Ongoing Clinical Trials for Hereditary Motor and Sensory Neuropathy
There are currently 2 clinical trials exploring new treatments for hereditary motor and sensory neuropathy (also known as Charcot-Marie-Tooth Disease). These trials are investigating different medications aimed at improving symptoms and quality of life for patients. Studies are taking place across several European countries including Belgium, Denmark, France, and Spain.
Clinical trial locations
- Belgium
- Denmark
- France
- Spain
Study on Colchicine for Patients with Angina Symptoms and Coronary Microvascular Disease
This trial is investigating the use of colchicine, a medication commonly used to treat inflammation, in patients with Coronary Microvascular Disease (CMD). While this condition affects the heart rather than peripheral nerves, it shares certain characteristics with nerve-related conditions and is being studied in patients who may have overlapping symptoms.
Main inclusion criteria: Participants must have CMD with specific blood flow measurements showing reduced heart function. They must have a myocardial blood flow reserve below 2.5 or hyperemic myocardial blood flow less than 2.3 ml/g/min. Additionally, they should not have blockages in the main heart arteries, as confirmed by specialized heart scans. The age range for this study is 18 to 65 years, and both males and females can participate.
Main exclusion criteria: Patients with other serious heart conditions unrelated to CMD cannot join. Those who have experienced a heart attack or stroke within the last 6 months are excluded. People with severe liver or kidney disease, uncontrolled high blood pressure, or a history of severe allergic reactions to medications are also not eligible. Pregnant or breastfeeding women and those currently participating in another clinical trial cannot participate.
Focus and goal: The study aims to determine whether colchicine can improve blood flow in the small vessels of the heart and reduce chest pain symptoms in patients with CMD. Participants will undergo heart scans at the beginning and after six months of treatment to measure any improvements in blood flow. The trial will also assess changes in angina symptoms through questionnaires.
Investigational drug: The medication being tested is colchicine, administered as 500 microgram tablets taken orally. Participants will receive either the active medication or a placebo over a six-month period.
Study on the Safety and Effectiveness of NMD670 for Adults with Charcot-Marie-Tooth Disease Types 1 and 2
This trial directly focuses on Charcot-Marie-Tooth Disease (also known as hereditary motor and sensory neuropathy), specifically types 1 and 2. These are inherited disorders that affect the peripheral nerves responsible for movement and sensation in the limbs.
Main inclusion criteria: Participants must be between 18 and 70 years old and have a diagnosis of CMT type 1 or 2 confirmed through genetic testing. They need to demonstrate certain physical capabilities, including being able to walk a specific distance in a 6-minute walk test without assistive devices like ankle-foot orthoses, though orthopedic inserts are allowed. Their ankle strength must be tested at 4 or less manually. Participants should have a body mass index between 18 and 35 kg/m² and weigh at least 40 kg. Those already receiving physical or occupational therapy for more than 30 days before screening should continue this throughout the study.
Main exclusion criteria: Patients who do not have CMT type 1 or type 2 cannot participate. Those outside the specified age range or not meeting the physical capability requirements are excluded. Vulnerable populations may also be excluded from participation.
Focus and goal: The primary aim is to evaluate how effective and safe NMD670 is for improving mobility and overall function in people with CMT. The study will measure changes in walking distance over a set period using the 6-minute walk test and assess the time needed to complete certain walking tasks. These measurements will help determine whether NMD670 can improve the condition of patients as assessed by healthcare professionals.
Investigational drug: NMD670 is an oral tablet being studied in Phase 2a clinical trials. The medication targets specific pathways involved in nerve function, aiming to enhance nerve conduction and muscle strength. Participants will take the tablet daily for 21 days, with regular assessments throughout to monitor progress and any changes in their condition.
Summary
Two clinical trials are currently underway for patients with hereditary motor and sensory neuropathy and related conditions. The most directly relevant study focuses on NMD670 for CMT types 1 and 2, and is being conducted across four European countries: Belgium, Denmark, France, and Spain. This multi-country approach may help gather diverse data from different patient populations.
The trials represent different approaches to treatment, with one focusing on a novel medication specifically developed for CMT (NMD670) and another exploring the repurposing of an existing anti-inflammatory medication (colchicine) for a related condition affecting small blood vessels. Both studies emphasize objective measurements of physical function, such as walking ability, which are important practical outcomes for patients dealing with these conditions.
Patients interested in participating should note that these trials have specific eligibility requirements related to age, disease confirmation through genetic testing, and physical capabilities. Those already engaged in therapy or taking medications should discuss with their healthcare providers whether continuing their current treatment regimen is compatible with trial participation.