Retroperitoneal Fibrosis

Researchers are investigating the disease progression and treatment responses of patients with idiopathic retroperitoneal fibrosis (IRPF), a rare condition that causes inflammation and fibrosis around major abdominal arteries and can lead to kidney problems. This study focuses on Chinese patients diagnosed with IRPF to better understand their clinical features, demographics, laboratory and imaging results, and treatment outcomes. It is a prospective cohort study enrolling patients from across China. Patients with definite or possible IRPF will be included. Definite IRPF is identified by imaging showing retroperitoneal masses around the abdominal aorta and iliac arteries with ureteral involvement, no disease progression after glucocorticoid treatment, and specific histopathological signs of inflammation. Possible IRPF includes the imaging and treatment response criteria without biopsy confirmation. Patients with secondary forms of RPF or other autoimmune diseases are excluded. Data will be collected via an online platform, including symptoms, lab tests, imaging, pathology, treatment, and side effects. Participants will provide informed consent and contribute data over several years. Researchers will monitor organ involvement as the primary outcome over five years and assess treatment response, disease relapse, correlation between disease activity and relapse, survival rates, and imaging features during follow-up. Statistical analysis will summarize clinical and laboratory characteristics to improve understanding of IRPF and its management in the Chinese population.

Researchers are evaluating the safety and tolerability of inhaled mosliciguat combined with inhaled treprostinil in adults with pulmonary hypertension associated with interstitial lung disease (PH-ILD). This Phase 2, open-label, multi-center study focuses on participants who already use inhaled treprostinil to manage their condition. The trial aims to gather important safety information about this combination treatment in this specific lung disease population. Participants will receive daily inhaled mosliciguat during a 16-week open-label treatment period. After this period, those who complete the initial phase may continue to receive mosliciguat in an extension phase lasting beyond 16 weeks. Mosliciguat will be delivered using a dry powder inhaler device, and doses may vary among three different levels. All participants will continue treatment with inhaled mosliciguat during the extension. Throughout the study, researchers will monitor participants for treatment-emergent adverse events, serious adverse events, and any side effects leading to discontinuation, with assessments at baseline and at week 16. Participants must be able to perform a six-minute walk of at least 100 meters. The study includes imaging confirmation of interstitial lung disease and right heart catheterization to confirm pulmonary hypertension. The total participation duration may extend beyond 16 weeks depending on continuation in the extension phase.

Researchers are evaluating the safety and effectiveness of the Stenae Laparoendoscopic Surgical System C1000 for benign ureteral reconstruction surgery. This study is a single-arm, open-label, prospective, non-randomized cohort design including patients aged 18 to 80 years who require upper urinary tract repair. The trial aims to assess surgical outcomes such as treatment success and surgical non-referral rates. The study involves using the Cornerstone C1000 Robotic Surgery System, a minimally invasive device controlled by the surgeon for performing surgeries like pyeloplasty, stenectomy and reanastomosis, bladder reimplantation, and various patch repairs including oral mucosa and appendix patches. Different procedures such as ileal and colonic substitution ureter are also included. The treatment period lasts approximately 9 months, with 6 months for enrollment and 3 months follow-up. Participants will undergo surgery using the robotic system and be monitored for surgery-related outcomes including operating time, blood loss, pain scores, surgeon's physical and mental load, blood transfusion rates, hospital stay length, physician satisfaction, complication rates, adverse events, device defects, readmission, reoperation, and mortality. Follow-up assessments will continue for about 3 months after surgery to evaluate these measures and ensure safety.

Researchers are collecting biological samples from patients with various liver diseases including Non-Alcoholic Fatty Liver Disease (NAFLD), Non-Alcoholic Steatohepatitis (NASH), Hepatitis C (HCV), Hepatitis B (HBV), and Human Immunodeficiency Virus (HIV). This biorepository aims to support research by providing high-quality specimens to help advance understanding and treatment of liver diseases. The project also promotes collaboration among clinicians and researchers and supports training for new investigators to improve patient health nationwide. The biorepository collects blood samples such as serum, plasma, buffy coat, and liver tissue from patients seen in clinics, including rarer liver diseases. Samples are stored and made available for future research to facilitate new discoveries in liver disease mechanisms and therapies. Both children and teenagers are considered special populations for this protocol, and negative control samples from individuals without liver disease are also included. Participants provide samples during clinic visits, and the main measure tracked is the number of samples collected over 52 weeks. This observational study involves no treatment but focuses on gathering clinically annotated human specimens. Participation may involve periodic blood draws and sharing medical information to contribute to liver disease research. The study is open to all genders aged 7 years and older and continues until December 2099.

Researchers are evaluating the effectiveness and safety of laparoscopic renal artery perivascular nerve blockade surgery for patients who have retroperitoneal diseases and hypertension. The study aims to find out if this nerve blockade can reduce blood pressure after surgery and lessen the need for blood pressure medications. It also aims to assess the safety of the procedure and whether it causes any complications. Participants will be randomly assigned to one of two groups. One group will receive surgery related to their primary disease along with the nerve blockade procedure, while the other group will only have the surgery related to their primary disease, such as laparoscopic partial nephrectomy or adrenalectomy. The nerve blockade involves carefully dissecting the renal artery to separate nerve tissue within specific measurements, ensuring surgical safety. During the study, patients will keep their antihypertensive medication unchanged for three months following surgery. After this period, they will have a 24-hour ambulatory blood pressure monitoring visit at the hospital. Researchers will measure blood pressure at three months post-surgery as the primary outcome. Secondary outcomes include blood pressure at other time points, medication use, health status, and pain scores, assessed up to two years after surgery.

Researchers are evaluating the effectiveness of endoscopic Coeliac Artery Release (eCAR) compared to a sham operation in patients suspected of Median Arcuate Ligament Syndrome (MALS). MALS involves significant external compression of the coeliac artery causing chronic disabling abdominal pain, weight loss, and related symptoms. The study aims to resolve ongoing debates about this condition by providing evidence on whether surgical release improves symptoms and quality of life. Participants are randomly assigned to either the eCAR procedure or a sham operation, both involving similar incisions to maintain blinding. The eCAR involves cleaving the median arcuate ligament via an endoscopic retroperitoneal approach using a specific four-trocar technique. The sham group undergoes incisions and anesthesia for a similar duration without ligament release. Procedures are performed by experienced surgeons, and patients and observers remain blinded to treatment allocation. During the study, participants are followed up for two years with assessments of abdominal pain, symptom relief using VAS and PGI-I scales, quality of life, productivity loss, healthcare use, and anatomical success measured by imaging. Safety is monitored through complication classification and additional treatments recorded. The primary outcome is symptom reduction measured six months after randomization. This comprehensive monitoring helps determine the value of eCAR for managing MALS and its impact on patient wellbeing and healthcare costs.

Chronic liver disease, especially cirrhosis, is a leading cause of death worldwide with limited medical treatments that can alter its progression. Malnutrition plays a significant role in worsening cirrhosis outcomes. Copper, an essential trace metal regulated mainly by the liver, has been found deficient in many liver disease patients and linked to severe disease, higher infection rates, and increased mortality. This research investigates whether copper supplementation can improve biochemical changes and patient outcomes in cirrhosis. Participants in this pilot randomized, placebo-controlled, crossover trial will receive oral copper gluconate 4 mg daily or placebo for a 6-week period, followed by a 3-week washout, then switch to the alternate treatment for another 6 weeks. Two groups alternate the order: one starts with copper then placebo, and the other starts with placebo then copper. The study evaluates copper's biochemical effects, safety, and patient-reported outcomes during these periods. Throughout the 15-week trial, participants will be monitored at baseline, after each 6-week intervention, and after the washout period. Researchers will measure plasma copper concentrations, biomarkers of copper status, safety indicators, nutritional and functional status, and patient-reported outcomes. This detailed monitoring will assess copper supplementation's impact on cirrhosis-related biochemical and clinical changes under careful safety observation.

Researchers are investigating the role of diaphragm function as a biomarker in patients with respiratory diseases such as Asthma, COPD, Fibrosis, and Pulmonary Hypertension. This study aims to better understand the causes of breathlessness (dyspnea) and how diaphragm dysfunction may relate to symptoms and disease outcomes. The research will include up to 800 patients from these four groups to explore the connection between respiratory muscle function and patient health. Participants will undergo comprehensive respiratory muscle testing, including diaphragm and intercostal muscle ultrasounds, lung function tests, exercise tolerance evaluations, and symptom questionnaires during one day at the study center. Follow-up will be conducted primarily by phone at 3, 6, 12, and 18 months after the initial visit. A smaller subgroup of patients starting certain approved drug therapies will have additional in-person follow-ups with repeated non-invasive measurements. Throughout the study, participants will complete various assessments such as dyspnea scales, walking tests, breathlessness ratings, and respiratory questionnaires. Researchers will also monitor hospitalizations and steroid use related to exacerbations to assess the prognostic significance of diaphragm dysfunction. The study includes detailed measurement of respiratory pressures, blood gas analysis, and electromyography, with participation lasting up to 18 months after enrollment.

Researchers are studying how the length of ureteral rest—the time between changing a ureteral stent to a percutaneous nephrostomy tube—affects the length of ureteral strictures before surgery to repair the ureter. The study focuses on patients with ureteral strictures who are preparing for ureteral reconstruction surgery. The goal is to better understand this timing to potentially improve surgical outcomes. Participants will undergo antegrade and retrograde pyelogram procedures before their ureteral repair surgery. These imaging tests are used to measure the ureteral stricture length and quality at two time points: 2 weeks and 6 weeks after the procedure. The study is observational, and all participants will receive these imaging evaluations as part of the research. During the study, participants will be monitored through these imaging assessments to evaluate the stricture length and whether the narrowed segment is classified as narrowed or obliterated. The study will review these measurements to assess the effect of ureteral rest duration. Participation involves signing informed consent and completing the imaging procedures, with no additional treatments or interventions required. The study is expected to continue until November 2030.

This research focuses on adult patients diagnosed with idiopathic retroperitoneal fibrosis (IRF) to study the early discontinuation of steroid treatment in patients with negative FDG-PET/CT scans. The study is a prospective multicenter cohort design assessing the effects of prednisone administered orally for 9 to 21 months. It aims to better understand IRF relapse rates after stopping steroids and the role of FDG-PET/CT imaging in disease activity. Patients with positive FDG-PET/CT scans receive oral prednisone at a starting dose of 1 mg/kg/day for one month, followed by a tapering schedule to reduce the dose below 10 mg/day by six months and below 7.5 mg/day by nine months. Those with negative FDG-PET/CT results are excluded from the study. Follow-up visits are scheduled at 6, 9, 12, 15, and 21 months, with additional visits if relapse occurs. Treatment continuation or discontinuation after nine months depends on remission status and imaging results. Participants undergo clinical exams, blood and urine tests, abdominal CT scans, and FDG-PET/CT imaging at baseline and during follow-ups. Researchers monitor symptoms, medication adherence, adverse events, and disease progression. The primary outcome is the cumulative IRF relapse rate 12 months after stopping steroids. The study includes safety assessments and analyzes changes in disease hypermetabolism over time, lasting until November 2028.