516 matching studies

Sponsor Condition of Interest
Natural History of Rett Syndrome & Related Disorders
University of Alabama at Birmingham Rett Syndrome MECP2 Duplication dIsorder CDKL5 Disorder FOXG1 Syndrome
The purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), CDKL5, FOXG1, and individuals with MECP2 mutations who do not have RTT including the range of clinical involvement and to correlate... expand

The purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), CDKL5, FOXG1, and individuals with MECP2 mutations who do not have RTT including the range of clinical involvement and to correlate genotype-phenotype over a broad spectrum of phenotypes. While much has been learned about RTT, improvements are required in understanding the role of factors such as X chromosome inactivation, genetic background, and others including the environment, on the great variability observed even between individuals with the same MECP2 mutation. These data will be essential to the development and conduct of clinical trials that are anticipated from ongoing studies in animal models for RTT. This study will not include clinical trials, but should set the stage for such trials and other translational research projects (e.g., development of biomarkers).

Type: Observational

Start Date: Nov 2015

open study

Non-Encapsulated Heme Levels in Adult Cardiac Surgery
University of Alabama at Birmingham Adult Cardiac Surgery
Non-encapsulated (free) heme, a breakdown component of hemoglobin, is associated with oxidative stress and inflammation with consequent cellular and tissue injury (Ryter & Tyrrell, 2000) (D T G Wagener, et al., 2001). Cardiopulmonary bypass is known to cause trauma to cellular... expand

Non-encapsulated (free) heme, a breakdown component of hemoglobin, is associated with oxidative stress and inflammation with consequent cellular and tissue injury (Ryter & Tyrrell, 2000) (D T G Wagener, et al., 2001). Cardiopulmonary bypass is known to cause trauma to cellular components of the blood, to trigger an inflammatory response, and alter the rheology of the circulation. Prior research has demonstrated an increase in levels of free heme with cardiopulmonary bypass (Wetz, Richardt, Schotola, Bauer, & Bräuer, 2017) (Kubota, Egi, & Mizobuchi, 2017). The stress response to cardiac surgery and utilization of blood salvage techniques independent of cardiopulmonary bypass may affect plasma free heme levels as well as regulation of heme metabolism pathways. It is unclear whether and to what degree plasma levels of free heme may vary at discreet time points in the perioperative period during cardiac surgery involving both cardiopulmonary bypass and "off-pump" techniques. This is an observational, prospective cohort study in which we will assess for red blood cell trauma, free heme levels, and biomarkers for acute kidney injury at various time points throughout cardiac surgery. Urine and arterial blood samples will be collected at our routine, standard of care time points pre-, intra-, and post-operatively and in addition to the standard clinical tests that will be performed (arterial blood gas analysis and activated clotting time) several additional serum biomarkers will be analyzed. Clinical correlation will be performed with levels. Sub-analysis will be performed on the basis of off vs. on pump CABG cases and also in patient that do/not develop AKI post-operatively.

Type: Observational

Start Date: Jun 2019

open study

GKT137831 in IPF Patients With Idiopathic Pulmonary Fibrosis
University of Alabama at Birmingham Idiopathic Pulmonary Fibrosis
A placebo-controlled, multicenter, randomized trial to test GKT137831 in ambulatory patients with idiopathic pulmonary fibrosis. This drug is an inhibitor of nicotinamide adenine dinucleotide phosphate (NADPH) oxidase (NOX) isoforms. The investigators hypothesize the drug... expand

A placebo-controlled, multicenter, randomized trial to test GKT137831 in ambulatory patients with idiopathic pulmonary fibrosis. This drug is an inhibitor of nicotinamide adenine dinucleotide phosphate (NADPH) oxidase (NOX) isoforms. The investigators hypothesize the drug will decrease pulmonary injury due to reactive oxygen species (ROS) generated by NOX enzymes, which are believed to play an important role in the development of IPF. Treatment with GKT137831 could result in significant benefit for a lung disease that has, until now, been almost invariably inexorable. This clinical trial represents the bedside application of a series of NOX translational and basic studies and discoveries, over several years, from the laboratory of Dr. Victor Thannickal.

Type: Interventional

Start Date: Sep 2020

open study

A Phase 1/2 Study to Evaluate SNDX- 6352 in Subjects With Active cGVHD
Syndax Pharmaceuticals Chronic Graft-versus-host-disease
This is a Phase 1/2, Open-label, Dose Escalation study to investigate SNDX-6352 in subjects with active cGVHD. expand

This is a Phase 1/2, Open-label, Dose Escalation study to investigate SNDX-6352 in subjects with active cGVHD.

Type: Interventional

Start Date: Nov 2018

open study

Biobanking of Rett Syndrome and Related Disorders
University of Alabama at Birmingham Rett Syndrome MECP2 Duplication CDKL5 FOXG1 Disorders
The overarching purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), RTT-related disorders including CDKL5, FOXG1, and individuals with MECP2 mutations who do not have RTT. Although all these... expand

The overarching purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), RTT-related disorders including CDKL5, FOXG1, and individuals with MECP2 mutations who do not have RTT. Although all these disorders are the result of specific genetic changes, there remains broad clinical variation that is not entirely accounted for by known biological factors. Additionally, clinical investigators currently do not have any biomarkers of disease status, clinical severity, or responsiveness to therapeutic intervention. To address these issues, biological materials (DNA, RNA, plasma, cell lines) will be collected from affected individuals and in some cases from unaffected family members, initial evaluation performed to identify additional biological factors contributing to disease severity, and these materials will be stored for future characterization.

Type: Observational

Start Date: Sep 2017

open study

Cycled Phototherapy
NICHD Neonatal Research Network Hyper Bilirubinemia Premature Infant
Cycled phototherapy (PT) is likely to increase survival over that with continuous PT among extremely premature infants (< 750 g BW or <27 weeks GA). expand

Cycled phototherapy (PT) is likely to increase survival over that with continuous PT among extremely premature infants (< 750 g BW or <27 weeks GA).

Type: Interventional

Start Date: Jul 2020

open study

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry...
Idorsia Pharmaceuticals Ltd. Fabry Disease
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease expand

A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease

Type: Interventional

Start Date: Dec 2018

open study

Euglycemia After Antenatal Late Preterm Steroids, the E-ALPS Study
University of North Carolina, Chapel Hill Pregnancy Preterm Neonatal Hypoglycemia Hyperglycemia Drug Induced
Annually in the U.S 300,000 neonates are born late preterm, defined as 34 weeks 0 days - 36 weeks 6 days. The Antenatal Late Preterm Steroids (ALPS) Trial demonstrated that maternal treatment with betamethasone in the late preterm period significantly reduces neonatal respiratory... expand

Annually in the U.S 300,000 neonates are born late preterm, defined as 34 weeks 0 days - 36 weeks 6 days. The Antenatal Late Preterm Steroids (ALPS) Trial demonstrated that maternal treatment with betamethasone in the late preterm period significantly reduces neonatal respiratory complications, but also increases neonatal hypoglycemia, compared to placebo. This research study will attempt to answer the following primary question: Does a management protocol aimed at maintaining maternal euglycemia after ALPS decrease fetal hyperinsulinemia, compared to usual antepartum care?

Type: Interventional

Start Date: Jun 2017

open study

A Study of VB-111 With Paclitaxel vs Paclitaxel for Treatment of Recurrent Platinum-Resistant Ovarian...
Vascular Biogenics Ltd. operating as VBL Therapeutics Recurrent Platinum Resistant Ovarian Cancer
The purpose of this phase 3, randomized, multicenter study is to compare VB-111 and paclitaxel to placebo and paclitaxel in adult patients with Recurrent Platinum-Resistant Ovarian Cancer. expand

The purpose of this phase 3, randomized, multicenter study is to compare VB-111 and paclitaxel to placebo and paclitaxel in adult patients with Recurrent Platinum-Resistant Ovarian Cancer.

Type: Interventional

Start Date: Dec 2017

open study

Study to Evaluate the Efficacy and Safety of Ibrexafungerp in Patients With Fungal Diseases That Are...
Scynexis, Inc. Invasive Candidiasis Mucocutaneous Candidiasis Coccidioidomycosis Histoplasmosis Blastomycosis
This is a multicenter, open label, non-comparator, single arm study to evaluate the efficacy and safety of ibrexafungerp (SCY-078) in patients ≥ 18 years of age with a documented fungal disease that has been intolerant or refractory (rIFI) to Standard of Care (SoC) antifungal... expand

This is a multicenter, open label, non-comparator, single arm study to evaluate the efficacy and safety of ibrexafungerp (SCY-078) in patients ≥ 18 years of age with a documented fungal disease that has been intolerant or refractory (rIFI) to Standard of Care (SoC) antifungal treatment.

Type: Interventional

Start Date: Apr 2017

open study

Neuroinflammation After Myocardial Infarction - Imaging Substudy
University of Alabama at Birmingham Myocardial Infarction
The purpose of the study is to see if positron emission tomography and magnetic resonance imaging (PET/MRI) with an investigational drug called [18F]DPA-714 will show inflammation in the brain after a heart attack. This study may help physicians and researchers better understand... expand

The purpose of the study is to see if positron emission tomography and magnetic resonance imaging (PET/MRI) with an investigational drug called [18F]DPA-714 will show inflammation in the brain after a heart attack. This study may help physicians and researchers better understand the role of brain inflammation in heart disease and develop new treatments to protect the brain.

Type: Interventional

Start Date: Jul 2019

open study

Longitudinal Study of the Porphyrias
Icahn School of Medicine at Mount Sinai Acute Porphyrias Cutaneous Porphyrias
The objective of this protocol is to conduct a longitudinal multidisciplinary investigation of the human porphyrias including the natural history, morbidity, pregnancy outcomes, and mortality in people with these disorders. expand

The objective of this protocol is to conduct a longitudinal multidisciplinary investigation of the human porphyrias including the natural history, morbidity, pregnancy outcomes, and mortality in people with these disorders.

Type: Observational

Start Date: Nov 2010

open study

Sleep and Stigma: Novel Moderators in the Relationship Between Weight Status and Cognitive Function
University of Alabama at Birmingham Obesity, Childhood Overweight, Childhood
The investigators aim to assess the relationship between overweight/obesity and decreased cognitive function in adolescents. While this relationship has been seen in past literature, the causal mechanisms are still unclear. Thus, the present study will assess sleep and stigma... expand

The investigators aim to assess the relationship between overweight/obesity and decreased cognitive function in adolescents. While this relationship has been seen in past literature, the causal mechanisms are still unclear. Thus, the present study will assess sleep and stigma as possible moderators. As sleep is related to both weight and cognitive abilities it may be an important factor in the relationship between these two variables. Further, people with overweight/obesity have higher risk for stigma experiences which may increase inflammation through chronic stress and elevated cortisol. Because inflammation is theorized to play a role in the relationship between elevated BMI and decreased cognitive function, stigma may be an important moderator. 60 adolescent participants will complete two sleep conditions (adequate and restricted) in a randomized order, each followed by a lab visit during which participants will complete a short cognitive battery. At these visits, participants will also be given a self serve breakfast with a variety of whole and processed food options to further evaluate the relationship between overweight/obesity, sleep, nutritional intake, and cognitive function.

Type: Interventional

Start Date: Sep 2020

open study

Bougie or Stylet In Patients Undergoing Intubation Emergently (BOUGIE)
Vanderbilt University Medical Center Acute Respiratory Failure
Complications are common during tracheal intubations performed outside of the operating room. Successful intubation on the first attempt has been associated with a lower rate of procedural complications, but the proportion of critically ill patients intubated on the first... expand

Complications are common during tracheal intubations performed outside of the operating room. Successful intubation on the first attempt has been associated with a lower rate of procedural complications, but the proportion of critically ill patients intubated on the first attempt during tracheal intubations outside of the operating room is less than 90%. The bougie, a thin semi-rigid tube that can be placed into the trachea, allowing a Seldinger-like technique of intubating a patient's airway, has been traditionally reserved for difficult or failed airways. However, a recent single center trial of adult patients intubated in an emergency department demonstrated that use of the bougie on the first attempt improved intubation success, compared to use of a traditional stylet. Theinvestigators propose a multi-center randomized trial to compare first-attempt bougie use versus endotracheal tube with stylet use for tracheal intubation of critically ill adults in the ED and ICU.

Type: Interventional

Start Date: Apr 2019

open study

Scale Up Evaluation of a Physical Activity Program for Adults With Physical Disability
University of Alabama at Birmingham Physical Disability
The study uses a theory-driven eHealth platform and innovative physical activity (PA) program referred to as movement-to-music (M2M) to deliver a customized, home-based PA intervention for adults with physical disability (PWD). Participants will be recruited through a large... expand

The study uses a theory-driven eHealth platform and innovative physical activity (PA) program referred to as movement-to-music (M2M) to deliver a customized, home-based PA intervention for adults with physical disability (PWD). Participants will be recruited through a large physical medicine and rehabilitation network of clinics specializing in treating patients with physical disability. Participants will be randomized to one of three groups: a) M2M; b) M2M plus social networking (M2Mplus); and c) attention control (AC). Participants will be followed for 48 weeks to obtain objective measures on physical activity, fitness and self-reported measures on health at four time points. The study will compare the effectiveness of M2M and M2Mplus in increasing physical activity and adherence compared to the AC group, estimate the improvements in health of M2M and M2Mplus compared to the AC group, and examine the mediators and moderators of the hypothesized treatment effect to understand for whom and how the intervention is effective. This multi-level, scalability study will strengthen our understanding of the potential benefits of eHealth to eliminate barriers to PA participation among PWD.

Type: Interventional

Start Date: Apr 2018

open study

Pulmonary Fibrosis Foundation Patient Registry
University of Michigan Interstitial Lung Disease (ILD) Idiopathic Pulmonary Fibrosis (IPF)
The Pulmonary Fibrosis Foundation Patient Registry will collect data on at least 2,000 patients with interstitial lung disease (ILD) at approximately 40 clinical sites in the US. The Registry is targeting enrollment of approximately 60% of the 2,000 ILD participants to have... expand

The Pulmonary Fibrosis Foundation Patient Registry will collect data on at least 2,000 patients with interstitial lung disease (ILD) at approximately 40 clinical sites in the US. The Registry is targeting enrollment of approximately 60% of the 2,000 ILD participants to have idiopathic pulmonary fibrosis (IPF). The aim of the Registry is to create a cohort of well-characterized patients with interstitial lung disease (ILD) for participation in retrospective and prospective research

Type: Observational [Patient Registry]

Start Date: Mar 2016

open study

TOSS Feasibility + Fitbit Community = Reduced Obesity in Older Black Women
University of Alabama at Birmingham Obesity
The primary goal of this proposed physical activity (PA) intervention is to develop a realistic and an economical intervention to promote regular PA among older, overweight or obese Black women. Physical inactivity is prevalent among older Black women and this lifestyle behavior... expand

The primary goal of this proposed physical activity (PA) intervention is to develop a realistic and an economical intervention to promote regular PA among older, overweight or obese Black women. Physical inactivity is prevalent among older Black women and this lifestyle behavior is both a destructive and expensive public health issue. Because many in this population do not engage in sufficient amounts of regular PA, this group miss the cardio-protective effects that may reduce many chronic conditions that disproportionately affect Blacks such as hypertension, diabetes, and obesity. The proposed obesity-reduction intervention is aligned with Stage 1 of the National Institute on Aging Stage Model for Behaviors Interventions Development. For the proposed hypothesis, the investigators propose a 2-group randomized controlled pilot, feasibility study in which 30 women will be randomized to receive either the TOSS PA Text messages Plus Fitbit community or the Control group and followed for 12 weeks to detect intervention effect.

Type: Interventional

Start Date: Jul 2020

open study

Treatment of Chronic Antibody-mediated Rejection in Kidney Transplant With Acthar
University of Maryland, Baltimore Transplant Glomerulopathy
This is an open label safety and feasibility trial using Acthar® in addition to the investigators center-specific standard therapy, which could include increase in maintenance immunosuppression, high dose IVIG (2 g/Kg), and/or Rituximab, in patients with CAMR. expand

This is an open label safety and feasibility trial using Acthar® in addition to the investigators center-specific standard therapy, which could include increase in maintenance immunosuppression, high dose IVIG (2 g/Kg), and/or Rituximab, in patients with CAMR.

Type: Interventional

Start Date: Aug 2016

open study

Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia
University of Alabama at Birmingham Sickle Cell Disease Renal Disease Glomerular Disease
The investigators will attempt to develop a more accurate equation to estimate eGFR in pediatric and adult sickle cell patients expand

The investigators will attempt to develop a more accurate equation to estimate eGFR in pediatric and adult sickle cell patients

Type: Observational

Start Date: Aug 2020

open study

Non-Surgical Treatment for Rotator-Cuff Tears Using Platelet-Rich-Plasma
University of Alabama at Birmingham Partial Tear of Rotator Cuff Tendinopathy of Rotator Cuff
The goal of this project is to identify an effective and conservative approach to treating partial thickness rotator cuff tears (PTRCT) that otherwise would end with a surgical correction needed. expand

The goal of this project is to identify an effective and conservative approach to treating partial thickness rotator cuff tears (PTRCT) that otherwise would end with a surgical correction needed.

Type: Interventional

Start Date: Jul 2014

open study

CD24Fc for the Prevention of Acute GVHD Following Myeloablative HSCT
OncoImmune, Inc. Hematopoietic Stem Cell Transplantation Acute Graft Versus Host Disease Acute Myeloid Leukemia Acute Lymphoblastic Leukemia Myelodysplastic Syndromes
The study is designed as a randomized, placebo-controlled, double blind, multicenter, phase III trial comparing two acute graft-versus-host disease (aGVHD) prophylaxis regimens: CD24Fc vs placebo with the standard GVHD prophylaxis of tacrolimus / methotrexate. expand

The study is designed as a randomized, placebo-controlled, double blind, multicenter, phase III trial comparing two acute graft-versus-host disease (aGVHD) prophylaxis regimens: CD24Fc vs placebo with the standard GVHD prophylaxis of tacrolimus / methotrexate.

Type: Interventional

Start Date: Sep 2020

open study

Piloting Home-based Movement-to-Music Among Adolescents With Cerebral Palsy: A Randomized Controlled...
University of Alabama at Birmingham Cerebral Palsy
This study pilots the efficacy of a home-based movement-to-music program for increasing physical activity participation among adolescents with cerebral palsy. Half of the participants will receive the movement-to-music program immediately, which will include 4 weeks of exercise... expand

This study pilots the efficacy of a home-based movement-to-music program for increasing physical activity participation among adolescents with cerebral palsy. Half of the participants will receive the movement-to-music program immediately, which will include 4 weeks of exercise videos and periodic behavioral coaching calls. The other half of participants will wait 4 weeks before receiving the M2M program.

Type: Interventional

Start Date: Sep 2020

open study

Assessment of Neuroinflammation in Central Inflammatory Disorders Using [F-18]DPA-714.
University of Alabama at Birmingham Fibromyalgia Chronic Fatigue Syndrome Multiple Sclerosis Healthy
The primary objective of this study is to measure the concentration and the regional brain distribution of activated brain microglia/macrophages using the PET radiopharmaceutical [F-18]DPA-714 in individuals with chronic pain and fatigue suspected to be associated with neuroinflammation.... expand

The primary objective of this study is to measure the concentration and the regional brain distribution of activated brain microglia/macrophages using the PET radiopharmaceutical [F-18]DPA-714 in individuals with chronic pain and fatigue suspected to be associated with neuroinflammation. The PET tracer [F-18]DPA-714 binds to the 18 kDa translocator protein (TSPO, also known as the peripheral benzodiazepine receptor) in the mitochondria of activated microglia/macrophages and provides a non-invasive measure of neuroinflammation. The primary objective of this study is to determine if pain and fatigue patients have higher levels of neuroinflammation than HC individuals as measured with [F-18]DPA-714-PET/MRI.

Type: Interventional

Start Date: Feb 2019

open study

Personalized Theratyping Trial
University of Alabama at Birmingham Cystic Fibrosis
The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs. expand

The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs.

Type: Interventional

Start Date: Aug 2019

open study

An Upstream Palliative Care Intervention for Rural and African-American Advanced Cancer Family Caregivers
University of Alabama at Birmingham Cancer, Metastatic Family Members
The burden placed on advanced cancer family caregivers (FCGs) and the toll on their mental and physical health have never been greater and is particularly pernicious for underserved groups in the Southeastern U.S., including rural-dwellers and African-Americans, for whom interventions... expand

The burden placed on advanced cancer family caregivers (FCGs) and the toll on their mental and physical health have never been greater and is particularly pernicious for underserved groups in the Southeastern U.S., including rural-dwellers and African-Americans, for whom interventions have not been specifically developed and tested. The purpose of this K99/R00 is provide the awardee with the knowledge and training necessary to become a leading independent clinical investigator in developing and testing telehealth palliative care interventions for family caregivers (FCGs) of underserved persons with advanced cancer. The research specific aim during the K99 phase was to develop and tailor the content, format and delivery of a telehealth intervention for FCGs of persons with advanced cancer in the rural South. To meet this aim, qualitative semi-structured interviews were conducted with 26 lay healthcare navigators, 20 FCGs, and 18 patients with advanced cancer to elicit feedback on a FCG intervention outline based on published evidence-based interventions. After thematic analysis, major findings included that the intervention should: have topical content that is flexible and based on continuous assessment; include at least some face-to-face contact; cautiously consider internet-based technologies because internet access in rural areas is poor; and potentially have navigators lead the intervention with appropriate oversight and additional training. K99 study findings have been used to adapt and develop a potentially scalable FCG intervention in the Southern U.S. population (Project Cornerstone). The K99 study phase in combination with accomplishing the training objectives has successfully catalyzed the awardee's transition from a Postdoctoral Fellow to a Tenure-Track Assistant Professor at the University of Alabama at Birmingham School of Nursing. The overall goal during the R00 phase will be to conduct a small-scale pilot RCT of Cornerstone with 70 FCGs of rural-dwelling and/or African-American patients with advanced cancer to assess acceptability, feasibility, and potential efficacy. The research specific aims during the R00 phase are to: evaluate the acceptability of Cornerstone and the feasibility of enrolling and retaining 70 participants into a RCT of the FCG intervention for 24 weeks (Aim 1) and evaluate the potential efficacy of Cornerstone in improving caregiver (Aim 2) and care recipient (Aim 3) outcomes compared to usual care (quality of life and distress [anxiety/depressive symptoms]). The results will directly support an R-01 application to conduct an efficacy RCT of an early, telehealth PC intervention for Southern FCGs of underserved persons with advanced cancer.

Type: Interventional

Start Date: Oct 2018

open study