Safety and Efficacy Study in Infant With SBS
Short Bowel SyndromeThe objective of this clinical study is to evaluate the efficacy and safety of NTRA-9620 compared with placebo in pediatric subjects (aged 28 weeks postmenstrual age to 52 weeks old) with SBS following surgical resection
Controlled Study of Rigosertib Versus Physician's Choice of Treatment in MDS Patients After Failure...
Myelodysplastic SyndromeMDS2 moreThe study's primary objective [in a population of patients with MDS after failure of treatment with azacitidine (AZA) or decitabine (DAC)], is to compare the overall survival (OS) of patients in the rigosertib group vs the Physician's Choice group, in all patients and in a subgroup of patients with IPSS-R very high risk.
Evaluation of the Efficacy and Safety of Lubiprostone in Adults With Mixed or Unsubtyped Irritable...
Irritable Bowel SyndromeA study of the efficacy and safety of Lubiprostone in subjects diagnosed with IBS-M/IBS-U.
Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus...
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus SyndromeThis is an open label, Phase 1/2 study of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase, catalytic subunit alpha (PIK3CA)-related Overgrowth Spectrum (PROS) and Proteus Syndrome (PS) (MOSAIC).
Hyperbaric Oxygen Therapy Effect on Post Concussion Syndrome in Children
Hyperbaric Oxygen TherapyTraumatic Brain Injury1 moreDue its high incidence, mTBI and its consequences of PPCS are a major public health issue. There is no consensus regarding the treatment of PPCS in pediatrics. Relying on its results in adults, HBOT offers a promising new direction of treatment, which targets the basic pathological processes responsible for post-concussion symptoms. The effect of hyperbaric oxygen therapy in pediatric TBI has never been evaluated. The aim of the current study is to evaluate in a prospective cross-over, randomized study, the effect of HBOT on children with PPCS due to mild TBI.
Botulinum for Chronic Exertional Compartment Syndrome
Chronic Exertional Compartment SyndromeWe will investigate the feasibility of a simple outpatient one time injection regimen for the treatment of Chronic Exertional Compartment Syndrome (CECS). We think botulinum toxin injections will be a potentially cost-effective, low-risk alternative to surgery in reducing pain and returning patients to full activity.
Deferasirox in Treating Patients With Very Low, Low, or Intermediate-Risk Red Blood Cell Transfusion...
AnemiaMyelodysplastic SyndromeThis phase II trial studies how well deferasirox works in treating patients with very low, low, or intermediate-risk anemia or myelodysplastic syndrome that depends on red blood cell transfusions. Deferasirox may treat too much iron in the blood caused by blood transfusions.
Efficacy and Safety of Roxadustat for Treatment of Anemia in Participants With Lower Risk Myelodysplastic...
Primary MDS (Very LowLow or Intermediate IPSS-R With <5% Blasts)The purpose of this study is to determine whether FG-4592 is safe and effective in the treatment of anemia in participants with lower risk MDS and low red blood cell transfusion burden.
Irradiated Donor Cells Following Stem Cell Transplant in Controlling Cancer in Patients With Hematologic...
Acute Lymphoblastic LeukemiaAcute Myeloid Leukemia in Remission17 moreThis pilot clinical trial studies the side effects of irradiated donor cells following stem cell transplant in controlling cancer in patients with hematologic malignancies. Transfusion of irradiated donor cells (immune cells) from relatives may cause the patient's cancer to decrease in size and may help control cancer in patients receiving a stem cell transplant.
Melatonin Treatment for Night-Eating Syndrome
Night-eating SyndromeThis is an 8-week randomized, double-blind, placebo-controlled, parallel, fixed-dose pilot clinical trial to test the effectiveness of melatonin treatment versus placebo on symptoms of Night Eating Syndrome (NES). The investigators will also look at the effect of melatonin treatment on weight, metabolic parameters and sleep parameters in individuals with NES.