UAB - Center for Clinical and Translational Science

The objective of this study is to determine if early high volume intravenous fluid administration (hyperhydration) may be effective in mitigating or preventing complications of shiga toxin-producing E. coli (STEC) infection in children and adolescents when compared with traditional approaches (conservative fluid management).

Type: Interventional

Start Date: Sep 2022

open study

The purpose of ORACLE is to demonstrate the ability of a novel ctDNA assay developed by Guardant Health to detect recurrence in individuals treated for early-stage solid tumors. It is necessary that ctDNA test results are linked to clinical outcomes in order to demonstrate clinical validity for recurrence detection and explore its value in a healthcare environment subject to cost containment.

Type: Observational

Start Date: Sep 2021

open study

This first-in-patient phase 2a pilot study will assess the safety and tolerability of MW01-6-189WH (hereafter called MW189) in patients with Intracerebral Hemorrhage (ICH).

Type: Interventional

Start Date: Oct 2022

open study

This phase II trial determines if the combination of ONC201 with different drugs, panobinostat or paxalisib, is effective for treating participants with diffuse midline gliomas (DMGs). Despite years of research, little to no progress has been made to improve outcomes for participants with DMGs, and there are few treatment options. ONC201, panobinostat, and paxalisib are all enzyme inhibitors that may stop the growth of tumor cells by clocking some of the enzymes needed for cell growth. This phase II trial assesses different combinations of these drugs for the treatment of DMGs.

Type: Interventional

Start Date: Oct 2021

open study

Study consists of two parts, a part 1 dose escalation and a part 2 cohort expansion in combination with dexamethasone and carfilzomib intravenously across two cohorts with a monotherapy component as well.

Type: Interventional

Start Date: Sep 2021

open study

The purpose of this Phase 3 study is to determine whether iptacopan (LNP023) is efficacious and safe for the treatment of aHUS in adult patients who are treatment naive to complement inhibitor therapy.

Type: Interventional

Start Date: Jan 2022

open study

The purpose of this protocol is to begin an exercise program combined with behavioral counseling for patients who are hospitalized with a cystic fibrosis exacerbation. The exercise program will begin during the hospital stay. Beginning an exercise program during this period of reduced mobility and isolation may be an ideal time to deliver a structured exercise prescription along with a behavioral program to promote long-term adherence to exercise (structured physical activity) . Hospitalized patients have an acute awareness that their lung function is declining and may be more motivated and open to changing their behavior and adding exercise to their treatment regimen.

Type: Interventional

Start Date: Dec 2020

open study

Objectives of this study are to determine whether active VNS Therapy treatment is superior to a no stimulation control in producing a reduction in baseline depressive symptom severity, based on multiple depression scale assessment tools at 12 months from randomization.

Type: Interventional

Start Date: Sep 2019

open study

The main purpose of this study is to identify a safe and potentially effective dose of tuspetinib to be used in future studies in study participants diagnosed with acute myeloid leukemia (AML), myelodysplastic syndromes with increased blasts grade 2 (MDS-IB2), or chronic myelomonocytic leukemia (CMML) that is relapsed or refractory after at least one line of prior therapy, or in study participants with newly diagnosed AML. Tuspetinib will be administered as a single agent or in combination with other drugs (venetoclax or venetoclax plus azacitidine), as specified for each part of the study.

Type: Interventional

Start Date: Mar 2019

open study

The Hydrocephalus Clinical Research Network (HCRN) has been established by philanthropic funding to conduct multi-institutional research (clinical trials and observational studies) on pediatric hydrocephalus. In addition to philanthropic funding, the HCRN has also received an NIH NINDS Challenge Grant to support the network infrastructure which allows for the conduct of this and other network studies. The HCRN consists of multiple Clinical Centers and the Data Coordinating Center (DCC). The HCRN Core Data Project will obtain data about all neurosurgical hydrocephalus events from the network Clinical Centers, and create a database to be used by HCRN investigators. The ongoing maintenance of the Core Data Project serves two main purposes: 1) it will help investigators understand the variability, progression, and current treatment practices for hydrocephalus in children, with an ultimate goal of better guiding and assessing therapeutic intervention and providing recommendations on patient care and, 2) it will provide pilot and descriptive data necessary for hypothesis generation and study design (i.e. preliminary power analyses, recruitment projections) for studies under development by the HCRN. This multi-institutional database will be maintained throughout the lifetime of the HCRN, and may be useful for tracking trends in pediatric hydrocephalus over time. The Core Data Project will be an invaluable resource to the HCRN and will help stimulate new research protocols, identify potential need for future expansion of the network to incorporate additional patient populations, and provide a descriptive understanding of children with hydrocephalus cared for within the network.

Type: Observational

Start Date: Apr 2008

open study

Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. Many individuals find themselves being passed from physician to physician, undergoing countless and often repetitive tests in the hopes of finding answers and insight about what the future may hold. This long and arduous journey to find a diagnosis does not end for many patients- the Office of Rare Diseases Research (ORDR) notes that 6% of individuals seeking their assistance have an undiagnosed disorder. In 2008, the National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP) was established with the goal of providing care and answers for these individuals with mysterious conditions who have long eluded diagnosis. The NIH UDP is a joint venture of the NIH ORDR, the National Human Genome Research Institute Intramural Research Program (NHGRI-IRP), and the NIH Clinical Research Center (CRC) (1-3). The goals of the NIH UDP are to: (1) provide answers for patients with undiagnosed diseases; (2) generate new knowledge about disease mechanisms; (3) assess the application of new approaches to phenotyping and the use of genomic technologies; and (4) identify potential therapeutic targets, if possible. To date, the UDP has evaluated 3300 medical records and admitted 750 individuals with rare and undiagnosed conditions to the NIH Clinical Center. The NIH UDP has identified more than 70 rare disease diagnoses and several new conditions. The success of the NIH UDP prompted the NIH Common Fund to support the establishment of a network of medical research centers, the Undiagnosed Diseases Network (UDN), for fiscal years 2013-2020. The clinical sites will perform extensive phenotyping, genetic analyses, and functional studies of potential disease-causing variants. The testing performed on patients involves medically indicated studies intended to help reach a diagnosis, as well as research investigations that include a skin biopsy, blood draws, and DNA analysis. In addition, the UDN will further the goals of the UDP by permitting the sharing of personally identifiable phenotypic and genotypic information within the network. By sharing participant information and encouraging collaboration, the UDN hopes to improve the understanding of rare conditions and advance the diagnostic process and care for individuals with undiagnosed diseases.

Type: Observational

Start Date: Sep 2015

open study

Our goal is to better understand how DBS modifies local neuronal activity and to pioneer device technologies that can record local DBS-evoked potentials (DLEPs) to guide therapy. Our vision is for a patient's unique electrophysiology to guide both electrode targeting during surgery and programming in clinic, eventually as an integrated component of the implanted pulse generator. Our results will inform directional DBS for PD and serve as a model for translation to other diseases where knowledge on DBS circuit interactions is at an even earlier stage.

Type: Interventional

Start Date: Jan 2024

open study

AFM13-203 is a phase 2, open-label, multi-center, multi-cohort study with a safety run-in followed by expansion cohorts. The study is evaluating the safety and efficacy of AFM13 in combination with AB-101 in subjects with R/R classical HL and CD30-positive PTCL.

Type: Interventional

Start Date: Oct 2023

open study

The purpose of this study is to learn about the effects, safety and how PF-06823859 is processed in adults with cutaneous lupus erythematosus (CLE) or systemic lupus erythematosus (SLE) showing some skin symptoms. This study is seeking for participants who: - are adults of 18 years of age or older. - are confirmed to have CLE or SLE with involvement of the skin. - have a Cutaneous Lupus Erythematosus Disease Area and Severity Index activity (CLASI-A) score of at least 8. About 48 participants will be selected to receive active study medicine (PF-06823859) or placebo (an infusion without drug). About 32 are grouped to receive the active study medicine and 16 are to receive placebo. They will be receiving the treatments by intravenous infusion (injected directly into the veins). At week 16 all participants receiving the active study drug since day 1 and participants who have received placebo since day 1 and are not responding clinically will receive active study medication. Patients who have received placebo since Day 1 and who have had a clinical response will continue to receive placebo till week 40. All participants will have last follow-up visit at Week 60. The study will compare participants receiving PF-06823859 to participants who receive placebo. This will help us see if PF-06823859 is safe and effective to treat CLE or SLE with skin symptoms and improve participant's CLASI-A score. Participants will take part in this study for about 65 weeks. This includes up to a 5-week selection period, a 12-week Q4Wk treatment period, a 36-week Q8Wk treatment period, and a 12-week follow-up period.

Type: Interventional

Start Date: Jul 2023

open study

The purpose of this study is to assess the objective response rate (ORR) of immunotherapy-based combination therapy and to assess the safety and tolerability of immunotherapy-based combination therapy.

Type: Interventional

Start Date: Feb 2023

open study

The purpose of this study is to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab in participants with anti-N-methyl-D-aspartic acid receptor (NMDAR) and anti-leucine-rich glioma-inactivated 1 (LGI1) encephalitis.

Type: Interventional

Start Date: Sep 2022

open study

This is a randomized, open-label study of Serplulimab plus chemotherapy (Carboplatin-Etoposide) in comparison with Atezolizumab plus chemotherapy in previously untreated US patients with ES-SCLC. Subjects in this study will be randomized to arm A or B at 1:1 ratio as follows: - Arm A (Serplulimab): Serplulimab + chemotherapy (carboplatin-etoposide) - Arm B (control): Atezolizumab + chemotherapy (carboplatin-etoposide)

Type: Interventional

Start Date: Nov 2022

open study

This is a multi-site, double-blind, randomized clinical trial of the 5-HT2A receptor agonist psilocybin for smoking cessation. Four sites with experience in conducting psilocybin research will be involved in this trial: Johns Hopkins University (JHU), the University of Alabama at Birmingham (UAB), New York University (NYU), and Sheppard Pratt (SP). The proposed study will treat 66 participants (22 at each site), randomized to receive either: 1) oral psilocybin (30 mg in session 1 and either 30 mg or 40 mg in session 2); or 2) oral niacin (150 mg in session 1 and either 150 mg or 200 mg in session 2), with sessions 1 week apart.

Type: Interventional

Start Date: Nov 2023

open study

This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo controlled study to evaluate the effect of venglustat on neuropathic and abdominal pain symptoms of Fabry disease in participants ≥16 years of age with Fabry disease who are treatment-naïve or untreated for at least 6 months. - Study visits will take place approximately every 3 months. - The double-blind period will be followed by an open-label extension (OLE) during which participants who have completed the double-blind period will be treated with venglustat for up to an additional 12 months.

Type: Interventional

Start Date: Mar 2022

open study

The overarching hypothesis of the ARRC trial is that administration of Azithromycin (AZM) during acute, Respiratory Syncytial Virus (RSV)-induced respiratory failure will be beneficial, mediated through the matrix metalloproteinase (MMP)-9 pathway.

Type: Interventional

Start Date: Feb 2022

open study

This is an open-label, two-part Phase 2 study investigating CGT9486 for the treatment of patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM (ASM), SM with Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL).

Type: Interventional

Start Date: Nov 2021

open study

Using a highly innovative methodology, the Multiphase Optimization Strategy (MOST), the purpose of this randomized factorial trial is to identify components of a intervention (CASCADE) to enhance the decision support skills of family caregivers of persons with newly-diagnosed advanced cancer. Using a 2x2x2x2 full factorial design, 256 family caregivers of persons with newly-diagnosed advanced cancer will be randomized to receive one or more nurse coach-delivered decision partnering training components, based on the Ottawa Decision Support Framework and Social Support Effectiveness Theory: 1) psychoeducation on effective decision partnering principles (1 vs. 3 sessions); 2) decision partnering communication training (yes vs. no); 3) Ottawa Decision Guide training (yes vs. no); and monthly follow (1 monthly follow-up call vs. monthly follow-up calls for 24 weeks).

Type: Interventional

Start Date: Jan 2022

open study

This study is a Phase I/II study evaluating the safety and effectiveness of focused radiation therapy (radiosurgery) together with olaparib, followed by immunotherapy, for patients with brain metastases from triple negative or BRCA-mutated breast cancers. This study will have a Phase I portion in which subjects will be enrolled based on 3+3 dose escalation rules. Three dose levels of olaparib will be studied. Cycle 1 of study treatment will consist of Olaparib given twice daily concurrently with stereotactic radiosurgery (SRS). Olaparib will start one week prior to SRS and continue during and following SRS (1-5 fractions) for up to 28 days total. The number of doses of Olaparib will be dependent on how long it takes a subject to recover from SRS (ideally the subject will be off steroids, if they are required, at the start of Cycle 2, with exceptions outlined later in this section). Once the subject has recovered from SRS (based on investigator discretion) that will be considered the DLT period. Cycle 2 will be initiated with physician's choice systemic therapy and durvalumab. Cycle 2+ will equal 21 days. During Cycles 2 and 3, physician's choice systemic monotherapy will be given along with durvalumab per protocol. Each cycle will last 21 days. Imaging to evaluate intracranial and extracranial disease will be performed after Cycle 3, and subjects with response will continue with the systemic therapy and durvalumab until progression (intracranial or extracranial), unacceptable toxicity or death.

Type: Interventional

Start Date: Mar 2022

open study

The 2021 ACR RA treatment guideline, based on widely acknowledged low to moderate quality evidence, recommends switching to a non-tumor necrosis factor (TNFi) biologic (choose among existing medications, currently, rituximab, abatacept, tocilizumab, or sarilumab) or a targeted synthetic DMARD arm (tsDMARD; choose among existing medications, currently, tofacitinib, baricitinib, upadacitinib) in patients with active RA despite the use of a TNFi-biologic. In practice, most patients receive another TNFi-biologic, i.e., a second TNFi-biologic first. This is not based on solid evidence, but on arbitrary algorithms often proposed by health insurance plans, and/or physician experience and habit (TNFis launched 22 yrs ago vs. the first tsDMARD 8 years ago vs. first non-TNF-biologic launched 17 years ago). This study will fill a critical knowledge gap by generating CER data for important PROs between these treatment options, switching to a non-TNFi biologic or a tsDMARD in patients with active RA despite the use of a TNFi-biologic.

Type: Interventional

Start Date: Sep 2021

open study

This phase II trial studies the effect of lutetium Lu 177 dotatate compared to the usual treatment (everolimus) in treating patients with somatostatin receptor positive bronchial neuroendocrine tumors that have spread to other places in the body (advanced). Radioactive drugs, such as lutetium Lu 177 dotatate, may carry radiation directly to tumor cells and may reduce harm to normal cells. Lutetium Lu 177 dotatate may be more effective than everolimus in shrinking or stabilizing advanced bronchial neuroendocrine tumors.

Type: Interventional

Start Date: Feb 2023

open study