Peripheral nervous system disorder

This early-stage study tested a new drug called PF-07284892, alone or with other medicines, in people with advanced solid tumors that had specific genetic changes. The goal was to find the safest dose and check for side effects. The study was stopped early, so results are limited…

This phase 2 study tested an experimental oral drug called BGB-45035 in 49 adults with moderate to severe rheumatoid arthritis who had not responded well to standard treatments. Participants received either the drug or a placebo to see if it could reduce joint pain and swelling. …

This study tested an experimental oral drug called TNG908 in people with advanced solid tumors that have lost a gene called MTAP. The goal was to see if the drug is safe and can shrink tumors. The study was planned for up to 192 participants but was terminated early, so results a…

This study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …

This study tested whether high-dose fish oil supplements could improve nerve function in people with prediabetes and early diabetic neuropathy. Researchers planned to enroll 40 adults aged 55–80, but the trial was terminated early with only 31 participants. Participants took eith…

This early-stage trial tested a new drug called DFF332, which targets a protein (HIF2α) that helps some cancers grow. The study included 40 adults with advanced clear cell renal cell carcinoma or other cancers with specific gene changes. DFF332 was tested alone and in combination…

This phase 2 trial tested the drug guadecitabine (SGI-110) in people aged 12 and older with rare cancers like wild-type GIST, pheochromocytoma, paraganglioma, and HLRCC-related kidney cancer that had not responded to other treatments. Participants received daily injections for 5 …

This early trial tested an experimental drug called HN2301, a type of CAR T cell therapy made inside the body, for people with refractory myasthenia gravis — a condition causing severe muscle weakness that doesn't respond well to standard treatments. The study planned to enroll 9…

This early-phase trial tested a new therapeutic vaccine (EO2401) combined with the immunotherapy drug nivolumab in 70 people with advanced adrenocortical carcinoma or malignant pheochromocytoma/paraganglioma. The goal was to see if the combination is safe and can shrink tumors or…

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 early-phase trial tested whether nicotinamide riboside (a form of vitamin B3 sold as Niagen) could help repair nerve damage in people with type 2 diabetes or prediabetes. Only 10 participants were enrolled before the study was terminated. The main goal was to measure changes…

This early-phase trial tested the drug AR-42 in 7 adults with vestibular schwannoma or meningioma brain tumors who were already scheduled for surgery. Participants took AR-42 pills for about 3 weeks before their operation. The main goal was to see if the drug could lower a protei…

This early-stage trial tested a gene therapy called SBT101 for adrenomyeloneuropathy (AMN), a rare nerve disease that causes walking difficulties. Eight adults received either the therapy or a sham procedure. The study was terminated early, so we have limited data on safety and e…

This early-phase trial tested a new immunotherapy approach for children with DIPG, a rare and aggressive brain stem tumor. After standard radiation and chemotherapy, patients received special vaccines and immune cells designed to attack the tumor. The study was small (11 particip…

This phase 3 trial tested the drug miglustat (Zavesca) in 30 infants with Sandhoff or Tay-Sachs diseases, rare genetic disorders that destroy nerve cells. The goal was to see if the drug could reduce hospitalizations, seizures, and feeding problems while improving motor function.…

This study tested tolebrutinib, an oral drug, in adults with moderate-to-severe generalized myasthenia gravis. The trial was a Phase 3, double-blind, placebo-controlled study that planned to enroll many participants but was terminated early with only 6 people enrolled. Because it…

This early-stage trial tested a gene therapy called AXO-AAV-GM2 in children with Tay-Sachs or Sandhoff disease, rare and fatal genetic brain disorders. The therapy delivers healthy genes directly into the brain and spinal fluid to try to restore a missing enzyme. The study was te…

This study compared a new, minimally invasive ultrasound-guided procedure to standard surgery for carpal tunnel syndrome. The goal was to see if the new technique could provide similar relief with a smaller incision and faster recovery. Only 7 people were enrolled before the stud…

This trial tested whether a TENS device, which sends mild electrical pulses to the skin, can help relieve nerve pain caused by chemotherapy in early-stage breast cancer patients. The study enrolled 17 people who were having taxane chemotherapy and had at least mild nerve pain in …

This study tested a drug called AVP-786 to see if it could safely reduce disinhibition—impulsive or inappropriate behavior—in people with neurodegenerative disorders like Alzheimer's or frontotemporal dementia. The trial planned to include many participants but was terminated ear…

This study looked at whether adding platelet-rich plasma (PRP) to standard carpal tunnel release surgery helps people with severe carpal tunnel syndrome. The trial planned to enroll 15 adults with severe nerve damage. It compared surgery with PRP to surgery alone. The study was t…

This study tested a device called Exablate, which uses focused sound waves to treat areas of the brain linked to pain. The goal was to see if it could reduce pain and improve quality of life in people with chronic nerve pain. Only 2 people enrolled before the study was stopped, s…

This small study tested whether an injection made from a patient's own blood (platelet lysate) could reduce leg pain from sciatica better than a saltwater injection. Only 2 people took part before the study was stopped early, so we cannot draw any firm conclusions. The approach r…

This study tested whether a 12-week program of walking and resistance exercises or nutrition education could reduce nerve pain caused by platinum chemotherapy. The trial enrolled 36 adults with cancer who were receiving oxaliplatin or cisplatin. The study was terminated early, so…

This study tested an experimental drug called RTA 901 to see if it could safely reduce nerve pain in people with diabetes. The trial planned to enroll 209 adults with diabetic peripheral neuropathic pain, comparing two doses of the drug against a placebo over 12 weeks. However, t…

This study tested an experimental drug called GSK3858279 in 147 adults with chronic nerve pain from diabetes. The goal was to see if it could reduce pain better than a placebo. However, the trial was terminated early, so we have less information than planned.

This study tested a device called BurstDR spinal cord stimulation to help people with painful diabetic neuropathy that didn't get better with standard medicines. The goal was to see if it could cut leg pain by at least half. Only 6 people joined before the study was stopped early…

This study tested a device called SinuSonic that uses sound vibrations and pressure to relieve sinus headache pain. The trial included 27 adults with facial pain lasting more than 3 months. Participants used either the real device or a sham device that looked the same but didn't …

This small, early-stage trial tested whether adding ondansetron (an anti-nausea drug) to pyridostigmine (a standard myasthenia gravis treatment) could reduce stomach side effects like nausea, vomiting, or diarrhea. Only 2 people with anti-AchR positive myasthenia gravis were enro…

This early-stage study aimed to compare how a tablet form of tafamidis is absorbed in the body compared to the existing capsule form. It involved 24 healthy adults who took a single dose of each form under fed conditions. The study was terminated early, so results may be limited.

This study looked back at 20 young people who came to the hospital with nerve problems after using nitrous oxide (laughing gas) for fun. Researchers measured muscle strength, balance, and disability to understand how the gas harms the nervous system. The goal was to learn more ab…

This study tested whether special insoles with pressure sensors (FEETME®) can accurately measure walking speed in people with CIDP, a nerve condition that causes walking difficulties. Researchers compared the insoles to a standard walkway system in 18 adults aged 18-75 with CIDP.…

This study looked at why people with type 1 diabetes often fail to sense dangerously low blood sugar (hypoglycemia). Researchers tested how opioid drugs like morphine and naloxone affect the body's ability to respond to low sugar. The goal was to better understand the problem, no…

This study tested a new method called cortical vestibular evoked potentials (C-VEPs) to measure how the brain processes balance signals from the inner ear. Researchers compared people with normal balance to those with vestibular neuronitis (a cause of vertigo) to see if the test …

This study looked at whether follow-up calls from a headache nurse could help patients with migraines, tension headaches, cluster headaches, or hemicrania continua stay on their preventive medication. 147 participants were randomly assigned to receive nurse calls at 2 and 6 weeks…

This study followed 65 adult men with a rare inherited nerve disease called AMN (a form of spastic paraplegia) to understand how their symptoms change over time. Researchers collected data on walking ability and quality of life. The goal was to fill gaps in knowledge about the di…

This study looked at muscle samples from 7 children with brachial plexus birth palsy who had shoulder stiffness. Researchers wanted to understand why the shoulder gets stiff again after surgery, by checking for muscle wasting and scarring. The study was terminated early, so findi…

This study tested a new 3D scanning device that aims to help surgeons match a patient's spine bones to their MRI or CT scans during surgery. The goal was to see if the scanner could accurately and consistently map the spine to improve surgical precision. Only one person took part…

This study aims to find out how common transthyretin amyloidosis cardiomyopathy (ATTR-CM) is in Russian patients with a certain type of heart failure. Researchers will review medical records and then invite some patients for extra heart tests to confirm or rule out ATTR-CM. The g…

This study follows up on children with Krabbe disease who received a one-time gene therapy infusion (FBX-101) in earlier trials. Researchers will monitor safety and measure motor skills over time. Only 2 participants are enrolled, so results are very limited.