Found 219 clinical trials
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
Background Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids, or steroids don t work. Other patients must get blood transfusions regularly which are time consuming and can have significant side effects. The drug eltrombopag can increase red blood cells. Researchers want to see if it …
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- 19 Feb, 2024
Gene Therapy for X Linked Severe Combined Immunodeficiency
A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCIDsevere combined immune deficiency .The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number …
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- 19 Feb, 2024
A Psychophysiological Database of Maximal Effort Tests.
The aim of this study is first to construct a database containing psychophysiological data from athletes performing a maximal effort test. Second, the investigators want to use the data to optimize the methodological approach during, and trainings advice after maximal effort tests.
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- 19 Feb, 2024
Gene Therapy for Fanconi Anemia Complementation Group A
The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the …
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- 19 Feb, 2024
Octagam 10% Therapy in COVID-19 Patients With Severe Disease Progression
This is a randomized, double-blind, placebo-controlled, multicenter, Phase 3 study to evaluate if high-dose Octagam 10% therapy can stabilize or improve clinical status in patients with severe Coronavirus disease
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- 19 Feb, 2024
Part II: Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia
The Investigators propose to test the hypothesis that GCSF enhances the clinical outcome of biliary atresia in a multi-institutional Phase 2 trial to prospectively evaluate the safety and efficacy of GCSF in each of the 2 groups of newly diagnosed BA patients: KBA (i.e., Kasai-operated) or NoK (i.e., patients who …
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- 19 Feb, 2024
Vaccine Responsiveness After CAR-T Cell Therapy
This phase I trial will use the inactivated rabies virus vaccine to assess immune function in patients who previously underwent B cell targeted chimeric antigen receptor-modified T cell immunotherapy (CARTx). A cohort of healthy volunteers will also be enrolled as a comparator group. CARTx is a new treatment for patients …
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- 19 Feb, 2024
Clinical Feasibility of Minimally Supervised Therapy After Stroke With a Hand Rehabilitation Robot
The ReHapticKnob is a robot for hand rehabilitation after stroke. We aim to investigate the feasibility of minimally supervised therapy with the ReHapticKnob with stroke patients in a rehabilitation clinic, evaluate the usability of the ReHapticKnob (user interface and implemented exercises which were adapted for independent usage), and quantify the …
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- 19 Feb, 2024
Metastasis-directed Therapy in Castration-refractory Prostate Cancer
The aim is to define the postponement of next line systemic treatment (NEST), by the use of metastasis-directed therapy in patients with oligoprogressive castration-refractory prostate cancer. This will be defined by the NEST-free survival. Furthermore the investigators will use 18F PSMA PET-CT as investigational imaging, to assess the predictive value …
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- 19 Feb, 2024
An Extension Study MRI/US Fusion Imaging and Biopsy in Combination With Nanoparticle Directed Focal Therapy for Ablation of Prostate Tissue
To determine the efficacy of using MRI/US fusion imaging technology to direct focal ablation of prostate tissue using nanoparticle-directed laser irradiation.
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- 19 Feb, 2024