Skeletal dysplasia

This study tested an experimental drug called SAR442501 in children with achondroplasia, a common form of dwarfism. The goal was to see if the drug was safe and could improve growth. However, the trial was stopped early after enrolling only 16 children, so we have very little inf…

This study tested whether the statin drug rosuvastatin, taken daily with supplements, could improve bone density and reduce heart disease risk in adults with long-term spinal cord injury. Only 8 people enrolled before the trial was stopped early. Participants received either rosu…

This Phase 3 trial tested an oral drug called venglustat in 75 adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that cause progressive nerve damage. The drug aimed to lower toxic fat buildup in the brain and slow disease worsening. However…

This study looked at whether adding an immunotherapy drug (pembrolizumab) to a targeted therapy (pemigatinib) works better than the targeted therapy alone or standard care for people with advanced bladder cancer that has a specific gene change (FGFR3). The study was stopped early…

This phase 2 study tested the drug pemigatinib in 111 people with advanced solid tumors that have specific FGFR gene mutations or translocations. The goal was to see if the drug could shrink tumors. The trial was terminated early, so the full results are not available.

This early-phase trial tested a new drug called INCB062079 in 25 people with advanced liver cancer and other solid tumors. The main goal was to check safety and find the best dose. The study was terminated early, so results are limited.

This early-stage trial tested a single dose of a drug called SAR439459 in 16 adults with osteogenesis imperfecta (brittle bone disease). The drug aims to block a protein that may weaken bones. The study focused on safety and how the drug moves through the body, but it was termina…

This trial investigates whether using 3D image fusion during iliac angioplasty reduces the amount of radiation patients receive compared to standard practice. The study involves 37 adults with iliac artery narrowing who need angioplasty. Researchers will measure radiation exposur…

This pilot study aims to understand why people with pseudohypoparathyroidism type 1A (PHP1A) have a higher risk of type 2 diabetes. Researchers will measure insulin sensitivity and beta-cell function in 14 participants with PHP1A or related conditions, comparing them to matched h…

This study aimed to help children aged 6-8 from low-income families learn how to avoid foods they are allergic to. The program used fun, hands-on activities to teach safety skills. The study was stopped early, so we don't have clear results on whether it worked.

This study looked at a type of spinal cord stimulation (SCS) that doesn't cause tingling, called paresthesia-free SCS, in 10 adults with chronic pain. Researchers wanted to see how well it works and how it affects pain perception. The study was terminated early, so the findings a…

This study looked at a specific hip implant shell (the Pinnacle Acetabular Shell) used in total hip replacement. Researchers wanted to see how much the implant moved (migrated) over two years using a special X-ray technique called RSA. They also tracked how patients felt and func…

This study aimed to understand how age influences serious health issues like infections, cancer, heart disease, and bone fractures in people with rheumatoid arthritis. Researchers compared patients over 65 with younger adults (18-50) who had the same disease. The study was stoppe…

This study collected bone, cartilage, skin, and fat tissue from 50 patients undergoing surgery at Children's National Medical Center. The goal was to grow cells from these tissues in the lab and analyze them to better understand bone and connective tissue diseases. The study was …

This study looked at bone density in HIV-positive men to see if antiretroviral drugs cause bone thinning. Researchers compared men starting HIV treatment with those not yet on therapy over two years. The goal was to understand why bone loss is more common in HIV patients.