.
- Measurable disease by cross sectional imaging. Patients with only bone marrow detectable disease (bone marrow aspirate or trephine) are NOT eligible for the study.
- At least 3 weeks or 5 half-lives, whichever is shorter, after treatment with agents on other early phase clinical trial.
- Performance status: Karnofsky (age ≥ 10 years) or Lansky (age < 10) score ≥ 50%.
- Creatinine ≤1.5 x Upper Limit of Normal (ULN) for age, if higher, an estimated (calculated) creatinine clearance must be ≥ 60 ml/min/1.73 m2.
- Left ventricular ejection fraction (LVEF) ≥ 50%
- Absolute lymphocyte count ≥ 0.25 x 109/L.
- Women of childbearing potential (WOCBP) must have a negative pregnancy test and agree to comply with the pregnancy reporting requirements of the protocol (if applicable).
- Written informed consent.
Exclusion Criteria:
- Patients with only bone marrow detectable disease in the absence of measurable disease by cross sectional imaging.
- Patients with active, inoperative central nervous system (CNS) disease including leptomeningeal disease.
- Active hepatitis B, C or HIV infection.
- Inability to tolerate leukapheresis.
- Clinically significant systemic illness or medical condition (e.g., significant cardiac, pulmonary, hepatic or other organ dysfunction), that in the judgement of the investigator is likely to interfere with assessment of safety or efficacy of the investigational regimen and its requirements.
- Any contraindication to lymphodepletion or to the use of Cyclophosphamide or Fludarabine as per the local SmPC.
- Any contraindication to the use of Anticoagulant Citrate Dextrose Solution.
- Known allergy to albumin, EDTA or DMSO.
- Primary immunodeficiency or history of autoimmune disease (e.g., Crohn's, rheumatoid arthritis, systemic lupus) requiring systemic immunosuppression /systemic disease modifying agents within the last 2 years.
- Prior treatment with investigational or approved gene therapy or cell therapy products.
- Life expectancy <3 months.
- Systemic corticosteroid therapy ≥ 0.05 mg/kg dexamethasone daily (or equivalent) at time of hBRCA84D CAR T cells infusion.
- Women who are pregnant or breastfeeding.
- Patients who have received any live vaccine in the six weeks prior to planned lymphodepletion
Exclusion criteria for the ATIMP infusion:
- Uncontrolled fungal, bacterial, viral, or other infection. Previously diagnosed infection for which the patient continues to receive antimicrobial therapy is permitted if responding to treatment and clinically stable at the time of scheduled hBRCA84D CAR T cell infusion.
- Systemic corticosteroid therapy ≥ 0.05 mg/kg dexamethasone daily (or equivalent) at time of hBRCA84D CAR T cell infusion.
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:hBRCA84D CAR T cells
Treatment with hBRCA84D CAR T cells
|
The hBRCA84D CAR T cells target B7-H3 positive 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 |
|---|---|---|
|
ATIMP 投与の安全性
時間枠:28日
|
ATIMPに因果的に関連するグレード3〜5の毒性、特に重度のサイトカイン放出症候群および重度の神経毒性の発生率。
|
28日
|
|
生成された治療薬の数と製造成功後に注入されたATIMPの数
時間枠:28日
|
ATIMP の生成の実現可能性は、製造に成功した後に生成される治療用製品の数と注入される ATIMP の数によって評価されます。
|
28日
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
無増悪生存期間 (PFS)
時間枠:1年
|
ATIMP静脈内投与後の無増悪生存期間(PFS)
|
1年
|
|
進行までの時間 (TTP)
時間枠:1年
|
ATIMP静脈内投与後の進行までの時間(TTP)
|
1年
|
|
全生存
時間枠:1年
|
ATIMP静脈内投与後の全生存期間
|
1年
|
|
Objective response rate
時間枠:1 year
|
Based on cross-sectional imaging after the ATIMP intravenous administration
|
1 year
|
協力者と研究者
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
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