IDH1 (AG 120) Inhibitor in Patients With IDH1 Mutated Myelodysplastic Syndrome
Myelodysplastic SyndromesAcute Myeloid Leukemiapatients with MDS (Myelodysplastic Syndrome) and mutated IDH1 patients will be treated with AG120 (IDH1 inhibitor)
Intravenous Immunoglobulins for the Treatment of Primary Sjögren's Syndrome Associated Painful Sensory...
Primary Sjögren's Syndrome Painful Sensory NeuropathiesTo summarise, the peripheral neurological complications experienced by patients with primary Sjögren's syndrome are particularly bothersome since they are common and often result in significant disability related to pain or motor impairment. There is currently no standard treatment for these patients. As these neuropathies are caused by an immune system dysfunction, which is related to a variety of different pathogenic mechanisms, the use of immunosuppressant or immunomodulator drugs is often justified. With the exception of the vascularitis-related multiplex mononeuropathies, other pSS-related neuropathies could be suitable candidates for IV Ig treatment.
Study in Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Patients to Determine...
Acute Myeloid LeukemiaMyelodysplastic SyndromeAn open-label, phase I, multi-center study to determine in relapsed/refractory (r/r) acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) patients the recommended dose of CYAD-02 after a non-myeloablative preconditioning chemotherapy followed by a potential CYAD-02 consolidation cycle for non-progressive patient. A maximum of 27 r/r AML/MDS patients will be evaluated in this study in case of no dose limiting toxicity (DLT) and no replacement of patients.
A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic...
Acute Myeloid LeukemiaMyelodysplastic SyndromeThis is an open-label, dose escalation study to evaluate the safety, toxicity, and pharmacokinetics (PK) as well as preliminary efficacy of BTX-A51 capsules in participants with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The study will be done in two phases. Phase 1a of this study is designed to determine the dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD) of orally administered BTX-A51 in up to 35 participants who are evaluable for toxicity. Once the MTD is determined, it is planned that an additional 15 participants will be enrolled in Phase 1b of this study for additional experience with safety and efficacy, and to determine the recommended Phase 2 dose (RP2D) which may or may not be different from the MTD. Continued treatment will be available under this study protocol for up to eight 28-day cycles (Continued Treatment Phase) if the Investigator judges the benefit outweighs the risk. Once BTX-A51 treatment has completed, participants will be contacted by telephone every 3 months for up to 2 years after their last treatment for survival status and anticancer therapy (Overall Survival Follow-up).
Early Use of Airway Pressure Release Ventilation (APRV) in ARDS
Acute Respiratory Distress SyndromeAirway pressure release ventilation (APRV) is a time-cycled, pressure controlled, intermittent mandatory ventilation mode with extreme inverse I:E ratios. Currently it is considered as a non-conventional ventilatory mode. The investigators aim to compare APRV with conventional mechanical ventilation (MV) in patients with acute respiratory distress syndrome (ARDS).
A Study of Low and Standard-dose Ticagrelor After Intervention for ACS Patients
Acute Coronary SyndromeCoronary Stent ImplantationThis study is to evaluate the safety and efficacy of 60mg ticagrelor plus 100mg Aspirin to prevent major adverse cardiovascular and cerebrovascular events in one years after drug-eluting stents implantation for Chinese ACS patients compared with 90mg ticagrelor plus 100mg Aspirin
Mogamulizumab + Low-Dose Total Skin Electron Beam Tx in Mycosis Fungoides & Sézary Syndrome
Sezary SyndromeMycosis FungoidesThe purpose of this study is to determine the efficacy of the combination of LD-TSEBT and mogamulizumab in patients with MF and SS. And to evaluate the secondary measures of clinical benefit of the combination therapy and to evaluate the safety and tolerability of the combination in patients with MF and SS.
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
Angelman SyndromeThe primary objective of the study is to evaluate the safety and tolerability of multiple-ascending doses of GTX-102 administered by intrathecal (IT) injection to participants with Angelman Syndrome (AS).
ASIA Down Syndrome Acute Lymphoblastic Leukemia 2016
Down SyndromeAcute Lymphoblastic Leukemia1 moreTo evaluate the outcome of a prednisolone and low dose methotrexate based protocol in Down syndrome children with ALL (DS-ALL) in an Asia-wide study. The treatment protocol was modified based upon backbone of Taiwan Pediatric Oncology Group (TPOG)-ALL protocol in which risk classification will be guided by level of flow minimal residual disease (MRD) instead.
Venetoclax Basket Trial for High Risk Hematologic Malignancies
Myelodysplastic Syndromesde Novo15 moreThis trial is evaluating the safety and tolerability of venetoclax with chemotherapy in pediatric and young adult patients with hematologic malignancies, including myelodysplastic syndrome (MDS), acute myeloid leukemia derived from myelodysplastic syndrome (MDS/AML), and acute lymphoblastic leukemia (ALL)/lymphoblastic lymphoma (LBL). The names of the study drugs involved in this study are below. Please note this is a list for the study as a whole, participants will receive drugs according to disease cohort. Venetoclax Azacitidine Cytarabine Methotrexate Hydrocortisone Leucovorin Dexamethasone Vincristine Doxorubicin Dexrazoxane Calaspargase pegol Hydrocortisone