In short
There are currently 3 clinical trials investigating treatments for metabolic disorders, focusing on rare genetic conditions like PMM2-CDG and weight management in obesity with thyroid-related complications. These studies are testing medications including GLM101 and levothyroxine, combined with lifestyle interventions, to improve patient outcomes and quality of life.
Clinical trial locations
- Spain
Long-Term Safety Study of GLM101 for Patients with PMM2-CDG
This trial examines the long-term safety of GLM101 in patients with PMM2-CDG, a rare genetic disorder that affects how the body processes certain sugars. PMM2-CDG can cause developmental delays, coordination problems, and various other health challenges.
Who can join: The study is open to individuals between 12 and 65 years of age who have successfully completed a previous GLM101 treatment period. Participants must be willing to follow birth control requirements if they are of reproductive age and must agree to comply with all study procedures throughout the trial.
Who cannot join: Those who have not previously received GLM101 treatment or who do not meet the age requirements cannot participate. Women who are pregnant or unwilling to use acceptable contraception are excluded, as are individuals unable or unwilling to follow the study protocol.
What the study involves: The main focus is monitoring the long-term safety of GLM101, which is given through an intravenous injection or infusion. The medication contains alfa-d-mannopyranosyl phosphate dipotassium. Researchers will track side effects, perform regular blood and urine tests, conduct heart monitoring with ECG, and check vital signs. The study also measures coordination and balance changes using the International Co-operative Ataxia Rating Scale every 3 to 6 months, and evaluates blood levels of Mannose-1-Phosphate at the 6-month mark.
Investigational drug: GLM101 is the experimental medication being tested for its safety in long-term use for patients with PMM2-CDG. It is administered intravenously to help manage symptoms of this rare genetic condition.
Study on Levothyroxine and Low-Calorie Diet for Weight Loss in Obese Patients with Subclinical Hypothyroidism or High-Normal TSH Levels
This trial explores whether adding levothyroxine sodium to a low-calorie diet and exercise program can enhance weight loss in people with obesity who also have mild thyroid issues or borderline-high thyroid-stimulating hormone levels.
Who can join: Participants must be between 25 and 60 years old with a Body Mass Index between 30 and 39.9, indicating Grade I-II obesity. They should either have subclinical hypothyroidism with TSH levels between 5 and 10, or normal thyroid function with TSH levels in the higher range of normal. All participants must provide informed consent.
Who cannot join: Individuals with any metabolic disease other than the specified thyroid conditions, those outside the age range, people without obesity or the specified thyroid conditions, and members of vulnerable populations cannot participate.
What the study involves: The trial compares levothyroxine sodium with a placebo to see which is more effective for weight loss over three months. Participants follow a reduced-calorie diet and receive physical activity recommendations. Researchers track changes in weight, body composition including BMI, waist and hip measurements, and fat mass. The study also monitors cardiovascular risk factors like blood pressure and cholesterol, energy expenditure, physical activity levels, heart function, metabolic activity, and quality of life. A follow-up period after the initial three months assesses long-term outcomes.
Investigational drug: Levothyroxine is being tested as an additional treatment to support weight loss efforts. The medication is given orally in tablet form called Eutirox 88 micrograms, aiming to enhance the effects of diet and exercise in appropriate candidates.
Study on the Effects of GLM101 for Patients with PMM2-CDG
This study investigates how GLM101 affects symptoms of PMM2-CDG, particularly problems with movement, balance, and coordination known as ataxia. PMM2-CDG is a rare genetic disorder caused by mutations in the PMM2 gene, affecting protein glycosylation throughout the body.
Who can join: The trial accepts male and female participants in specific age groups: 2 to 11 years, 12 to 17 years, or 18 to 65 years at the time of screening. Participants must have a confirmed diagnosis of PMM2-CDG with documented genetic changes and enzyme activity. Reproductive-age participants must follow birth control requirements, and all must be willing and able to provide informed consent either directly or through a legally authorized representative.
Who cannot join: Individuals without a confirmed PMM2-CDG diagnosis, those outside the specified age ranges, members of vulnerable populations requiring special protection, and those who do not meet other specific health or medical criteria set by the study cannot participate.
What the study involves: The trial assesses how GLM101 affects ataxia symptoms after 12 and 24 weeks of treatment. The medication, containing alfa-d-mannopyranosyl phosphate dipotassium, is given through intravenous infusion over a 24-week period. Some participants may receive a placebo for comparison. Researchers monitor how the body processes the medication, its safety, and how well participants tolerate it. Regular assessments include various tests to track progress, measure symptom changes, and ensure participant safety.
Investigational drug: GLM101 is the experimental medication being studied for its potential to improve ataxia in individuals with PMM2-CDG. It is administered intravenously, and the trial aims to understand its effects on symptoms, safety profile, and tolerability.
Summary
All three clinical trials are being conducted in Spain, showing a concentration of research efforts in this region for metabolic disorders. Two of the three studies focus specifically on PMM2-CDG, a rare genetic condition, testing the experimental medication GLM101 at different stages—one examining long-term safety in patients who have previously received the treatment, and another evaluating its effects on symptoms over 24 weeks. The third trial takes a different approach by investigating whether levothyroxine can enhance weight loss in people with obesity and thyroid-related issues when combined with diet and exercise.
The trials demonstrate varied approaches to metabolic disorders, from addressing rare genetic conditions affecting sugar processing in the body to managing obesity complicated by mild thyroid dysfunction. The research spans different age groups, from young children to older adults, reflecting the diverse populations affected by these conditions. Expected completion dates extend to 2029, indicating a commitment to long-term safety and efficacy monitoring.
