
CPX-351 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic...
Adult Acute Erythroid Leukemia (M6)Adult Acute Megakaryoblastic Leukemia (M7)14 moreThis phase 2 clinical trial studies how well CPX-351 (liposomal cytarabine-daunorubicin) works in treating patients with relapsed or refractory acute myeloid leukemia or myelodysplastic syndrome. Drugs used in chemotherapy, such as CPX-351, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.

Safety and Pharmacokinetics Study of SyB C-1101 in Patients With Recurrent/Relapsed or Refractory...
Myelodysplastic SyndromeThe purpose of this study is to investigate tolerability when SyB C-1101 is orally administered twice daily for 14 consecutive days to the patients with recurrent/relapsed or refractory myelodysplastic syndrome, to determine the dose-limiting toxicity and maximum tolerated dose, and to estimate the recommended dose for phase II studies. Pharmacokinetics and antitumor effects will also be investigated.

Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral Cytidine...
Myelodysplastic SyndromeMDSThis first-in-human, 3-stage, open-label study evaluated the safety and pharmacokinetics of ASTX727, as well as determined the dose for later stages.

Study of BMS-833923 in Two Specific Patients With Basal Cell Nevus Syndrome
Basal Cell Nevus SyndromeThis is an extension study of Protocol CA194002 to allow 2 specific participants with basal cell nevus syndrome in the CA194002 study at Princess Margaret Cancer Centre who are still benefitting from the study drug BMS-833923 to continue receiving the study drug. This study will continue to evaluate the safety and tolerability of BMS-833923 in these participants.

Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome
Angelman SyndromeRANDOMIZED CLINICAL TRIAL, PLACEBO COMPARED TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY)

Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
Alagille SyndromeThe study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.

Subcutaneous Peripheral Nerve Stimulation (PNS) as "Hybrid Stimulation" After Failure of Spinal...
Failed Back Surgery SyndromeDespite globally favourable outcomes of Spinal Cord Stimulation (SCS), a significant proportion of Failed Back Surgery Syndrome (FBSS) patients do not obtain adequate coverage of low back pain. Peripheral Nerve Stimulation (PNS) has obtained the European Conformity mark for the treatment of chronic refractory neuropathic pain and is now commonly used in some countries to target back pain. However, the potential value of combining SCS and PNS as "hybrid stimulation" remains poorly described with only isolated case reports or limited experience in various indications. The "CUMPNS" comparative randomized study is designed to demonstrate the potential analgesic efficacy of PNS in addition to previously implanted SCS, to treat the residual low back pain component pain in refractory FBSS patients.

Biopsychological Intervention in Chronic Fatigue Syndrome - a Pilot Study
Adolescent Chronic Fatigue SyndromeThis is a pilot study of a biopsychological intervention program for adolescent chronic fatigue syndrome. The program is related to cognitive behavioral therapy, which has been proven beneficial in this disorders, but includes other mental techniques, such as emotional control and metacognitive elaboration. The aim of this pilot study is to explore a) patients' experiences and b) possible positive effects on symptoms. We hypothesise that the intervention will be regarded feasible by the patients, and that fatigue score will improve during the intervention period.

Phase II Study Evaluating Efficacy, Safety and Pharmacokinetics of Pasireotide in Patients With...
Dumping Syndromemulti-center, phase II study evaluating efficacy, safety and pharmacokinetics of pasireotide in patients with dumping syndrome

Ad Hoc Percutaneous Coronary Intervention Study in Acute Coronary Syndrome Patients
Acute Coronary SyndromeThe purpose of this study is to assess the pharmacodynamic effect of ticagrelor in ACS patients undergoing an Ad Hoc PCI