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📍 Uni of Alabama at Birmingham · Birmingham, AL · +39 more US sites · Get directions →

This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo.

📍 Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's Hospital · Phoenix, AZ · +37 more US sites · Get directions →

In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have not yet demonstrated long-term safety and effectiveness beyond the pivotal trials that led to their approval.

📍 Univ of Alabama Birmingham · Birmingham, AL · +38 more US sites · Get directions →

Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.

📍 University of Alabama - Huntsville Regional Medical Campus /ID# 267818 · Huntsville, AL · +39 more US sites · Get directions →

Generalized anxiety disorder (GAD) is usually treated with antidepressant therapy (ADT); however, sometimes ADTs alone are not enough to adequately treat GAD. The purpose of this study is to assess how safe and effective ABBV-932 is when added to the antidepressant therapies in adult participants with GAD who have had an inadequate response ADTs.

📍 Children's of Alabama (MRD-HCT) · Birmingham, AL · +30 more US sites · Get directions →

The WeDecide study is a large observational study comparing the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) for pediatric patients with sickle cell disease (SCD). The study aims to assess health-related quality of life (HRQoL), cognitive function, risks, and benefits of both treatments, including survival rates, chronic complications, and organ damage prevention.

📍 Vanda Investigational Site · Phoenix, AZ · +30 more US sites · Get directions →

This is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of a single oral dose of VQW-765 compared to placebo in adults with social anxiety disorder.

📍 Clinical Site · Birmingham, AL · +29 more US sites · Get directions →

This is a multicenter, randomized, double-blind, placebo-controlled study to assess the efficacy, safety, and tolerability of ITI-1284 as monotherapy treatment in patients meeting Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, Text Revision (DSM-5-TR) criteria for GAD in patients who have had inadequate response to generalized anxiety disorder treatment.

📍 University of Alabama at Birmingham · Birmingham, AL · +16 more US sites · Get directions →

This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing Study BTX-AUT-001.

📍 Clinical Site · Chandler, AZ · +33 more US sites · Get directions →

This is a multicenter, randomized, double-blind, placebo-controlled study evaluating the efficacy, safety, and tolerability of ITI-1284 compared with placebo as adjunctive therapy to GAD treatment in patients meeting Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, Text Revision (DSM-5-TR) criteria for GAD who have an inadequate response to ongoing GAD treatment.

📍 University of Alabama at Birmingham- Site Number : 8400003 · Birmingham, AL · +15 more US sites · Get directions →

This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study details include: * Study duration: a 52-week double-blind period (Part A), followed by an open-label LTE period (Part B).

📍 University of Alabama at Birmingham · Birmingham, AL · +18 more US sites · Get directions →

The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.

📍 Lighthouse Psychiatry · Gilbert, AZ · +21 more US sites · Get directions →

The purpose of this study is to determine if treatment with a single dose of RE104 for Injection reduces anxiety symptoms in participants with Generalized Anxiety Disorder (GAD) as compared to placebo.

📍 University Of Alabama · Birmingham, AL · +13 more US sites · Get directions →

A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.

📍 University of South Alabama Medical Center · Mobile, AL · +13 more US sites · Get directions →

The study, known as the Peer suppoRt for adolescents and Emerging adults with Sickle cell pain: promoting ENgagement in Cognitive behavioral thErapy (PRESENCE), aims to determine the effectiveness of digital CBT in reducing pain, opioid use, and healthcare utilization among AYAs with SCD. It also seeks to understand the role of personalized peer support in enhancing engagement and outcomes of digital CBT interventions.

📍 Smilow Cancer Hospital · New Haven, CT · +23 more US sites · Get directions →

The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.

📍 Univ. of California, San Francisco Health Care · Oakland, CA · +15 more US sites · Get directions →

This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to evaluate the safety and tolerability of CSL889 in study participants, and to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD.

📍 University of Alabama, Birmingham (UAB) · Birmingham, AL · +8 more US sites · Get directions →

The goal of this observational study is to help us understand more about the best ways to help individuals living with Sickle Cell Disease (SCD) get the best care. The main question it aims to answer is: How to find individuals unaffiliated from SCD specialist care use three distinct pathways?

📍 University of Alabama · Birmingham, AL · +9 more US sites · Get directions →

The main purpose of this study is to create a longitudinal cohort of those with Sickle Cell Trait (SCT) to better understand the hematologic phenotype for those that carry HbS, assess for differences in those with varying quantities of HbS and assess for potential clinical complications of SCT.

📍 Vistagen Clinical Site · Sherman Oaks, CA · +8 more US sites · Get directions →

This U.S. multicenter, double-blind, placebo-controlled Phase 2 clinical trial is designed to evaluate the efficacy, safety, and tolerability of a repeat intranasal (i.n.) dose of Fasedienol Nasal Spray (fasedienol) (3.2 µg) to relieve symptoms of acute anxiety in adult subjects ages 18 through 65 with Social Anxiety Disorder induced by a public speaking challenge (PSC) in a clinical setting.

📍 SOAR Clinical Study Site · Encino, CA · +10 more US sites · Get directions →

The primary goal of this Phase 2 clinical trial is to determine the effects of a novel V1a receptor antagonist (NTX-1472) in adults with Social Anxiety Disorder (SAD). The main questions this trial aims to answer are: * Is NTX-1472 safe and well tolerated in adults with SAD?

📍 University Of Alabama · Birmingham, AL · +6 more US sites · Get directions →

This is a multi-center multi-national rollover study to allow continued access to crizanlizumab for patients with sickle cell disease (SCD) who are on crizanlizumab treatment in a Novartis-sponsored study (parent study) and are benefiting from the treatment as judged by the investigator.

📍 University of Alabama at Birmingham · Birmingham, AL · +6 more US sites · Get directions →

This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.

📍 University of Alabama Birmingham · Birmingham, AL · +5 more US sites · Get directions →

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).

📍 Children's National Health System · Washington D.C., DC · +4 more US sites · Get directions →

This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).

📍 UCSF Benioff Children's Hospital Oakland · Oakland, CA · +5 more US sites · Get directions →

The goal of this short term prospective Phase II study is to compare the effects of two alternate daily doses of zinc (25 and 40 mg/day) in 34 randomly assigned homozygous Sickle Cell Disease (SCD-SS) patients aged 15-40 years old. The main question it aims to answer is: Which biomarkers are most responsive to zinc supplementation, and what is the maximum tolerated zinc dose that induces the desired changes in biomarkers of bone turnover?

📍 UAMS Medical Center · Little Rock, AR · +4 more US sites · Get directions →

This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD. The primary aim of the study is to assess the safety of anumigilimab in participants with SCD.

📍 Site # 8400006 · Atlanta, GA · +4 more US sites · Get directions →

The purpose of this study is to measure whether PCV21 vaccine (investigational pneumococcal vaccine) is safe and can help the body to develop germ-fighting agents called "antibodies" (immunogenicity) compared with 20vPCV (licensed pneumococcal vaccine) when given as a single dose to children aged 2 to 17 years with sickle cell disease who had received or not a previous vaccination with pneumococcal conjugate or pneumococcal polysaccharide vaccine.

📍 Adams Clinical, Boston · Boston, MA · +5 more US sites · Get directions →

TRIAGE-Psych is a survey study designed to assess potential participants' eligibility to screen for industry-sponsored psychiatry clinical trials.

📍 Children's Hospital of Illinois at OSF-Saint Francis Medical Center · Peoria, IL · +5 more US sites · Get directions →

Despite the important work of previous sickle cell disease (SCD) cohort studies, there remain many understudied areas that require investigation. An important knowledge deficit is the slow but progressive process of chronic end-organ dysfunction.

📍 Children's Hospital Los Angeles · Los Angeles, CA · +5 more US sites · Get directions →

This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

📍 Children's National Medical Center · Washington D.C., DC · +4 more US sites · Get directions →

Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States.

📍 Phoenix VA Health Care System, Phoenix, AZ · Phoenix, AZ · +3 more US sites · Get directions →

The VA wants to understand what type of integrative and whole health approaches are helpful for Veterans. The study is comparing two primary care based mental health treatments, a mindfulness class that teaches mindfulness meditation and a problem-solving class that teaches problem-solving skills and how to build resilience, for Veterans who are experiencing symptoms of anxiety, depression, and/or PTSD.

📍 University of California Los Angeles · Los Angeles, CA · +3 more US sites · Get directions →

This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS.

📍 Nemours Children's Health · Wilmington, DE · +3 more US sites · Get directions →

The goal of this clinical trial is to evaluate the impact of SCThrive (a behavioral self-management intervention) on patient activation, self-management behaviors, daily functioning, and emergency room visits in 260 adolescents and young adults with sickle cell disease (SCD) ages 13-21 receiving care at 1 of 4 pediatric SCD clinics. The main question\[s\]it aims to answer are: * Does SCThrive improve patient activation?

📍 Children's Hospital of Colorado · Aurora, CO · +3 more US sites · Get directions →

This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD). This study will enroll and treat up to 24 participants.

📍 University of Illinois at Chicago · Chicago, IL · +3 more US sites · Get directions →

In a hybrid type I effectiveness-implementation trial, our three-center research teams aim to examine whether empowering adults with sickle cell disease (SCD) with patient-facing SCD-specific guidelines through an mHealth application with booklets will decrease acute healthcare utilization and be cost-effective over booklets with the guidelines alone. Our team, head will test our hypotheses with the following aims: Aim 1: evaluate the effectiveness of the patient-facing guidelines mHealth app + booklet intervention to decrease acute healthcare utilization (hospitalizations, emergency room visits, and day hospital visits) in adults with SCD over the standard care in a randomized controlled trial, Aim 2: evaluate the implementation outcomes of the mHealth app + booklet using the capability, opportunity, and motivation-behavior (COM-B) and reach, effectiveness, adoption, implementation, and maintenance (RE-AIM) frameworks, and Aim 3: evaluate the cost-effectiveness of patient-facing mHealth app + booklets vs.

📍 Yale School of Medicine · New Haven, CT · +3 more US sites · Get directions →

This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.

📍 New York Presbyterian Hospital - Morgan Stanley Children's Hospital · New York, NY · +2 more US sites · Get directions →

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.

📍 Connecticut Children's Medical Center · Hartford, CT · +3 more US sites · Get directions →

This research aims to answer the question: does a group training program specifically for teens with chronic sickle cell disease (SCD) pain that teaches skills to strengthen the mind and body help improve everyday functioning and reduce pain symptoms? The program will be tailored to address challenges related to frequent or chronic sickle cell pain and may improve participants' physical and emotional health.

📍 Arizona State University · Phoenix, AZ · +2 more US sites · Get directions →

In a randomized controlled trial (RCT), the investigators will recruit participants to an 8-week "app-based wellness" intervention, followed by a 12-week follow-up period. The investigators will recruit a total of 276 self-declared Chronic Hematological Cancer (CHC) patients who (representative of age, race/ethnicity, and gender) will be on stable CHC pharmacologic therapy (if any), self-identify as sleep disturbed (\>5 on Pittsburgh Sleep Quality Index), do not have a sleep disorder diagnosis, do not take sleep medication/supplements \>3 times per week, and are not currently practicing regular meditation.

📍 Johns Hopkins Center for Indigenous Health · Chinle, AZ · +2 more US sites · Get directions →

This project addresses the disproportionate morbidity and mortality associated with mental and behavioral health problems in American Indian and Alaska Native communities. Access to culturally competent and effective behavioral health services is limited in many of these communities.

📍 Colorado State University · Fort Collins, CO · +2 more US sites · Get directions →

The investigators are conducting research on factors related to the self-regulation of mood and arousal states across a range of everyday activities as well as different levels of stress. Behavioral interventions-such as meditation, listening to music, or visualizing art or nature-offer important alternatives and/or adjunctive strategies to pharmaceutical tools or other mechanisms supporting physical and emotional well-being.

📍 University of California, Los Angeles · Los Angeles, CA · +2 more US sites · Get directions →

There are few treatment options available for patients once they have failed standard psychopharmacological therapy for generalized anxiety disorder. Existing brain stimulation methods such as rTMS fail to target deep brain structures associated with anxiety disorders; structures such as the amygdala.

📍 Lurie Children's Hospital · Chicago, IL · +2 more US sites · Get directions →

This study is for caregivers of young children with sickle cell disease and adolescents with sickle cell disease who are currently prescribed hydroxyurea and are receiving care at one of the study sites. The study will assess retention and engagement during a pilot randomized control trial comparing video directly observed therapy (VDOT) to attention control.

📍 University of Alabama at Birmingham · Birmingham, AL · +2 more US sites · Get directions →

This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use.

📍 Memorial Sloan Kettering at Basking Ridge (Consent only) · Basking Ridge, NJ · +5 more US sites · Get directions →

Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD.

📍 Children's Hospital Los Angeles · Los Angeles, CA · +1 more US site · Get directions →

Babies with single ventricle congenital heart disease (SVCHD) are often diagnosed during pregnancy. While prenatal diagnosis has important clinical benefits, it is often stressful and overwhelming for parents, and many express a need for psychological support.

📍 University of Colorado Denver · Aurora, CO · +2 more US sites · Get directions →

Aerobic exercise has emerged as an effective treatment to reduce sport-related concussion symptom severity, yet existing work lacks rigor regarding the precise exercise volume and intensity required to elicit therapeutic effects, how exercise can alter concussion-related pathophysiology, and whether exercise can prevent the development of secondary sequelae. Our objective is to examine if a high dose exercise program (higher volume than currently prescribed at an individualized, safe intensity level) initiated within 14 days of concussion results in faster symptom resolution, altered physiological function, or reduced secondary sequalae.

📍 Rutgers University · New Brunswick, NJ · +2 more US sites · Get directions →

The goal of this study is to test the efficacy and feasibility of a clinician-guided, app-based cognitive behavioral therapy (CBT) program, SilverCloud, as a school-based mental health intervention for vulnerable youth. An open trial of SilverCloud will be conducted to determine preliminary efficacy in this sample and inform program refinements by collecting outcome self-report assessments and conducting interviews on feasibility and acceptability.

📍 Anxiety and Behavioral Health Clinic · Tallahassee, FL · +2 more US sites · Get directions →

The goal of this clinical trial is to test the effectiveness of a computerized anxiety sensitivity treatment (CAST) compared to a health education control (HEC) in older adults with mild cognitive impairment (MCI) or mild Alzheimer's Disease and related dementias (ADRD) and their care partners. The main questions it aims to answer are: 1.

📍 Lake Forest Hospital · Lake Forest, IL · +2 more US sites · Get directions →

The goal of this randomized crossover trial is to compare the differences in psychological and physiological effects of walking in two different outdoor environments (urban/suburban commercial environments vs. urban/suburban nature areas/preserves) in adults with prediabetes.

📍 University of California, San Diego · San Diego, CA · +1 more US site · Get directions →

This study seeks to understand if the medication pramipexole improves social connectedness and functioning in adults (ages 18-50) who experience anxiety or depression. The study plans to enroll 108 participants total across two sites (University of California San Diego and New York State Psychiatric Institute).

📍 Mayo Clinic Health System-Albert Lea · Albert Lea, MN · +1 more US site · Get directions →

The purpose of this study is to evaluate the feasibility of a structured breathing intervention in rural and non-rural adolescents diagnosed with anxiety and depression, recruited from outpatient pediatric and child/adolescent psychiatry clinics.

📍 University of North Carolina at Chapel Hill · Chapel Hill, NC · +1 more US site · Get directions →

Autistic adults are at a greater risk for mental health problems compared to the general population, with 50% meeting criteria for a co-occurring psychiatric condition. Depression and anxiety are the most common of these conditions among autistic adults, contributing to long-term detrimental effects on health, day-to-day functioning, and quality of life.

📍 Acacia Clinics · Sunnyvale, CA · +1 more US site · Get directions →

Participants will receive Transcranial Magnetic Stimulation (TMS) at a random location in the left prefrontal cortex, excluding sites that are potentially unsafe. Extensive behavioral testing will be conducted to determine which behaviors are modulated by stimulating which circuits.

📍 Montclair State University · Montclair, NJ · +1 more US site · Get directions →

The goal of this clinical trial is to investigate neural constructs related to anxiety symptoms in response to Facing Your Fears (FYF) treatment in autistic participants with co-occurring anxiety. Researchers will compare participants receiving immediate FYF intervention to those in the waitlist control group (receiving treatment as usual) to see if there are differences in neural constructs and anxiety symptom changes over the study period.

📍 University of California Los Angeles · Los Angeles, CA · +1 more US site · Get directions →

Anxiety disorders such as post-traumatic stress disorder (PTSD) and generalized anxiety disorder (GAD) affect a large number of individuals with a significant portion of patients failing to improve with current treatments. The purpose of this study is to understand the brain mechanisms that produce fear and anxiety in humans.

📍 University of Connecticut School of Medicine · West Hartford, CT · +1 more US site · Get directions →

Excessive anxiety among elementary students is highly prevalent and associated with impairment in academic, social, and behavioral functioning. The primary aim of this project is to evaluate the initial efficacy of a brief nurse-delivered intervention (CALM: Child Anxiety Learning Modules), relative to a credible comparison (CALM-R, relaxation skills only) and a waitlist control for reducing anxiety symptoms and improving education outcomes at post intervention and at a 1-year follow-up.

📍 Northwestern University · Evanston, IL · +1 more US site · Get directions →

People spend approximately one-third of their lives asleep, yet sleep is often underused as an opportunity to support psychological well-being. Contemplative traditions, including Tibetan Dream Yoga, have developed practices that use waking imagination and lucid dreaming to explore perception, awareness, and habitual patterns of thinking.

📍 Boston University · Boston, MA · +1 more US site · Get directions →

Anxiety-, obsessive-compulsive and trauma- and stressor-related disorders reflect a significant public health problem. This study is designed to evaluate the predictive power of a novel biomarker based on a CO2 challenge, thus addressing the central question "can this easy-to-administer assay aid clinicians in deciding whether or not to initiate exposure-based therapy?"