CTIS2023-503952-28-00招募中1 期
A Randomised, Open label, Controlled, Multicentre, Phase 2 Trial of First line Treatment with Mesenchymal Stromal Cells MC0518 Versus Best Available Therapy in Paediatric Participants with Steroid refractory Acute Graft versus host Disease After Allogeneic Stem Cell Transplantation (BALDER Trial) - MC MSC.2/aGvHD
Medac Gesellschaft fuer klinische Spezialprapaerate mbH0 个研究点目标入组 54 人开始时间: 2023年5月19日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 54
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 17（—）
- 性别
- All
入选标准
- •Participant had a previous allogeneic HSCT as indicated for non-malignant (including inborn errors of metabolism, primary immunodeficiencies, haemoglobinopathies, and bone marrow failure syndromes) or haematological malignant disease or neuroblastoma., Participant has been clinically diagnosed with Grade II to IV aGvHD according to Harris et al. A biopsy of the involved organs with aGvHD is encouraged but not required., Participant has experienced failure of previous first line aGvHD treatment (ie, SR aGvHD), defined as: - aGvHD progression within 3 to 5 days of therapy onset with = 2 mg/kg/day of prednisone equivalent or - failure to improve within 5 to 7 days of treatment initiation with = 2 mg/kg/day of prednisone equivalent or - incomplete response after > 28 days of immunosuppressive treatment including at least 5 days with = 2 mg/kg/day of prednisone equivalent., Male or female participant who is = 28 days and < 18 years of age and has a minimum BW of 3.2 kg at the Screening Visit., Participant has an estimated life expectancy of > 28 days., Participant, if female and of childbearing potential, agrees to use a highly effective contraceptive measure starting at the Screening Visit and continuing throughout the entire trial period. The definition of women of childbearing potential and a complete list of highly effective contraceptive measures are included in an appendix to the protocol., Participant, if a fertile male, agrees to sexual abstinence or to use a condom during sexual activity with their female partner of childbearing potential or pregnant partner. Additionally, if their partner is a woman of childbearing potential, then their partner must use an additional highly effective contraceptive method during sexual activity starting at the Screening Visit and continuing throughout the entire trial period. The definition of fertile men and a complete list of highly effective contraceptive measures are included in an appendix to the protocol., A written informed consent of the participant’s parent(s) / legal guardian(s) (and participant’s assent, when applicable) has been obtained according to national regulations.
排除标准
- •Participant has overt relapse or progression or persistence of the underlying disease., Participant has an uncontrolled infection (eg, sepsis or multi-organ failure) including significant bacterial, fungal, viral, or parasitic infection requiring treatment., Participant has received treatment with any other investigational agent within 30 days or 5 half lives (whichever is longer) before the Screening Visit, Participant has received the last HSCT for a solid tumour disease other than neuroblastoma., Participant has graft-versus-host disease overlap syndrome as defined by Jagasia et al., Participant has received systemic first line treatment for aGvHD other than steroids and a prophylaxis with other than calcineurin inhibitors, mammalian target of rapamycin inhibitors, anti thymocyte globulin, mycophenolate mofetil, methotrexate, abatacept, or cyclophosphamide. Please Note: In vitro or in vivo graft manipulation to prevent graft-versus-host disease (eg, T cell depletion) during HSCT is permitted. Restart of initial prophylaxis with calcineurin inhibitors, mammalian target of rapamycin inhibitors, or mycophenolate mofetil after aGvHD onset is permitted., Participant has received prior mesenchymal stromal cell (MSC) treatment, including MC0518/Obnitix®., Participant has a known pregnancy (as confirmed by a positive pregnancy test result at the Screening Visit) and / or is breastfeeding., Participant has a known hypersensitivity to MC0518 and / or its excipients (dimethyl sulfoxide, human serum albumin, isotonic sodium chloride solution)., Participant has a known hypersensitivity or any contraindication to the Investigator’s choice BAT (extracorporeal photopheresis, anti thymocyte globulin, etanercept, infliximab, or ruxolitinib) and / or its excipients. For a list of excipients please refer to the respective Summary of Product Characteristics., Participant has an underlying or current medical or psychiatric condition that, in the opinion of the Investigator, would interfere with the evaluation of the participant.
研究者
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