Furosemide Alone or Unexpectedly With Solution of Hypertonic Saline in Nephrotic Syndrome
Nephrotic SyndromeThe trial will test the hypothesis that hypertonic saline on top of standard diuretic treatment will help achieve adequate diuresis in patients with nephrotic syndrome.
A Trial of Metformin in Individuals With Fragile X Syndrome (Met)
Fragile X SyndromeFragile X Mental Retardation Syndrome10 moreThis study is a controlled trial of metformin in individuals with fragile X syndrome between the ages of 6 and 35 years. Participants will be randomized in a double-blind design to either drug or placebo and will attend three visits to the study site in a 4-month period for a series of tests. The primary objectives are to assess safety, tolerability, and efficacy of metformin in the treatment of language deficits, behavior problems, and obesity/excessive appetite in individuals with fragile X syndrome.
Conditioning Electrical Stimulation to Improve Outcomes in Carpal Tunnel Syndrome
Carpal Tunnel SyndromeCarpal tunnel syndrome is common, identified in 3% of the general population. Symptoms including numbness and pain are due to compression of the median nerve as it travels through a tunnel entering the wrist and can result in weakened grip strength and poor dexterity. Despite surgical release, nerve damage due to chronic compression often cannot be completely reversed, with resulting sensorimotor deficits. Postoperative electrical stimulation (ES) has been well-reported to improve nerve regeneration and is currently standard of practice at our institution. Investigators of this study have recently shown in an animal model that by changing the timing of the ES from postoperative to preoperative, this "conditioning" electrical stimulation (CES) significantly improves nerve regeneration. Patients with severe carpal tunnel syndrome will be identified in plastic surgery clinics. Patients who consent to participating will undergo baseline testing including nerve conduction studies, sensory evaluation, motor testing, and patient-reported outcomes. Participants will be randomized to three groups: i) CES, ii) postoperative ES, and iii) no ES. CES will be delivered in clinic by placing a percutaneous needle alongside the median nerve, and stimulation will be delivered for one hour, with patient comfort dictating the voltage of stimulation. At the completion of one hour, the needle will be removed, and a standard carpal tunnel release will be performed by their plastic surgeon 4-7 days later. Patients will the second cohort will undergo postoperative ES immediately following their carpal tunnel release, using the same stimulation parameters as CES. The third cohort will receive only carpal tunnel release without stimulation.In all patients, sensory and motor reinnervation, using the same testing modalities as preoperative assessment, will be evaluated at 3, 6, and 12 months post-operative.
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).
Venetoclax and Azacitidine for the Treatment of High-Risk Recurrent or Refractory Myelodysplastic...
Chronic Myelomonocytic LeukemiaMyelodysplastic Syndrome3 moreThis phase I/II trial studies the side effects and best dose of venetoclax when given together with azacitidine in treating patients with high-risk myelodysplastic syndrome that has come back (recurrent) or does not respond to treatment (refractory). Drugs used in chemotherapy, such as venetoclax and azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
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).
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.
Safety and Tolerability Study of INCB057643 in Participants With Myelofibrosis and Other Advanced...
MyelofibrosisMyelodysplastic Syndrome5 moreThe purpose of this study is to evaluate the safety, tolerability, and preliminary efficacy of INCB057643 as monotherapy or combination with ruxolitinib for participants with myelofibrosis and other myeloid neoplasms.
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.