Chronic lymphocytic leukemia (CLL) is the most common leukemia (cancer of blood cells). The purpose of this study is to assess the safety of venetoclax in combination with obinutuzumab or acalabrutinib in the treatment of CLL. Adverse events and change in disease activity will be assessed. Venetoclax in combination with obinutuzumab or acalabrutinib is being investigated in the treatment of CLL. Study doctors put the participants in 1 of 4 groups, called treatment arms. Participants will receive oral venetoclax in combination with intravenously (IV) infused obinutuzumab or oral acalabrutinib at in different dosing schemes as part of treatment. Approximately 170 adult participants with CLL who are being treated with venetoclax will be enrolled in the study in approximately 80 sites worldwide. Participants in Arm A will receive oral venetoclax in combination with IV infused obinutuzumab, with a 5 week venetoclax ramp up. Participants in Arm B will receive oral venetoclax in combination with oral acalabrutinib, with a 5 week venetoclax ramp up. Participants in Arm C and Arm D will receive oral venetoclax in combination with oral acalabrutinib, with differing venetoclax ramp up periods. The total study duration is approximately 28 months. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

Type: Interventional

Start Date: Aug 2024

open study

The goal of this clinical trial is to assesses the safety of autologous RD2 Ver.02 as compared to a control for managing transsphincteric and intersphinsteric anal fistulas. The main questions it aims to answer are: Assess the safety and efficacy of RD2 Ver.02 in anal fistula application, compared to control. Complication rate by 6 months of anal fistula treatment with RD2 Ver.02 compared to control. Recurrence of anal fistula at 12 months post-treatment Incidence of perirectal infection by 6 months in anal fistulas treated with RD2 Ver.02 compared to control. Patients will be randomized in to 2 arms. For all patients, blood will be drawn to ensure the blinding of the patients, the fistula will be evaluated and debrided, and then the internal fistula opening will be suture-closed, and a water leak test will be performed to ensure sealing. Following the water leak test, In the treatment arm, the patient's own coagulating blood will be applied into the entire fistula tract, allowing it to clot and serve as a provisional matrix inside the fistula tract. In the control arm, the blood sample will be discarded and saline will be applied to the fistula tract.

Type: Interventional

Start Date: Jan 2024

open study

This phase III trial compares the effect of bevacizumab and osimertinib combination vs. osimertinib alone for the treatment of non-small cell lung cancer that has spread outside of the lungs (stage IIIB-IV) and has a change (mutation) in a gene called EGFR. The EGFR protein is involved in cell signaling pathways that control cell division and survival. Sometimes, mutations in the EGFR gene cause EGFR proteins to be made in higher than normal amounts on some types of cancer cells. This causes cancer cells to divide more rapidly. Osimertinib may stop the growth of tumor cells by blocking EGFR that is needed for cell growth in this type of cancer. Bevacizumab is in a class of medications called antiangiogenic agents. It works by stopping the formation of blood vessels that bring oxygen and nutrients to tumor. This may slow the growth and spread of tumor. Giving osimertinib with bevacizumab may control cancer for longer and help patients live longer as compared to osimertinib alone.

Type: Interventional

Start Date: Dec 2020

open study

This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine clinical care in the US. The study has a planned maximum duration of 5 years for the first enrolled patients, including a 24-month enrollment period and a minimum of 2 years of follow-up.

Type: Observational

Start Date: Oct 2025

open study

Foralumab is a human anti-CD3 monoclonal antibody being developed for the treatment of autoimmune and inflammatory diseases. The goal of this Phase 2a, randomized, double-blind placebo-controlled, multicenter dose-ranging study is to evaluate the use of nasal foralumab in patients with non-active secondary progressive multiple sclerosis (SPMS). The primary objectives that this study aims to answer are: 1. To determine the safety and tolerability of 50 μg/dose and 100 μg/dose of foralumab nasal compared to placebo 2. To investigate the effect of foralumab relative to placebo on the change from baseline [18F]PBR06-positron emission tomography (PET) scans for microglial activation, after 12 weeks (3) months of study treatment.

Type: Interventional

Start Date: Nov 2023

open study

Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. This phase III trial aims to maintain excellent outcomes in patients with very low risk rhabdomyosarcoma (VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine and dactinomycin (VA) and examines the use of centralized molecular risk stratification in the treatment of rhabdomyosarcoma. Another aim of the study it to find out how well patients with low risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment. Finally, this study examines the effect of therapy intensification in patients who have RMS cancer with DNA mutations to see if their outcomes can be improved.

Type: Interventional

Start Date: Aug 2022

open study

This phase II/III trial compares the effect of adding durvalumab to chemotherapy versus chemotherapy alone before surgery in treating patients with upper urinary tract cancer. Immunotherapy with monoclonal antibodies, such as durvalumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Chemotherapy drugs, such as methotrexate, vinblastine, doxorubicin, cisplatin, and gemcitabine work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Durvalumab in combination with chemotherapy before surgery may enhance the shrinking of the tumor compared to chemotherapy alone.

Type: Interventional

Start Date: Nov 2021

open study

This is a prospective, observational cohort study. Participants with non-idiopatic pulmonary fibrosis, interstitial lung disease (ILD) will be followed for 24 months to systematically collect clinical, imaging, and biospecimen data. The primary objective is to optimize progressive pulmonary fibrosis (PPF) classification and establish PPF incidence for key ILD subtypes. Additional exploratory objectives are to 1) Prospectively validate a novel PPF classifier and assess performance durability over time, and 2) Determine whether multi-dimensional PPF prediction outperforms component approaches.

Type: Observational

Start Date: May 2025

open study

The REACH study is for people with CF who do not take cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The goal of the REACH study is to collect research data, including health data and specimens, from people with CF who do not take CFTR modulators. This data may be used to inform CF research, help design CF clinical trials and support the development of new treatments for people with CF who do not take CFTR modulators. Another goal of this study is to learn about research involvement for people with CF who do not take CFTR modulators, engage them in research, and give them an opportunity to learn about what is involved in participating in a CF research study.

Type: Observational

Start Date: Sep 2024

open study

The purpose of this study is to perform a randomized controlled trial among 180 pregnant women with type 2 diabetes mellitus (T2DM) comparing continuous glucose monitor (CGM) use to the standard of care of multiple daily fingerstick glucose monitoring and its impact on large for gestational age infants, maternal glycemic control, patient satisfaction, and additional adverse perinatal outcomes.

Type: Interventional

Start Date: May 2024

open study

This is a multi-center retrospective chart review to compile a data repository of the management and outcomes of children with biliary atresia. Overall, investigators aim to evaluate which specific factors contribute to improved patient outcomes, to help guide potential improvements in patient care and resource utilization.

Type: Observational

Start Date: May 2024

open study

A prospective, multicenter, single arm, interventional study. The target patient population for this study are adult subjects with WNBAs of the anterior and posterior intracranial circulation. The primary effectiveness outcome of the study is adequate intracranial aneurysm occlusion on the 1 year angiogram as adjudicated by a core laboratory.

Type: Interventional

Start Date: Aug 2022

open study

The goal of this clinical trial is to learn how safe and tolerable EPI-321 is and whether there may be early signs it is working in male or female adult (18 to 75 years) participants with facioscapulohumeral muscular dystrophy (FSHD) Type 1 condition. The main questions it aims to answer are: How safe is EPI-321 and how well can people handle it over time? How does EPI-321 interact with its target and does it show early signs of working? Participants will receive a single dose of EPI-321 through a vein while being closely watched in a hospital and visit the clinic regularly for tests and checkups for about 5 years after getting EPI-321.

Type: Interventional

Start Date: May 2025

open study

This 4-year study will randomize 1,000 people with co-occurring opioid use and mental health disorders (COD) at medication for opioid use disorder (MOUD) clinics to evaluate the effectiveness of MISSION, a multi-component team approach, or its components with MOUD versus MOUD alone, as well as the incremental benefits of MISSION or its components for improving outcomes. We expect that individuals receiving MISSION or its parts + MOUD will show greater improvement over MOUD alone on: engagement, substance use, and mental health.

Type: Interventional

Start Date: Mar 2022

open study

This is an Open-label, Prospective, Multicenter Study to Assess the SAfety of Regional Citrate Anticoagulation Delivered by the multiFiltratePRO system in Adult Patients Requiring Continuous Renal Replacement Therapy (SARCA Study).

Type: Interventional

Start Date: Oct 2023

open study

A prospective, single-arm, multi-center post-market surveillance study of the BD® WavelinQ™ EndoAVF System for the Creation of Arteriovenous (AV) Fistula in Patients Requiring Dialysis.

Type: Interventional

Start Date: Mar 2021

open study

The purpose of this study is to look at pulmonary exacerbations in people with cystic fibrosis (CF) that need to be treated with antibiotics given through a tube inserted into a vein (intravenous or IV). A pulmonary exacerbation is a worsening of respiratory symptoms in people with CF that needs medical intervention. Both doctors and CF patients are trying to understand the best way to treat pulmonary exacerbations. This study is trying to answer the following questions about treating a pulmonary exacerbation: - Do participants have the same improvement in lung function and symptoms if they are treated with one type of antibiotic (called beta-lactams or β-lactams) versus taking two different types of antibiotics (tobramycin and β-lactams)? - Is taking one type of antibiotic just as good as taking two types?

Type: Interventional

Start Date: Apr 2023

open study

This is a Phase 1 study of IMS001, given as a single dose to subjects with Multiple Sclerosis who experience inadequate response and/or intolerability to disease modifying treatments. IMS001 is a human embryonic cell derived (hESC) mesenchymal stem cell (MSC). MSCs have the potential to modulate disease course.

Type: Interventional

Start Date: Aug 2021

open study

KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB).

Type: Interventional

Start Date: Jun 2025

open study

This study is open to adults and adolescents aged 12 to under 18 with bronchiectasis. People can participate in this study if they produce sputum and have had flare-ups (also called exacerbations). The purpose of this study is to find out whether a medicine called BI 1291583 helps people with bronchiectasis. Participants are put into 2 groups randomly, which means by chance. One group takes BI 1291583 tablets and the other group takes placebo tablets. A placebo tablet looks like the BI 1291583 tablet but does not contain any medicine. Participants take 1 tablet once a day for up to 1 year and 6 months. Participants are in the study for up to 1 year and 8 months. During this time, participants visit the study site up to 10 times and get about 13 phone calls from the site staff. Participants regularly complete a diary on a smartphone about their bronchiectasis symptoms and study doctors regularly check for any changes. The study doctors document when participants experience flare-ups. The number of flare-ups is compared between the participants who receive BI 1291583 and those who receive the placebo. The study doctors also regularly check participants' health and take note of any unwanted effects.

Type: Interventional

Start Date: Jun 2025

open study

Primary immune thrombocytopenia (ITP) is a condition where the immune system mistakenly destroys platelets, which are cells that help stop bleeding. This leads to a low number of platelets, making it easier to bruise or bleed. The main aim of this study is to learn whether mezagitamab, when given just under the skin (subcutaneously [SC]), is effective in keeping the platelet count of adults with ITP stable when compared to a placebo. A placebo looks like medicine but doesn't have any active ingredients in it. The participants will be treated with mezagitamab for up to 6 months. During the study, participants will visit their study clinic several times. Participants who complete the TAK-079-3002 study or do not have any response to study treatment by week 16 (according to study criteria) will be given the opportunity to participate in a continuation study to receive open label mezagitamab (if they are eligible and the site is able to open the continuation study).

Type: Interventional

Start Date: Feb 2025

open study

The purpose of this study is to determine if the method for injecting local anesthesia affects patients' pain and opioid usage after surgery. The investigators will compare subcutaneous anesthesia, injections of anesthesia under the skin, to a method called erector spinae plane block (ESPB). An ESPB injection involves placing local anesthesia along the muscles and bones in the back, using a special type of x-ray called fluoroscopy for guidance. The Investigators will use patient reported outcomes (PROs) and track subjects' opioid usage to find out if there is a difference between ESPB and subcutaneous anesthesia. The investigators hypothesize that patients who get ESPB injections will use less opioids and report less pain after lumbar fusion surgery compared to patients who receive subcutaneous anesthesia injections.

Type: Interventional

Start Date: Sep 2024

open study

This phase III trial studies using risk factors in determining treatment for children with favorable tissue (histology) Wilms tumors (FHWT). Wilms Tumor is the most common type of kidney cancer in children, and FHWT is the most common subtype. Previous large clinical trials have established treatment plans that are likely to cure most children with FHWT, however some children still have their cancer come back (called relapse) and not all survive. Previous research has identified features of FHWT that are associated with higher or lower risks of relapse. The term "risk" refers to the chance of the cancer coming back after treatment. Using results of tumor histology tests, biology tests, and response to therapy may be able to improve treatment for children with FHWT.

Type: Interventional

Start Date: Apr 2025

open study

The purpose of this study is to evaluate the effectiveness and safety of ozanimod (RPC1063) in achieving and maintaining clinical remission. Ozanimod will be administered orally to pediatric participants with moderate to severe active ulcerative colitis (UC) who have had an inadequate response to conventional therapy.

Type: Interventional

Start Date: May 2022

open study

RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus

Type: Interventional

Start Date: Feb 2024

open study