Idiopathic disease

This study looked at whether the drug riociguat could improve right heart size and function in people with two types of pulmonary hypertension (PAH and CTEPH). It planned to enroll 30 adults, but was terminated early, so results are limited. The goal was to use echocardiograms to…

This study tests whether an additional dose of a COVID-19 vaccine can improve immune protection in adults and children with autoimmune diseases like rheumatoid arthritis, lupus, multiple sclerosis, and others who are taking immunosuppressive medications. Participants who had a we…

This study tested the anti-inflammatory drug naproxen sodium for treating obsessive-compulsive symptoms in children aged 6-15 with PANDAS, a condition linked to strep infections. The trial was double-blind and placebo-controlled, but it was terminated early after enrolling only 2…

This study looked at whether adding a drug called G-CSF to the usual treatment (ATG and Cyclosporin A) helps people with newly diagnosed severe aplastic anemia. Severe aplastic anemia is a rare condition where the bone marrow stops making enough blood cells. The trial included 20…

This study tested a device called the Braive Growth Modulation System in 10 children with scoliosis (curved spine). The goal was to see if it could safely correct the curve as the child grows. The study was stopped early, so results are limited.

This study tested a new medicine, GSK3915393, in 158 people with idiopathic pulmonary fibrosis (IPF), a chronic lung disease that causes scarring and breathing difficulty. The goal was to see if the drug could slow lung function decline compared to a placebo. The trial was termin…

This study looked at whether wearing a back brace can stop spine curves from getting worse in teens with scoliosis. About 383 teens with mild to moderate curves were either assigned to wear a brace for at least 18 hours a day or just be observed. The goal was to see if bracing le…

This early-stage study tested a new drug called VB119 in 6 adults with primary membranous nephropathy, a kidney disease. The main goal was to check safety and how the body handles the drug, not to cure the disease. The study was terminated early, so results are limited.

This study tested the drug vixarelimab against a placebo in people with idiopathic pulmonary fibrosis (IPF) or systemic sclerosis-associated interstitial lung disease (SSc-ILD). The goal was to see if it could preserve lung function over 52 weeks. The trial was terminated early, …

This study tested an oral drug called INF904 in people with moderate to severe chronic spontaneous urticaria (long-term hives) or hidradenitis suppurativa (a painful skin condition). The trial was open-label and lasted 28 days of treatment plus follow-up. It aimed to check safety…

This study tested whether metformin (a diabetes drug) and doxycycline (an antibiotic) could help treat head and neck cancer when taken before surgery. The trial aimed to see if these drugs could change certain proteins in the tumor's supportive tissue. Only 7 people took part bef…

This phase 3 trial tested aflibercept (Eylea) eye injections against placebo in 16 people with macular telangiectasia type 1, a rare condition that causes vision loss. The study aimed to see if aflibercept could reduce swelling in the macula. However, the trial was terminated ear…

This study tested the drug riociguat in people with early pulmonary vascular disease, a condition where blood vessels in the lungs narrow, making the heart work harder. The trial included 35 adults with connective tissue disease or an inherited form. Participants received either …

This study tested new single treatments for idiopathic pulmonary fibrosis (IPF), a lung disease that causes scarring and makes breathing hard. It included 46 adults aged 40 and older with mild to moderate IPF. The main goal was to see if the treatments improved lung function, mea…

This study tested a medicine called Hizentra (IgPro20) in 134 adults with dermatomyositis, a rare disease causing muscle weakness and skin rashes. The goal was to see if weekly shots under the skin could improve symptoms better than a placebo. The study was stopped early, but res…

This study looked at whether adding cyclosporine to rituximab is more effective than rituximab alone for treating idiopathic membranous nephropathy, a kidney disease that causes protein leakage. Only 12 adults with severe proteinuria took part before the study ended early. The go…

This study tested an experimental drug called HZN-825 in people with idiopathic pulmonary fibrosis (IPF), a lung disease that causes scarring and makes breathing hard. The trial aimed to see if the drug could slow the decline in lung function over a year. About 153 adults with IP…

This study tested the drug nintedanib (Ofev) in 11 people with myositis-related interstitial lung disease, a condition where lung scarring occurs alongside muscle inflammation. The goal was to see if the drug is safe and tolerable when added to standard immune-suppressing treatme…

This early-phase study tested a new radioactive imaging drug called 11C-MC1 to see if it can help detect inflammation in people with rheumatoid arthritis or idiopathic inflammatory myopathy, as well as in healthy volunteers. Participants received PET/CT scans after an injection o…

This study tested whether osteopathic care (hands-on manipulation) can help reduce pain in people with fibromyalgia. 32 participants received osteopathic treatments over 8 months, with a follow-up 3 months later. The goal was to see if this approach could improve pain and quality…

This study aimed to find the ideal dose of a spinal pain medicine (hydromorphone) for children undergoing back surgery for scoliosis. Researchers tested several doses in 31 patients to balance good pain control with fewer side effects. The trial was stopped early, so the results …

This study looked at whether a brief, noninvasive brain stimulation technique called tDCS could immediately reduce pain and improve function in people with fibromyalgia or chronic fatigue syndrome. About 20 participants received one session, and researchers measured changes in pa…

This Phase 2 trial tested whether FSD201 tablets could reduce chronic pain in adults with mast cell activation syndrome. The study planned to enroll many participants but was terminated early with only 2 people. No results are available to show if the drug works or is safe.

This study aimed to see if a personalized physical activity program, with one face-to-face session followed by phone coaching, could improve quality of life for people with fibromyalgia compared to standard group exercise classes. The trial was terminated after enrolling only one…

This study tested an oral drug called CIN-102 in 72 adults with gastroparesis (delayed stomach emptying) caused by diabetes or unknown reasons. Participants received one of three doses of CIN-102 or a placebo to see if it improved stomach emptying and reduced nausea. The trial wa…

This study tested an experimental drug called BI 1839100 in adults with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF) who have a chronic cough. Participants took different doses of the drug or a placebo for 12 weeks. The goal was to see if the drug c…

This Phase 2 trial tested an experimental drug called EP262 in 113 people with chronic spontaneous urticaria (long-lasting hives with no clear cause). Participants took either EP262 or a placebo daily for 6 weeks, while continuing their usual antihistamines. The study aimed to se…

This study aimed to find the best amount of exercise needed to improve dizziness symptoms in people with balance disorders. Researchers used virtual reality (VR) to measure how much exercise helps and compared VR exercises to standard physical therapy. The study included people w…

This study tested whether a small device that sends mild electrical pulses to the vagus nerve (through the ear) can reduce pain and inflammation in children with juvenile idiopathic arthritis. The trial planned to enroll children ages 5 to 18, but was terminated early after only …

This study looked at whether giving teenagers a numbing medicine (lidocaine) through an IV during and after spine surgery could lower their need for strong painkillers (opioids) and help them recover faster. The trial planned to include 15 teens aged 12-18 with scoliosis, but it …

This study looked at two different combinations of anesthesia medicines given through an IV to teenagers having surgery for scoliosis. The goal was to see if one combination leads to less pain and less need for opioid painkillers after surgery. The study was small and ended early…

This study tested a wearable device called JessieHug for newborns and infants. The goal was to see if parents find it easy to use, if babies tolerate wearing it, and if it collects accurate health data like oxygen levels. About 31 healthy infants participated, and parents used th…

This study looked for new markers in blood and joint fluid to help doctors tell the difference between septic arthritis (a joint infection) and juvenile idiopathic arthritis (an inflammatory condition) in children. Researchers planned to analyze samples from 100 children under 15…

This study aimed to describe the features and natural history of idiopathic interstitial pneumopathy, a group of rare lung diseases, in both children and adults. Researchers planned to collect data from 1,600 participants, including medical history, imaging, and genetic tests, to…

This study aimed to see if a tiny camera on a thin tube (bronchoscopy) could help doctors diagnose diffuse lung diseases without needing a surgical lung biopsy. The camera takes live microscopic images of the air sacs. The study was stopped early, but it involved 38 adults with c…

This study aimed to compare the gut bacteria of children newly diagnosed with juvenile idiopathic arthritis, type 1 diabetes, or inflammatory bowel disease with those of their healthy siblings. Researchers wanted to see if differences in gut microbes and diet could explain why so…

This study looked at whether using a CPAP machine at night could reduce abnormal acid reflux and lung inflammation in people with idiopathic pulmonary fibrosis (IPF) who also have sleep apnea. Only 7 people enrolled before the study was terminated early. The goal was to measure c…

This observational study measured vagal nerve activity after eating in 43 people with gastroparesis (a stomach disorder) and healthy volunteers. The goal was to establish normal and abnormal nerve function patterns. The study used an FDA-approved device to track heart-based nerve…