Miami, FL Paid Clinical Trials

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications. Read Less

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. Read Less

This study will evaluate the efficacy and safety of erenumab in migraine prevention in children (6 to <12 years) and adolescents (12 to <18 years) with episodic migraine. The study hypothesis is that in pediatric participants with episodic migraine, the combined erenumab dose group has a greater reduction from baseline to week 9 through week 12 (month 3) in monthly migraine days (MMDs) when compared with placebo in the double-blind treatment phase (DBTP). Read Less

To compare the efficacy and safety of remibrutinib versus teriflunomide in patients with relapsing multiple sclerosis (RMS) Read Less

The Beads vs Vac trial is a multi-centre randomized controlled trial of 312 participants with a severe open tibia fracture requiring multiple irrigation and debridement surgeries. Eligible participants will be randomized to receive either an antibiotic bead pouch or negative pressure wound therapy (NPWT) for their temporary open fracture wound management. Outcomes will be assessed at 6 weeks, 3 months, and 6 months post-surgery. The primary outcome will be a composite outcome to evaluate clinical status six months after randomization. Components of the composite outcome will be hierarchically assessed in the following order: 1) all-cause mortality, 2) injury-related amputation of the lower extremity, 3) unplanned reoperation to manage wound complications, infection, or delayed fracture healing, and 4) clinical fracture healing as assessed using the Functional IndeX for Trauma (FIX-IT) instrument. The secondary outcomes will independently assess the four components of the primary outcome. This is a Phase III trial. Read Less

This is a parallel group, Phase 3, multinational, multicenter, randomized, double blind, placebo-controlled, 3-arm monotherapy study for treatment of participants diagnosed with moderate to severe atopic dermatitis (AD), whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. The purpose of this study is to measure the efficacy and safety of treatment with amlitelimab solution for SC injection compared with placebo in participants with moderate to severe AD aged 12 years and older. Study details include: At the end of the treatment period, participants will have an option to enter a separate study: the blinded extension study EFC17600 (ESTUARY). For participants not entering the blinded extension Study EFC17600 (ESTUARY), the study duration will be up to 44 weeks including a 2 to 4-week screening, a 24-week randomized double-blind period, and a 16-week safety follow-up. For participants entering the blinded extension Study EFC17600 (ESTUARY), the study duration will be up to 28 weeks including a 2 to 4-week screening and a 24-week randomized double-blind period. The total treatment duration will be up to 24 weeks. The total number of visits will be up to 10 visits (or 9 visits for those entering the blinded extension study EFC17600] (ESTUARY). Read Less

To evaluate the efficacy of romiplostim for the treatment of CIT in patients receiving chemotherapy for the treatment of NSCLC, ovarian cancer, or breast cancer measured by the ability to administer on-time, full-dose chemotherapy Read Less

The goal of this study is to evaluate the efficacy of stepped care strategies to improve ART adherence among adult Latino MSM with HIV using a sequential, multiple assignment, randomized trial (SMART). The trial will compare a stepped care strategy of delivering TXTXT ("Treatment Text") first and stepping up to remote patient navigation for non-responders vs. a stepped care strategy of delivering TXTXT + e-Navigation first and stepping up to EMA-supported e-Navigation for non-responders. Both TXTXT and the foundations of the e-Navigation interventions are CDC evidence-based interventions (EBI). We propose to use a SMART design which explicitly allows building, testing, and optimizing stepped care strategies without compromising rigor or randomization. We propose three specific aims: Aim 1. Compare the immediate (6-month) and sustained (9- and 12-month) efficacy of two static (non-stepped) treatment regimens (TXTXT alone vs. TXTXT + e-Navigation) on ART adherence and viral suppression among Latino MSM with HIV. Hypothesis 1a. TXTXT + e-Navigation will be more efficacious than TXTXT alone. Aim 2. Compare the immediate (6-month) and sustained (9- and 12-month) efficacy of two stepped care strategies (TXTXT with added e-Navigation for non-responders vs. TXTXT + e-Navigation with added EMA support for non-responders) on ART adherence and viral suppression among Latino MSM with HIV. Hypothesis 2a: TXTXT + e-Navigation with added EMA support for non-responders at the 3-month follow-up will be more efficacious than TXTXT with added e-Navigation for non-responders at the 3-month follow-up. Aim 3. Identify baseline and time-varying moderators on the association between stepped care strategy and ART adherence and viral suppression among Latino MSM with HIV. Hypotheses 3a-c: TXTXT with added e-Navigation for non-responders will be less efficacious than TXTXT + e-Navigation with added EMA support for non-responders for individuals who are: (a) older at baseline, or report (b) substance use, or (c) symptoms of depression between baseline and the 3-month follow-up. Read Less

Primary Objective: Evaluate the efficacy of itepekimab compared with placebo on the annualized rate of acute moderate-or-severe COPD exacerbations in former smokers with moderate-to-severe COPD Secondary Objectives: Evaluate the efficacy of itepekimab compared with placebo on pulmonary function in former smokers with moderate-to-severe COPD Evaluate the efficacy of itepekimab compared with placebo on occurrence of acute exacerbation of COPD (AECOPD) in former smokers with moderate-to-severe COPD Evaluate the efficacy of itepekimab compared with placebo on severe AECOPD in former smokers with moderate-to-severe COPD Evaluate the efficacy of itepekimab compared with placebo on corticosteroid-treated AECOPD in former smokers with moderate-to-severe COPD Evaluate the efficacy of itepekimab compared with placebo on respiratory symptoms in former smokers with moderate-to-severe COPD Evaluate the efficacy of itepekimab compared with placebo on Forced Expiratory Volume in 1 second (FEV1) slope in former smokers with moderate-to-severe COPD Evaluate the efficacy of itepekimab compared with placebo on health-related quality of life (HRQoL) as assessed by St. George's Respiratory Questionnaire (SGRQ) in former smokers with moderate-to-severe COPD Evaluate the safety and tolerability of itepekimab in former smokers with moderate-to-severe COPD Evaluate the pharmacokinetic (PK) profile of itepekimab in former smokers with moderate-to-severe COPD Evaluate immunogenicity to itepekimab in former smokers with moderate-to-severe COPD Read Less

Semaglutide is a medicine studied in patients with NASH. Semaglutide is a well-known medicine, which is already used by doctors to treat type 2 diabetes in many countries. Participants will either get semaglutide or a dummy medicine - which treatment participants get is decided by chance. Participants will need to inject themselves with medicine under the skin. Participants will need to do this once a week. The study will last for about 5 years. Participants will have up to 21 clinic visits and 9 phone calls with the clinical staff during the study. Some of the clinic visits may be spread over more than one day. Participants with other chronic liver diseases cannot take part in this study. Women cannot take part in the study if they are pregnant, breast-feeding or plan to become pregnant during the study period. Read Less

The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of VX-147 in adult and pediatric participants with apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease. Read Less

The purpose of this study is to evaluate the efficacy and safety of rocatinlimab in monotherapy and combination therapy treatment in adolescent subjects. Read Less