About 20,700 results

Seclidemstat and Azacitidine for the Treatment of Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

Jan 21st, 2022 - PRIMARY OBJECTIVES: I. To determine the safety, tolerability and maximum tolerable dose (MTD) of seclidemstat in combination with azacitidine. II. To assess overall response rate (ORR) to seclidemstat in combination with azacitidine. SECONDARY OBJECTIVES: I. To assess overall survival (OS), duration of response (DOR), relapse-free survival (RFS), and leukemia-free survival (LFS) and safety prof...

Clinical, Genetic, and Epidemiologic Study of Children and Adults With RASopathies

Jan 21st, 2022 - Study Description: The RASopathies are a clinically defined group of disorders caused by pathogenic germline variants in genes encoding components of the Ras/mitogen-activated-protein kinase (Ras/MAPK) pathway. These disorders have overlapping clinical features due to Ras/MAPK dysfunction, including a predisposition to the development of certain malignancies. The aims of this prospective l...

Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy

Jan 21st, 2022 - The study is designed as a multicenter, single-arm, open label study to demonstrate the efficacy and safety of LNP023 (iptacopan) at a dose of 200 mg b.i.d. in adult patients with aHUS who are treatment naive to complement inhibitor therapy (including anti-C5 antibody). The study will enroll approximately 50 participants and assess the effects of iptacopan on a range of efficacy assessments rel...

Revlimid in Transfusion Dependent Patients

Jan 21st, 2022 - The first Revlimid® study reported by List et al included all transfusion dependent MDS patients, but given the early experience which showed a better response in del(5q) patients and those with lower risk disease, more patients belonging to this category were entered on the trial. The subsequent multi-center studies were focused entirely on the low/Int-1 patients who were transfusion dependent...

A Study of Brexanolone for Acute Respiratory Distress Syndrome Due to COVID-19

Jan 21st, 2022 - The purpose of this study is to evaluate the efficacy and safety of brexanolone in participants on ventilator support for acute respiratory distress syndrome (ARDS) due to COVID-19.

Efficacy and Safety of Plecanatide in Children 6 to <18 Years of Age With Irritable Bowel Syndrome With Constipation (IBS-C)

Jan 21st, 2022 - The purpose of this study is to evaluate the safety, efficacy and pharmacokinetic (PK) parameters of plecanatide of once daily oral 4 dose levels of plecanatide (0.5, 1.0, 2.0, or 3.0 mg) for 4 weeks as treatment in children 6 to <18 years of age with IBS-C. The study will include a 28-day Screening/Baseline Period, a 4 week treatment period, and a 2-week post-treatment follow-up period. Patien...

The Effect of Theraworx Foam in Carpal Tunnel Syndrome

Jan 21st, 2022 - The PI will identify potential participants from his clinical practice. Evaluation of symptoms will be by exam, questionnaires, and ultrasound of the median nerve. These will document baseline/change in the size of the median nerve and improvement in function/symptoms. The questionnaires are 1.) Boston Carpal Tunnel (BCT). 2.) Carpal Tunnel-6 (CTS-6); and 3.) Disabilities of the Arm, Shoulder a...

Three-dimensional Analysis of EMMPRIN on Conjunctival Epithelial Cells Surface in Severe Dry Eye Syndrome (ALTESSE)

Jan 21st, 2022 - Modifications of cell surface markers (including EMMPRIN) were observed in conjunctival epithelial cells during dry eye syndrome ; this study aims to describe the modifications of the repartition of these cell surface markers before and after initiation of a treatment.

Botox for the Treatment of Recurrent Chronic Exertional Compartment Syndrome

Jan 21st, 2022 - Participants will be enrolled in study at initial clinic visit by study staff at which time diagnosis of R-CECS will be established based on elevated pressure measurements in patients who have had surgical release of the affected compartments. Pressure testing will be performed outside of the clinical study and will be required to establish the diagnosis of R-CECS. Initial clinic visit with stu...

ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Jan 21st, 2022 - PRIMARY OBJECTIVES: I. To establish the maximum tolerated dose (MTD) of the combination of decitabine and cedazuridine (ASTX727), venetoclax and gilteritinib in patients with relapsed/refractory FLT3- mutated acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). (Phase I) II. To determine the complete response (CR)/incomplete hematologic recovery (CRi) rate of the regimen in...

ARQ 092 (Miransertib) in Proteus Syndrome

Jan 21st, 2022 - Proteus syndrome is a rare mosaic overgrowth disorder caused by a somatic gain of function variant, c. 49G>A, p.(Glu17Lys) in the oncogene AKT1, encoding the AKT1 kinase. The disorder is progressive, with high morbidity and mortality - there are very few living adults with this disease. Tissues and cell lines from patients with Proteus syndrome harbor admixtures of mutant alleles that ranged fr...

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

Jan 21st, 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...

A Phase 2/3 Study of RAD011 (Cannabidiol Oral Solution) for the Treatment of Patients With Prader-Willi Syndrome

Jan 21st, 2022 - This is a seamless Phase 2/3, double-blind, randomized, placebo-controlled clinical study in patients diagnosed with PWS. Following consent (or legal guardian consent and patient assent as appropriate), patients will be screened for eligibility to participate in this study.

Study of Efficacy and Safety of MBG453 in Combination With Azacitidine in Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic ...

Jan 21st, 2022 - This is a Phase III multi-center, randomized, two-arm parallel-group, double-blind, placebo controlled study of MBG453 or placebo added to azacitidine in adult subjects with intermediate, high or very high risk myelodysplastic syndrome (MDS) as per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2). The primary objective of this study is to compare overall survival (OS) in the MBG453 plus az...

BMS-986253 in Myelodysplastic Syndromes

Jan 21st, 2022 - Background: The myelodysplastic syndromes (MDS) are a group of clonal bone marrow neoplasms characterized by ineffective hematopoiesis, cytopenia, and high risk for transformation to acute myeloid leukemia (AML). MDS is primarily a disease of the elderly, with about 80% of participants being older than 65-years of age; with 10,000 new diagnoses per year in the U.S. The only curative treatment f...

Assessment of Hypotonia in Children With Down Syndrome

Jan 21st, 2022 - Hypotonia is usually diagnosed by observation and clinical assessment. Aiming to develop an instrument for early diagnosis, a systematic review was carried out which compiled characteristics and testing that are attributed to or used in the diagnosis of hypotonia. They were presented as an item group and a first stage was created and administered to 50 children with Down's Syndrome between the ...

Predictive Value of Induced Sleep Endoscopy on Surgical Indication in Obstructive Sleep Apnea Syndromes

Jan 21st, 2022 - Obstructive sleep apnea syndrome (OSA) is a pathology that affects 2 to 15% of the French adult population and more than 30% of subjects over 65 years old. It consists of repeated collapses of the upper airways during sleep leading to interruptions in ventilation (apneas) or significant reductions in ventilation (hypopneas). Balagny et al. have demonstrated the occurrence of hypertension in pat...

AutoInflammatory Disease Alliance Registry (AIDA)

Jan 21st, 2022 - The AIDA registry service is based on REDCap (Research Electronic Data Capture, project-redcap.org), a secure web application for building and managing online surveys and databases, designed to support data capture for research studies. The platform is directly accessible through the AIDA website, after inserting a personal username and password. Currently, 9 registries are available, each one ...

Efficacy of Empagliflozin or Linagliptin as an Alternative to Metformin for Treatment of Polycystic Ovary Syndrome

Jan 21st, 2022 - Patients with polycystic ovary syndrome will be randomized to three arms: Metformin (standard care) Empagliflozin or Linagliptin Resolution of the syndrome in addition to normalization of sex hormones, metabolic and inflammatory parameters will be tested

Eltrombopag in Myelodysplastic Syndrome (MDS) Patients With Thrombocytopenia

Jan 21st, 2022 - Study was originally designed as a Phase I/II but was terminated during the Phase I portion due to lack of efficacy