Carbohydrate metabolism disease

Researchers test a one-time gene therapy infusion in children and adults with Sanfilippo syndrome (MPS IIIB), a rare inherited disease that damages the brain and body. The therapy delivers a working copy of the NAGLU gene to help the body make an enzyme that is missing or faulty.…

This phase 3 trial tests a drug called tralesinidase alfa in 14 children aged 1-5 with Sanfilippo syndrome type B, a rare genetic disease that causes severe brain damage. The drug is given directly into the brain fluid to replace a missing enzyme. The goal is to see if it can slo…

This early-stage trial is testing an experimental oral medication called S-606001 in adults with late-onset Pompe disease, a condition that causes progressive muscle weakness and breathing problems. The main goal is to see if the drug is safe and tolerable. Researchers will also …

This trial tests whether different intermittent fasting schedules—5:2, alternate-day, and time-restricted eating—can improve blood sugar, cholesterol, inflammation, and body composition in adults with prediabetes. Participants will be randomly assigned to one of three fasting pla…

This study tests the long-term safety and effectiveness of an experimental oral drug called S-606001 in adults with late-onset Pompe disease, a genetic condition that causes progressive muscle weakness. Participants who completed a prior S-606001 study can join, and they will rec…

This study looks at whether diazoxide, a drug that reduces insulin release, can help children with type 1 glycogenosis maintain steadier blood sugar levels. Type 1 glycogenosis is a rare inherited condition where the body cannot properly release glucose from stored glycogen, lead…

This study tests whether verapamil, a common blood pressure medication, can help protect insulin-producing cells in people with cystic fibrosis who have early blood sugar problems or diabetes. Participants take verapamil for several weeks, and researchers measure changes in insul…

This study tests whether a once-weekly injectable drug called mazdutide can help adults with prediabetes achieve normal blood sugar levels. About 150 participants will receive either a low dose, a higher dose, or a placebo for 24 weeks, along with diet and exercise advice. The ma…

This early-stage study tests whether high-dose Vitamin C is safe for people with two rare inherited blood disorders—Pyruvate Kinase Deficiency (PKD) and a form of G6PD deficiency. Both conditions cause red blood cells to break down too quickly, leading to anemia. The study will e…

This study tests whether adding Vitamin C, Omega-3 fatty acids, lignin, and lactulose to standard care can improve recovery in 108 adults after coronary artery bypass grafting (CABG). Participants will take the supplements daily for 10 weeks starting 15 days after surgery. The go…

This study tests whether a medication called chiglitazar sodium, combined with healthy lifestyle changes, can help people with prediabetes return to normal blood sugar levels. About 472 adults with prediabetes will take either the drug or a placebo for 52 weeks, along with lifest…

This early-stage trial tests a single dose of a gene therapy called DANAGALEX in 9 adults and children with aspartylglucosaminuria (AGU), a rare genetic disorder. The goal is to see if the treatment is safe and can reduce harmful substances in the body. Researchers will monitor s…

This study will test three types of intermittent fasting (5:2, alternate-day, and time-restricted eating) in 120 adults with prediabetes. Researchers will measure changes in blood sugar, cholesterol, body fat, and inflammation over 4-6 weeks. The goal is to see which fasting patt…

This study tests whether a liquid carob extract can improve blood sugar levels in 70 adults with prediabetes. Participants take the extract or a placebo twice daily for 90 days. Researchers will measure blood glucose and insulin responses to see if the extract helps control blood…

This study tests a supplement called GlycoDual in 90 adults with prediabetes (higher-than-normal blood sugar). For 90 days, half will take GlycoDual and half a placebo. Researchers will check if GlycoDual lowers fasting and after-meal blood sugar and improves how the body uses in…

This study tests whether replacing half the wheat in rotis with barnyard millet, barley, or bengal gram flour can lower blood sugar and help reverse prediabetes. 140 adults with prediabetes will eat these special rotis for three months while researchers track their glucose levels…

This trial tests whether measuring glucose in umbilical cord blood, already collected at birth, can screen newborns for congenital hyperinsulinism, a condition that causes dangerously low blood sugar and can lead to brain damage if missed. Researchers will check the test's accura…

This trial tests whether a finger-prick HbA1c test can detect type 2 diabetes better than the standard random blood glucose test in at-risk adults living in rural Dali, China. Researchers will compare detection rates and costs among 720 permanent rural residents with no known dia…

This study looks at whether the triglyceride-glucose (TyG) index, a simple measure from a blood test, can predict insulin resistance in people with pre-diabetes and chronic kidney disease. Researchers will enroll 60 adults with and without kidney problems to see how well the TyG …

Researchers are testing an AI chatbot that gives personalized, culturally tailored advice on nutrition, physical activity, and goal setting to adults at risk for type 2 diabetes. About 30 adults aged 18 to 55 will use the chatbot for 6 weeks. The study will check whether the chat…

This study tests whether two types of fish oil, EPA and DHA, can improve metabolic health in people at high risk for type 2 diabetes. Over 12 weeks, 84 participants will take daily capsules of EPA, DHA, or a placebo (corn oil) and undergo specialized tests to measure insulin func…

This study tests whether combining continuous glucose monitors (CGMs) with group lifestyle sessions can help people with prediabetes lower their blood sugar and prevent type 2 diabetes. Twenty adults with prediabetes will attend regular group meetings on diet, exercise, and stres…

This study tests a 12-session virtual program to help adults with prediabetes in the UAE prevent type 2 diabetes. The program covers diet, physical activity, sleep, and stress management, and is led by a health coach from the same cultural background. Thirty participants will att…

This study tests whether maple syrup, mixed with water and electrolytes, can improve cycling performance in trained male cyclists. Thirty-two participants will drink different amounts of maple syrup or a placebo during long rides. Researchers will measure how fast they finish a 2…

This study looks at how beta-cells (which make insulin) respond to growth hormone in people with prediabetes. Researchers will measure insulin release in 10 adults with impaired fasting glucose or impaired glucose tolerance. The goal is to better understand what raises the risk o…

This study tracks how Sanfilippo syndrome type C, a rare genetic disorder that affects the brain, progresses over time. Caregivers of children and young adults aged 1 to 25 will record videos of daily activities and answer questionnaires using a smartphone app every six months fo…

This study will take 3D scans of the inside of the mouth from 240 people with rare bone or cartilage diseases and from healthy volunteers. Researchers will use shape analysis and artificial intelligence to see if these scans can help tell different diseases apart. The goal is to …

This observational study will check vitamin C levels in 40 people with osteoporosis, comparing those who have had a spine fracture to those who haven't. The goal is to see if lower vitamin C is more common in the fracture group. No treatment is given; the study simply measures an…

This study uses a wearable glucose sensor and artificial intelligence to understand how healthy adults process sugar. Researchers will track blood sugar patterns for 14 days and link them to diet, activity, and health measures. The goal is to develop personalized nutrition tools …

This study will ask 75 people with diabetes and their healthcare providers about their experience using the Instara-1 continuous glucose monitor. Participants will wear the device and answer questionnaires about comfort, ease of use, and how it helps manage their diabetes. The go…

This study aims to validate a blood test for measuring oxalate levels, which is currently not available in Israel. Researchers will compare results from a local lab with an external lab using 50 blood samples. They will also explore how vitamin C levels relate to oxalate in patie…

This study looks at whether wearing a continuous glucose monitor (CGM) and receiving education can help teenagers with prediabetes improve their blood sugar levels and adopt healthier behaviors. Twenty-five teens aged 13-17 will take part in a 20-week program, first receiving sta…

This study will look at health records of 10,000 patients in Qatar who were hospitalized for heart problems and have diabetes or prediabetes. Researchers will use artificial intelligence to find patterns that predict future heart attacks, strokes, or death. The goal is to create …

The SIRENA study will follow 300 children and adults who have tested positive for islet autoantibodies, which are early markers of type 1 diabetes. Over up to 10 years, researchers will track how their blood sugar, immune system, and metabolism change. The goal is to better predi…

This study looks at whether eating a breakfast with two types of carbohydrates (glucose and fructose) can increase glycogen stored in the liver. Twelve healthy male athletes will be tested after different breakfasts using MRI scans and blood samples. The goal is to learn the best…

This study tests whether a drink made from broccoli sprouts can improve how well endurance athletes perform and recover after cycling. Thirty healthy adults who exercise regularly will drink either the broccoli sprout drink or a placebo at different times before a cycling test. R…

This study will look at 3000 young Asians in Singapore who do not have diabetes yet. Researchers will use blood tests, glucose monitors, food diaries, and body measurements to find better ways to detect diabetes early. The goal is to create new risk tools to identify young people…

This study will look at how getting only 3 hours of sleep (instead of 8) affects blood sugar, blood vessel stiffness, and thinking skills in healthy adults aged 20-45. Researchers will also test if regular exercise can help protect against these negative effects. The goal is to b…

This study will test 70 people with IBS-related diarrhea to see how many actually have lactose intolerance. Researchers will use a PCR test and a symptom questionnaire to find links with age and gender. The goal is to better understand if lactose intolerance is a common, overlook…

This study looks at whether not getting enough sleep affects blood sugar control differently in healthy men and women. Researchers will ask 32 adults to sleep only 4 hours a night for three nights and then measure their blood sugar and hormone responses after a meal. The goal is …

This study will look at 35 children with joint problems that are not caused by inflammation, such as stiffness or deformity. Researchers will use exams, lab tests, and imaging to find the true cause, which could be rare genetic conditions like mucopolysaccharidoses or osteogenesi…