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Methods of T Cell Depletion Trial (MoTD)
A multi-centre phase II trial of GvHD prophylaxis following unrelated donor stem cell transplantation comparing Thymoglobulin vs. Calcineurin inhibitor or Sirolimus-based post-transplant cyclophosphamide.
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MITHRIDATE: Ruxolitinib Versus Hydroxycarbamide or Interferon as First Line Therapy in High Risk Polycythemia Vera
The trial will be a phase III, randomised-controlled, multi-centre, international, open-label trial consisting of ruxolitinib versus best available therapy, where best available therapy is a choice of interferon alpha, any formulation permitted (IFN) or hydroxycarbamide (HC), and which will be elected by the Investigator prior to randomisation.
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Mos-FED (Mosaicism in Focal Epilepsy Cortical Dysplasia Tissue)
Focal cortical dysplasia (FCD) is a malformation of brain development, the most common cause of drug-resistant epilepsy and often caused by mutations in mammalian target of rapamycin (mTOR) pathway genes. Patients with FCD develop drug-resistant seizures. This study will look at FCD tissue removed during epilepsy surgery and aims to detect mutations in mTOR pathway genes in brain cells. Secondly, the investigators will establish if evidence of mutations found in brain cells can also be detected as circulating free DNA (cfDNA) in blood. By looking at which genes are made into proteins in individual cells found in epilepsy surgical ...
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MOS Prebiotic Potential in Older Adults
Mannooligosaccharides (MOS) derived from baker's yeast may have prebiotic properties such as improving gastrointestinal function. The aims of this study are to determine the effect of MOS on gastrointestinal function and symptoms and fecal microbiota in older adults.
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Mutant CALR-peptide Based Vaccine in Patients With Mutated CALR Myeloproliferative Neoplasm
The primary objective of this study is to assess the safety and tolerability of administrating mutated-CALR peptide Vaccine to patients with MPN. The researchers plan to enroll 10 patients over a 12 month period. Maximum length of participation in 80 weeks. Patients will be asked to complete questionnaires, bone marrow biopsies, research lab collection, and standard of care lab draw. This research will be taking place only at The Mount Sinai Hospital, specifically at the Ruttenberg Treatment Center.
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Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders
This is a Phase II study of allogeneic hematopoietic stem cell transplant (HCT) using a myeloablative preparative regimen (of either total body irradiation (TBI); or, fludarabine/busulfan for patients unable to receive further radiation). followed by a post-transplant graft-versus-host disease (GVHD) prophylaxis regimen of post-transplant cyclophosphamide (PTCy), tacrolimus (Tac), and mycophenolate mofetil (MMF).
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Myeloproliferative Neoplasms (MPNs) Patient Registry
The mandate of this MPN registry is to collect clinical information, including molecular results, from consenting patients with a variety of MPNs at different time points during the course of their disease.
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Myeloproliferative Neoplastic Diseases Observatory From Brest
All patients diagnosed or followed in Brest University Hospital for Philadelphia negative myeloproliferative neoplasms will be included in this observational study. Myeloproliferative neoplasms recorded included: polycythemia vera, essential Thrombocythemia and Primary Myelofibrosis. This is a not interventional study. Alive patients need to sign a non-opposition consent form. Patients will be followed until last news (death, change of reference centre...).
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Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Adult Essential Thrombocythemia
Objectives: To compare the efficacy and safety in Adult patients (≥18 years) diagnosed as essential thrombocythemia treated with the Pegylated Interferon Alfa-2b vs. Interferon Alfa. Study Design: A prospective, open-label, multicenter, randomized controlled clinical trial.
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Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Childhood and Adolescent Essential Thrombocythemia
Objectives: To compare the efficacy and safety in childhood and adolescent patients (<20 years) diagnosed as essential thrombocythemia treated with the Pegylated Interferon Alfa-2b vs. Interferon Alfa. Study Design: A prospective, open-label, nonrandomized, single-center clinical trial
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