Ongoing Clinical Trials for Hereditary Ataxia
Currently, there are 3 ongoing clinical trials investigating potential treatments for hereditary ataxia, specifically focusing on spinocerebellar ataxia types 1, 3, and related conditions. These studies are being conducted across several European countries and are testing different investigational medications to improve symptoms and quality of life for patients affected by these progressive neurological disorders.
Clinical trial locations
- Denmark
- France
- Germany
- Netherlands
- Portugal
- Spain
Study on the Safety of VO659 for Patients with Spinocerebellar Ataxia Types 1, 3, and Huntington’s Disease
This trial is testing a new medication called VO659, which is designed to target the genetic causes of spinocerebellar ataxia types 1 and 3. The medication is given as an injection into the space around the spinal cord, allowing it to reach the brain and nervous system directly.
Main inclusion criteria: Participants must be between 25 and 60 years old and have a genetically confirmed diagnosis of spinocerebellar ataxia type 1, type 3, or Huntington’s disease. For ataxia types 1 and 3, the condition should be mild to moderate. Participants should be in good general health otherwise, though stable chronic conditions like high blood pressure are acceptable. A minimum weight of 50 kg and a body mass index between 18 and 32 are required. Participants must also agree to use effective birth control methods during and after the study.
Main exclusion criteria: People without the specific diagnoses being studied cannot participate. Individuals outside the specified age range or those considered part of vulnerable populations requiring special protection are excluded from the study.
Study focus: The main purpose of this research is to evaluate how safe and well-tolerated multiple doses of VO659 are when given to participants. The study will monitor various health parameters including physical and neurological examinations, vital signs, and laboratory tests. It will also examine how the body processes the medication, how long it stays in the system, and how it is eliminated. Participants will receive increasing doses of the medication to observe how their bodies respond.
Investigational drug: VO659 is a type of medication known as an antisense oligonucleotide. It works at the molecular level by targeting specific genetic material in the body, potentially reducing the production of harmful proteins that cause these conditions. This medication is administered as an intrathecal injection, meaning it is delivered directly into the fluid surrounding the spinal cord.
Study of fampridine treatment for patients with spinocerebellar ataxia SCA27B caused by FGF14 gene mutation
This study is investigating whether fampridine can help improve symptoms in people with a specific type of hereditary ataxia called SCA27B, which is caused by a mutation in the FGF14 gene. This rare genetic condition affects movement and balance due to problems in the cerebellum, the part of the brain that controls coordination.
Main inclusion criteria: Participants must be at least 18 years old and have a confirmed genetic diagnosis of SCA27B with at least 250 GAA repeats in the FGF14 gene. They need to have a SARA score above 3, with at least a score of 1 for walking ability. SARA is a scale that measures movement and coordination difficulties. Participants must be able to take medication by mouth, attend all study visits, and have active social security coverage. Both men and women can participate.
Main exclusion criteria: People younger than 18 or older than 65 cannot participate. Pregnant or breastfeeding women, individuals with a history of seizures or epilepsy, and those with moderate to severe kidney disease are excluded. People currently taking medications that might interact with fampridine, those with significant heart problems, severe mental health conditions, or a history of drug or alcohol abuse within the past year are also not eligible. Participation in another clinical trial within the past 30 days is not allowed.
Study focus: The trial will test whether taking fampridine tablets twice daily for 12 weeks can improve symptoms in patients with SCA27B. Some participants will receive the actual medication while others will receive a placebo. The study lasts 16 weeks total, including 12 weeks of treatment followed by 4 weeks of observation after stopping the medication. Doctors will monitor movement abilities, balance, daily activities, and overall well-being throughout the study.
Investigational drug: Fampridine is a medication that helps improve walking and movement by enhancing nerve signal transmission through damaged nerve fibers. It is taken orally twice daily as prolonged-release tablets, with a maximum daily dose of 20 mg. While it is already approved for improving walking ability in multiple sclerosis patients, this trial is investigating whether it can also help people with SCA27B.
Study on Trehalose Dihydrate for Treating Spinocerebellar Ataxia in Adults
This clinical trial is studying SLS-005, a treatment containing trehalose dihydrate, for adults with spinocerebellar ataxia type 3. Trehalose is a type of sugar that researchers believe may help protect nerve cells and prevent abnormal protein buildup that contributes to this disease.
Main inclusion criteria: Participants must be between 18 and 75 years old, both men and women, with a genetically confirmed diagnosis of spinocerebellar ataxia type 3. They need a total m-SARA score of 4 or higher and a gait score of 1 or higher at the screening visit. The m-SARA scale measures the severity of ataxia symptoms, particularly walking ability. Body mass index must be between 18 and 35, and all other medications must be at stable doses for at least 30 days before screening. Female participants who can become pregnant must have a negative pregnancy test and be willing to use contraception.
Main exclusion criteria: People without a confirmed diagnosis of spinocerebellar ataxia type 3 cannot participate. Those unable to provide informed consent, pregnant or breastfeeding women, and individuals currently participating in another clinical trial are excluded. Patients with other serious health conditions that might interfere with the study or those with a history of allergic reactions to the study medication are not eligible. Use of certain medications that might interfere with the study within a specific timeframe before the study starts also disqualifies potential participants.
Study focus: The study aims to determine if SLS-005 can improve symptoms or slow down disease progression in adults with spinocerebellar ataxia type 3. The trial lasts approximately one year, during which participants receive regular infusions and attend follow-up visits. Researchers will assess various aspects including ability to perform daily activities and changes in symptoms. The goal is to see if the treatment can improve quality of life by reducing symptom severity. Safety monitoring includes regular laboratory tests and electrocardiograms.
Investigational drug: SLS-005 (Trehalose Injection) is delivered directly into the bloodstream through an intravenous infusion at a dose of 0.75 grams per kilogram of body weight. Trehalose is believed to work by helping stabilize proteins and prevent their abnormal clumping, which contributes to the disease. It is classified as a sugar molecule with potential neuroprotective properties. This medication is currently being studied in clinical trials to evaluate its safety and effectiveness.
Summary
The three ongoing clinical trials for hereditary ataxia reflect a diverse approach to treating these challenging neurological conditions. The trials are concentrated primarily in Western European countries, with Germany and France hosting multiple studies, and additional research taking place in Spain, Portugal, Denmark, and the Netherlands.
Each trial is testing a different type of medication with distinct mechanisms of action. VO659 represents a genetic approach using antisense oligonucleotide technology to target the disease at its molecular root. Fampridine offers a more established medication being repurposed for a rare form of ataxia, focusing on improving nerve signal transmission. SLS-005 takes yet another approach, using trehalose to potentially protect nerve cells from protein damage.
The trials vary in their stage of development and duration, from early safety studies to longer efficacy trials lasting up to one year. This range of approaches and medications being tested provides hope that researchers are exploring multiple pathways to find effective treatments for these progressive conditions that currently have limited treatment options.
