About 9,735 results

A Phase I Pilot Study of Abaloparatide + Bevacizumab in Myelodysplastic Syndromes

Aug 12th, 2022 - The main objective of this study is to determine the safety and tolerability of combined abaloparatide + bevacizumab in MDS patients. This will be a single center, single arm, phase 1 trial in MDS patients. In cycle 1, patients will be treated with single-agent, subcutaneous (SQ) abaloparatide at a dose of 80 mcg/day for 28 days. This will enable the investigator to monitor for any potential to...

Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation

Aug 12th, 2022 - Background: Disorders of T-cell proliferation and/or dysregulation (TCP/D) can lead to T-cell lymphoproliferative disorders, autoimmunity, infection, and aberrant immune activation with resulting organ dysfunction, morbidity, and mortality. Allogeneic hematopoietic cell transplantation (HCT) has the potential to cure disorders of TCP/D. Subjects with TCP/D may be at higher risk for graft reject...

Defining the Brain Phenotype of Children With Williams Syndrome

Aug 12th, 2022 - Williams syndrome (WS) is a rare disorder caused by hemizygous microdeletion of approximately 1.6 megabases on chromosomal band 7q11.23, typically by spontaneous mutation. The disorder is characterized by a collection of unique neuropsychiatric manifestations, including marked visuospatial construction deficits and hypersociability. Because the genes involved in WS are known, the study of neura...

Androgen Receptor, Implications for Health and Wellbeing: Natural History Study of Individuals With Androgen Insensitivity

Aug 12th, 2022 - Androgen effects in humans are usually (but not always) mediated by the androgen receptor which is coded for by the androgen receptor gene (AR gene). Individuals with abnormalities of this receptor gene can present with androgen insensitivity syndrome (AIS). There are a variety of phenotypes including complete female phenotype (complete androgen insensitivity or CAIS), ambiguous genitalia in ca...

Study in Pediatrics With HypEREosinophilic Syndrome (SPHERE)

Aug 12th, 2022 - The purpose of this study is to investigate the efficacy and safety of mepolizumab in children and adolescents with hypereosinophilic syndrome (HES) who are receiving standard of care (SoC) therapy.

The Pathogenesis and Natural History of Sjogren s Syndrome

Aug 12th, 2022 - BACKGROUND Sj(SqrRoot)(Delta)gren s syndrome (SS) is an autoimmune disease characterized by chronic inflammation involving the exocrine glands. Salivary and lacrimal glands are predominantly affected leading to dry mouth and dry eyes but other exocrine organs are also frequently involved. It is one of the most common rheumatic autoimmune diseases, which effects between 1-4 million Americans, pr...

PT2385 for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma

Aug 12th, 2022 - This open-label Phase 2 study will evaluate the efficacy, safety, PK, and PD of PT2385 in patients with VHL disease who have at least 1 measurable VHL disease-associated ccRCC tumor (as defined by RECIST 1.1). PT2385 will be administered orally and treatment will be continuous. Changes in VHL disease-associated non-ccRCC tumors will also be evaluated.

A Study of ARGX-110 in Combination With Azacytidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) or High Risk Myelodysplatic Syndrome (MDS)

Aug 12th, 2022 - The purpose of this study is to determine the maximum tolerated dose (MTD) of ARGX-110 and/or the recommended Phase II dose (RP2D) in combination with a standard dose of azacytidine (AZA) in Phase 1; and to evaluate efficacy of ARGX-110 when administered at a RP2D level established in Phase I in combination with a standard dose of AZA (proof-of concept) by evaluating overall response rate (ORR)...

Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome: A Phase IIb Clinical Trial.

Aug 12th, 2022 - The purpose of this study is to test whether Lomecel-B™ works in treating patients with hypoplastic left heart syndrome (HLHS) and to gather additional information about the safety of Lomecel-B. Lomecel-B contains human mesenchymal stem cells (MSCs) as the active ingredient. MSCs are special cells in the body that are able to change into other types of cells, such as heart, blood, and muscle ce...

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

Aug 12th, 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...

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

Aug 12th, 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...

Predictors of Pregnancy Outcome in Systemic Lupus Erythematosus (SLE) and Antiphospholipid Syndrome (APS)

Aug 12th, 2022 - Thrombosis and pregnancy loss are common features of systemic lupus erythematosus (SLE), particularly in the presence of antiphospholipid (aPL) antibodies. The in vivo mechanisms by which aPL antibodies lead to vascular events and, specifically, to recurrent fetal loss are largely unknown. Studies in a mouse model of antiphospholipid antibody syndrome (APS) indicate that in vivo complement acti...

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

Aug 12th, 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...

Natural History and Biospecimen Acquisition for Myelodysplastic Syndromes

Aug 12th, 2022 - Background: Myelodysplastic syndromes (MDS) are heterogenous stem cell disorders characterized by ineffective hematopoiesis resulting in cellular dysplasia, peripheral cytopenias, and increased risk for transformation to acute myeloid leukemia (AML). There are limited treatments options, all of which have unimpressive response rates and limited response durations, with the only potential cure b...

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

Aug 12th, 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...

Study of BOTOX Injections to Assess Change in Disease Activity and Adverse Events in Adult Female Participants With Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS)

Aug 12th, 2022 - Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS) is a chronic and debilitating urological complex of disorders characterized by symptoms of bladder pain or discomfort, mostly upon bladder filling, and often accompanied by lower urinary tract symptoms (LUTS). This study will assess how safe and effective BOTOX (onabotulinumtoxinA) is in treating IC/BPS. Adverse events and change in disease s...

BMS-986253 in Myelodysplastic Syndromes

Aug 12th, 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...

Study of Cariprazine Oral Capsules or Solution to Assess Adverse Events and Change in Irritability Due to Autism Spectrum Disorder (ASD) in Participants Aged 5-17 Years With ASD

Aug 12th, 2022 - Autism spectrum disorder (ASD) consists of deficits in social, communication, and cognitive development, repetitive and stereotypic behaviors. Many ASD patients show notably high levels of irritability, including verbal and physical aggression, self injury, and/or property destruction. Autistic infants tend to avoid eye contact and show little interest in others. This study will assess how safe...

Brain Imaging of Childhood Onset Psychiatric Disorders, Endocrine Disorders and Healthy Volunteers

Aug 12th, 2022 - Objective: Our work is driven by the core hypotheses that many of the most severe neuropsychiatric disorders of childhood onset are associated with deviations from the path of normal brain development, the neuroanatomical substrates of which can be detected by magnetic resonance imaging. Consequently, the long-term goals of the protocol are to: (1) map neuroanatomic and neurophysiological traje...

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

Aug 12th, 2022 - Study Description: This study will allow for the long term follow up of patients with acquired and inherited bone marrow failure, both treated and untreated. Objectives: Primary Objective The primary objective is to characterize disease and treatment related long-term outcomes in subjects with inherited or acquired marrow failure. Secondary Objectives Rates of disease progression requiring inte...