About 21,843 results

Mitochondrial Complex I Dysfunction in PWS

Aug 5th, 2022 - Investigators will determine the clinical efficacy of CoQ10 in a prospective, randomized, double-blind, controlled, cross-over study. Primary outcome will include determination of muscle function based on a 3 minute step test which showed good sensitivity in the investigators' pilot study. Secondary outcomes will determine effects on muscle power (maximal vertical jump), strength (hand grip), e...

"Overlap Syndrome and PSC: Evaluating Role of Gut Microflora and Its Identification With Antibiotics in Children"

Aug 5th, 2022 - The used antibiotic is Metronidazole (Flagyl), during 14 days, as induction therapy or rescue therapy. Study participants will provide blood and stool samples in order to evaluate bile acids profile and microbiome, before and after the course of metronidazole, and then comparison will be made pre- and post-antibiotics. The investigators will determine the benefit of oral metronidazole therapy t...

Self Administered Cognitive Behavior Therapy for Irritable Bowel Syndrome

Aug 5th, 2022 - Irritable bowel syndrome (IBS) is a chronic, prevalent, often disabling, GI disorder for which there is no reliable and satisfactory medical option for its full range of symptoms (abdominal pain, bowel dysfunction). An accumulating body of evidence indicates that a specific psychosocial treatment called cognitive behavioral therapy (CBT) is associated with significant reductions in IBS symptoms...

A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia

Aug 5th, 2022 - PRIMARY OBJECTIVES: I. To determine in a randomized manner if the addition of 2 cycles of blinatumomab to standard therapy improves disease-free survival (DFS) in patients with standard risk (SR) B-ALL and higher risk features (SR-High), and patients with standard-risk average (SR-Avg) B-ALL who are negative for minimal residual disease (MRD) by flow cytometry but have detectable or indetermina...

A Study of Soticlestat as an Add-on Therapy in Children, Teenagers, and Adults With Lennox-Gastaut Syndrome

Aug 5th, 2022 - The drug being tested in this study is called soticlestat (TAK-935). Soticlestat will be assessed for efficacy, safety, and tolerability in pediatric and adult participants with LGS. The study will enroll approximately 234 patients. Participants will be randomly assigned (by chance, like flipping a coin) in a 1:1 ratio to receive standard of care (SOC) plus one of the following adjunctive thera...

A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome

Aug 5th, 2022 - The drug being tested in this study is called soticlestat (TAK-935). Soticlestat as an adjunctive therapy will be assessed for efficacy, safety, and tolerability in pediatric and adult participants with DS. The study will enroll approximately 142 pediatric and young adult patients. Participants will be randomized at a 1:1 ratio to receive standard of care (SOC) plus one of the following adjunct...

Duvelisib and Nivolumab for the Treatment of Stage IIB-IVB Mycosis Fungoides and Sezary Syndrome

Aug 5th, 2022 - PRIMARY OBJECTIVE: I. To determine the recommended phase II dose (RP2D) or maximum tolerated dose (MTD) of the combination of duvelisib with nivolumab in patients with advanced mycosis fungoides/Sezary syndrome (MF/SS). SECONDARY OBJECTIVES: I. To observe and record anti-tumor activity. Ia. To determine the overall response rate at four months to the combination of nivolumab and duvelisib. Ib. ...

Addressing Post-Intensive Care Syndrome Among Survivors of COVID (APICS-COVID)

Aug 5th, 2022 - Despite the accumulation of data documenting the reality of extensive functional impairments following ICU stay, the specific treatment needs of individuals experiencing Post-Intensive Care Syndrome (PICS) are not well known largely because of a relative lack of knowledge about the specific unmet needs of patients at risk for PICS at the time of hospital discharge. These possible needs include ...

IFN-γ to Treat Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) That Has Relapsed After Allogeneic Hematopoietic Stem Cell Transplantation

Aug 5th, 2022 - Allogeneic hematopoietic stem cell transplantation (alloSCT) can cure patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). However, relapsed AML/MDS is the most significant single cause of treatment failure, and the majority of relapsed patients ultimately succumb. Alloreactive T cells in the donor graft can kill residual leukemia cells, mediating the graft-vs-leukemia...

A Study to Evaluate VIP152 in Subjects With Relapsed/Refractory Chronic Lymphocytic Leukemia or Richter Syndrome

Aug 5th, 2022 - Determine the safety, tolerability, pharmacokinetics, maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of VIP152 in patients with Chronic Lymphocytic Leukemia (CLL) or Richter Syndrome

Effectiveness of Major Ozone Autohemotherapy in the Treatment of Fibromyalgia Syndrome

Aug 5th, 2022 - Patients who were admitted to the Physical Medicine and Rehabilitation (PMR) outpatient clinic of our hospital between January 2017 and December 2020 and were treated with Major ozone autohemotherapy with a diagnosis of Fibromyalgia syndrome will include to the study. Visual anolog scale (VAS), Fibromyalgia Impact Questionnare (FIQ) and Short Form Health Survey-36 (SF-36) scores of the patients...

A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome

Aug 5th, 2022 - Background: In 2019, investigators at the National Institutes of Health defined a new disease syndrome named VEXAS: Vacuoles in bone marrow cells, E1 enzyme mutations, X-linked, Autoinflammatory, Somatic syndrome. This syndrome is characterized by inflammatory and hematologic features and is frequently accompanied by marrow dysplasia, progressive bone marrow failure, and in some cases, the deve...

Effect of Dalcetrapib vs Placebo on CV Risk in a Genetically Defined Population With a Recent ACS

Aug 5th, 2022 - This is an event driven study to reach statistical power given all other assumptions. Subjects will visit the clinic 1 month after randomization and at regular intervals thereafter. Additionally, for any subject prematurely discontinuing study medication, assessments will be conducted every 6 months for the collection of study endpoints. Those who are likely to qualify will undergo Genotype Ass...

MK-7075 (Miransertib) in Proteus Syndrome

Aug 5th, 2022 - Study Description: The primary objective of this study is to determine the response rate of miransertib as measured by the change in CCTN involvement of the plantar surface from baseline, using blinded independent central review of lesional photography in individuals with Proteus syndrome (Cohort 1). Cohorts 2 and 3 will enroll up to 30 additional patients whose non-plantar CCTN Proteus syndrom...

Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial

Aug 5th, 2022 - Background: RASopathies are a group of neurodevelopmental genetic conditions caused by mutations affecting components within the RAS-map kinase (RAS-MAPK) cellular signaling pathway. Caregivers of children with a RASopathy are faced with numerous challenges related to the physical, cognitive, and behavioral symptoms associated with their child s condition. The use of virtual interventions has b...

Effect of Dietary Fiber Intervention on Patients With Polycystic Ovary Syndrome

Aug 5th, 2022 - The purpose of the study is to understand the effect of dietary fiber on patients with polycystic ovary syndrome.

Intensive Anti-Scoliosis Postural Intervention for Individuals With Rett Syndrome Supported by a Smartphone Application

Aug 5th, 2022 - Study Aims The current study aims to evaluate the effectiveness of an anti-scoliotic physiotherapy program carried out during daily life and focuses on hypercorrective postures and increasing the level of activity to assess whether or not it is possible to slow the progression of scoliosis in girls with Rett syndrome (RTT). The current research project has the following research objectives: Pri...

Performance and Safety Evaluation of MD-Shoulder Collagen Medical Device in the Treatment of Rotator Cuff Syndrome.

Aug 5th, 2022 - This is a pilot monocentric Clinical Investigation based on a one sample design. The purpose of this research is to evaluate, through the CMS functional scale, the performance of intra-articular treatment with a collagen-based medical device in recovering joint function and reducing associated pain. The safety of the treatment will also be assessed. Variables will be assessed at 6 different tim...

Mediterranean Diet- and Psychological Well-being Theory-based Intervention to Reverse Metabolic Syndrome in Chile

Aug 5th, 2022 - BACKGROUND: Non-communicable diseases (NCDs) are currently major causes of morbidity and mortality as well as economic healthcare burden worldwide, including Chile. Predisposing psychosocial as well as lifestyle risk factors -such as an unhealthy diet- have driven this global increase in NCDs. Overall, the ongoing epidemiological transition and current health situation in our country calls out ...

Evaluation of the Efficacy of the Use MD Tissue Collagen Medical Device in the Infiltrative Treatment of Greater Trochanter Pain Syndrome (GTPS)

Aug 5th, 2022 - This is a pilot monocentric Clinical Investigation based on a One sample design. In the ex vivo study, MD-Tissue Collagen Medical Device was used as a substrate for cell cultures of human gluteal tenocytes on culture plates. The results suggest how MD-Tissue can induce an anabolic phenotype in tenocytes by stimulating their proliferation and migration; it would also be able to promote the synth...