Hemophilia B

This Phase 1 trial tests a gene therapy that uses a lentivirus to deliver a working clotting factor IX gene into patients' own stem cells. Ten males with severe hemophilia B will receive the modified cells. The goal is to see if it's safe and can raise clotting factor levels, pot…

This trial tests whether inebilizumab, an antibody that wipes out certain immune cells called B cells, can improve physical function in adults with ME/CFS that started after an infection. Participants receive three infusions over six months, and the study measures changes in mobi…

This study tests whether a mechanical 3D ultrasound device can detect joint damage in people with hemophilia as accurately as a full ultrasound or MRI. Eleven participants will have their ankles scanned with the new device. If it works well, it could offer a faster, less expensiv…

This study tests whether a low dose of aripiprazole, a drug used for psychiatric conditions, can reduce fatigue and improve thinking in adults with Long COVID or other post-infection syndromes. About 138 participants will receive either the drug or a placebo for 8 weeks, then swi…

This observational study will take small liver samples from 8 adults with hemophilia A or B who had gene therapy at least 6 months ago. Researchers want to see how the gene therapy affected liver cells, including how many cells still carry the therapy and whether there is any liv…

This study uses ultrasound to examine the joints of adults with mild hemophilia, a group that often goes understudied. Researchers will scan six joints (ankles, knees, elbows) to look for early signs of damage that may not cause symptoms yet. The goal is to see how common joint p…

This study compares how well adults with hemophilia (ages 18–35) perform a 30-second sit-to-stand test versus healthy peers of the same age. The goal is to see if this simple test can detect differences in leg strength and endurance linked to joint damage from bleeding. Participa…

This study tests a smart sharps disposal container that helps hemophilia patients safely throw away used needles at home. About 23 adults with moderate or severe hemophilia will use the device for several weeks. Researchers want to see if patients find it easy to use and if they …

This study looks at how the sedative dexmedetomidine affects the smallest blood vessels in people with septic shock, a life-threatening condition caused by severe infection. Researchers will measure blood flow in the tongue's tiny vessels and other key blood pressure indicators b…

This pilot study will test a drug called anisodamine in 20 adults with septic shock who are on a ventilator. The goal is to see if the drug can improve blood flow in small blood vessels and help with a phenomenon called 'vascular waterfall.' This is a very early study to gather i…

This study looks at whether the two-minute step test is a reliable way to measure aerobic endurance in people with hemophilia. Researchers will test 35 people aged 6–18 with hemophilia A or B, comparing the step test with other measures like walking tests and balance assessments.…