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Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura
This is a single group, treatment, Phase 3, open-label, single-arm study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy (IST) without firstline therapeutic plasma exchange (TPE) with primary endpoint of remission in male and female participants aged 18 to 80 years with immune-mediated thrombotic thrombocytopenic purpura (iTTP). …
Palliative Spatially Fractionated (GRID) Radiotherapy Using Intensity Modulated Proton Therapy
Spatially fractionated radiotherapy (SFRT or GRID) addresses some limitations of traditional stereotactic body radiation therapy by relying on beam collimation to create high-dose "peaks" and intervening low-dose "valleys" throughout the target volume. Standard palliative radiotherapy regimens provide limited durability of response, and there are challenges with delivery to large tumors …
Baker Gordon Syndrome Natural History Study
The goal of this study is to conduct a prospective, longitudinal assessment of the natural clinical progression of children and adults with Synaptotagmin1-Associated Neurodevelopmental Disorder also known as Baker Gordon Syndrome (BAGOS). This will be performed by acquiring baseline measurements and developing effective outcome measures and diagnostic tools for the …
A MolEcularly Guided Anti-Cancer Drug Off-Label Trial
This is a prospective, open-label, non-randomized combined basket- and umbrella trial divided in two parts; a limited feasibility-oriented part 1 including 154 patients and 3 treatment cohorts and part 2 that will include an expanded cohort of patients and treatment cohorts. The overall aims of the study are to test …
Assessing Clinical Features and Outcome of Breast Cancer in PALB2 Mutation Carriers: the Palbreast Study
The Modena hereditary breast cancer group identified 3498 BRCA test candidates affected by breast cancer (BC). Among those, 392 were BRCA1/2 positive (11.2%). Since 2018, the site started to analyze eligible BC patients by multi gene panel (MGP) test. Fifty hundred sixty BRCA negative patients have been recalled, whereas other …
Shoulder Surgery Traditional vs Accelerated Rehabilitation Trial
This is a randomised controlled trial involving patients who have a double-row arthroscopic rotator cuff repair. Null hypothesis: There is no difference in outcome between standard rehabilitation and accelerated rehabilitation after arthroscopic rotator cuff repair. Following surgery, they will be randomised to one of two groups: Standard rehabilitation, with enforced …
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