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Trial of efficacy and safety of MC0518 versus best available therapy in patients with steroid-refractory acute graft versus host disease
EU CTIS ID: 2023-503952-28-00
What this study is testing
Comparative evaluation of the overall response rate in paediatric participants with steroid-refractory acute graft-versus-host disease (SR aGvHD) at Visit Day 28 after treatment with MC0518 or first used best available therapy (BAT)
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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.
You likely can't join if
- 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.
See the full eligibility criteria
Who can join
- 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.
What rules you out
- 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.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.