- RecruitingPHASE3
High-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN)
This study looks at new ways to treat high-risk neuroblastoma, a type of cancer, in children and young people. Researchers are testing different combinations of chemotherapy and radiation to find the most effective treatments to help patients live longer and healthier lives.
For: High-Risk Neuroblastoma · Patient With Insufficient Response ChemoimmunotherapyAustralia · Austria · Belgium - Ongoing, recruitingTherapeutic confirmatory (Phase III)
A Phase 3, multicenter, open-label, randomized study to evaluate the efficacy and safety of fedratinib compared to best available therapy in subjects with DIPSS - intermediate or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis and previously treated with ruxolitinib
This study is comparing a new medicine, fedratinib, with other common treatments for people with a type of bone marrow disorder called myelofibrosis. It's for patients whose myelofibrosis has been treated before with another drug called ruxolitinib. We want to see how well fedratinib shrinks the spleen.
For: Primary myelofibrosis · post-polycythemia vera myelofibrosis · or post-essential thrombocythemia myelofibrosisBelgium · Italy · Hungary - RecruitingPHASE3
A Study of Bomedemstat (IMG-7289/MK-3543) Compared to Best Available Therapy (BAT) in Participants With Essential Thrombocythemia and an Inadequate Response or Intolerance of Hydroxyurea (MK-3543-006)
This study looks at a new drug, bomedemstat, for essential thrombocythemia (ET). It compares bomedemstat to standard treatments for people whose current medicine, hydroxyurea, isn't working well or causes too many side effects. We want to see if bomedemstat is safer and works better.
For: Essential ThrombocythemiaUnited States · Argentina · Australia - Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humans
A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
This study is testing a new gene therapy called CTX001 for severe sickle cell disease. It aims to see if it's safe and helps improve symptoms by looking at things like how well the treatment works and if it reduces painful crises.
For: Severe sickle cell disease (SCD)Belgium · Italy - Ongoing, recruitingTherapeutic confirmatory (Phase III)
A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in subjects with Transfusion-Dependent β-Thalassemia
This study looks at a new gene therapy, Casgevy, for people with severe beta-thalassemia who need regular blood transfusions. Researchers want to see if this treatment is safe and can help patients produce enough healthy red blood cells on their own, reducing or stopping the need for transfusions.
For: Transfusion-dependent β-thalassemia (TDT)Germany - Ongoing, recruitingPhase I and Phase II (Integrated)- Other
An open-label phase 1/2 study to evaluate the safety, biological response and efficacy of a single dose of Temferon (autologous CD34+-enriched hematopoietic stem and progenitors cells genetically modified with human Interferon-α2) in patients with metastatic renal cell carcinoma
This study is testing a new treatment called Temferon for advanced kidney cancer that has spread. It checks if Temferon is safe, how the body reacts to it, and how well it works in people who have already tried standard treatments.
For: Patients with locally advanced/metastatic renal cell carcinoma with evidence of disease progression following standard of care treatmentItaly - AuthorisedTherapeutic confirmatory (Phase III)
Prospective multicenter clinical trial for risk estimation and treatment stratification in low and intermediate risk neuroblastoma patients - NB2015-LR
This study looks at treating a children's cancer called neuroblastoma. We want to find the best way to treat children who have a lower chance of a severe form of the disease. This will help doctors choose the right treatment to give children the best chance of getting better.
For: low and intermediate risk neuroblastomaGermany - RecruitingPHASE3
International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia
This study looks at new ways to treat children with a type of blood cancer called Acute Myeloid Leukaemia (AML). It compares different chemotherapy drugs, including a drug called gemtuzumab ozogamicin, and different types of stem cell transplants to find the most effective and safest treatments for young patients.
For: Acute Myeloid LeukaemiaAustralia · France · Ireland - RecruitingPHASE3
High Risk Neuroblastoma Study 1.8 of SIOP-Europe (SIOPEN)
This study looks at new ways to treat a serious childhood cancer called neuroblastoma. It tests different treatment combinations, including chemotherapy and immunotherapy, to find the best approach for children with high-risk forms of the disease. The aim is to improve their chances of getting better.
For: NeuroblastomaAustralia · Austria · Belgium