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Trehalose Dihydrate (Ph.eur.)

A groundbreaking clinical trial is underway to assess the safety and efficacy of Trehalose Dihydrate (Ph.eur.), known as SLS-005, for treating adults with Spinocerebellar Ataxia Type 3 (SCA3). This double-blind, randomized, placebo-controlled study aims to evaluate the potential of this investigational drug in managing the symptoms of this rare neurological disorder. The trial, named STRIDES, offers hope for patients suffering from SCA3 and could potentially lead to new treatment options for this challenging condition.

Table of Contents

What is Trehalose Dihydrate?

Trehalose Dihydrate, also known as SLS-005, is an investigational drug being studied for the treatment of Spinocerebellar Ataxia Type-3 (SCA3). It is a chemical compound that is being tested to see if it can help people with this rare neurological disorder[1]. Trehalose is a type of sugar that occurs naturally in some organisms and has shown potential in protecting brain cells.

Understanding Spinocerebellar Ataxia Type-3 (SCA3)

Spinocerebellar Ataxia Type-3 (SCA3), also known as Machado-Joseph disease, is a rare genetic disorder that affects the nervous system. It causes progressive problems with movement, balance, and coordination. People with SCA3 may experience difficulties with walking, speaking, and performing daily activities as the disease progresses[1].

Clinical Trial: STRIDES Study

A clinical trial called STRIDES (A Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia) is currently underway to test the safety and effectiveness of Trehalose Dihydrate in treating adults with SCA3[1]. This study is:

  • Double-blind: Neither the participants nor the researchers know who is receiving the actual drug or a placebo.
  • Randomized: Participants are randomly assigned to different treatment groups.
  • Placebo-controlled: Some participants receive a placebo (a substance with no active ingredients) for comparison.

The main goal of this study is to determine if Trehalose Dihydrate can effectively treat SCA3 in adults and to assess its safety and tolerability[1].

How is Trehalose Dihydrate Administered?

In the STRIDES study, Trehalose Dihydrate (SLS-005) is given as an intravenous infusion. This means the medication is delivered directly into the bloodstream through a vein. The dosage being tested is 0.75 g/kg (grams per kilogram of body weight)[1].

Who is Eligible for the Clinical Trial?

The study has specific criteria for who can participate. Some key eligibility requirements include:

  • Adults aged 18 to 75 years
  • Confirmed genetic diagnosis of SCA3
  • Certain scores on movement and gait assessments
  • Body Mass Index (BMI) between 18 and 35 kg/m²

There are also several conditions that would exclude someone from participating, such as other types of ataxia, certain medical conditions, or recent participation in other clinical trials[1].

How is the Effectiveness Measured?

The researchers use several methods to measure how well Trehalose Dihydrate works:

  • m-SARA score: This is a scale that measures the severity of ataxia symptoms.
  • Clinical Global Impression of Severity (CGI-S): This is the doctor’s assessment of the severity of the patient’s condition.
  • Patient Global Impression of Severity (PGI-S): This is the patient’s own assessment of their condition.
  • Friedreich’s Ataxia Rating Scale – Activities of Daily Living (FARS-ADL): This measures how well patients can perform everyday activities.

These assessments are done at various points throughout the 52-week study period to track changes over time[1].

Safety Considerations

As with any clinical trial, safety is a top priority. The researchers will closely monitor participants for any side effects or adverse reactions. Some safety measures include:

  • Regular check-ups and laboratory tests
  • Monitoring of liver and kidney function
  • Cardiovascular assessments
  • Screening for mental health concerns

It’s important to note that Trehalose Dihydrate is still an investigational drug, which means it has not yet been approved for general use in treating SCA3. The STRIDES study aims to provide more information about its safety and effectiveness[1].

Aspect Details
Study Name STRIDES
Drug Name SLS-005 (Trehalose Dihydrate)
Condition Spinocerebellar Ataxia Type 3 (SCA3)
Study Design Double-blind, randomized, placebo-controlled
Treatment Duration 52 weeks
Primary Endpoint Change in m-SARA total score at Week 52
Key Secondary Endpoints Changes in CGI-S, PGI-S, FARS-ADL scores
Administration Intravenous infusion
Dosage 0.75 g/kg (higher dose arm)
Age Range 18 to 75 years

FAQ

  • What is Spinocerebellar Ataxia Type 3 (SCA3)? Spinocerebellar Ataxia Type 3 (SCA3) is a rare genetic disorder that affects the nervous system. It causes progressive problems with movement, balance, and coordination due to damage in the cerebellum and other parts of the brain.
  • What is the main goal of the STRIDES clinical trial? The main goal of the STRIDES clinical trial is to determine the effectiveness of SLS-005 (Trehalose Dihydrate) at a dose of 0.75 g/kg for treating adults with Spinocerebellar Ataxia Type 3 (SCA3).
  • How is the drug SLS-005 administered in the trial? SLS-005 is administered as an intravenous infusion. The drug is a solution for infusion containing 90.5 mg/mL of Trehalose Dihydrate.
  • What are some of the key eligibility criteria for participating in the trial? Key eligibility criteria include being 18 to 75 years old, having a confirmed genetic diagnosis of SCA3, a minimum score on certain ataxia rating scales, and stable doses of other medications for at least 30 days before screening.
  • How long does the treatment period last in the STRIDES trial? The treatment period in the STRIDES trial lasts for 52 weeks (about one year). This is followed by a two-week safety follow-up period.

Ongoing Clinical Trials on Trehalose Dihydrate (Ph.eur.)

  • Study on Trehalose Dihydrate for Treating Spinocerebellar Ataxia in Adults

    Not recruiting

    4 1
    Investigated drugs:
    Germany Portugal Spain

Glossary

  • Spinocerebellar Ataxia (SCA): A group of inherited disorders characterized by progressive problems with movement, balance, and coordination due to damage in the cerebellum and other parts of the brain.
  • Trehalose Dihydrate: The active substance in SLS-005, being investigated for its potential to treat Spinocerebellar Ataxia Type 3.
  • m-SARA: Modified Scale for the Assessment and Rating of Ataxia, a tool used to measure the severity of ataxia symptoms.
  • Intravenous Infusion: A method of delivering medications directly into the bloodstream through a vein.
  • Double-blind: A study design where neither the participants nor the researchers know who is receiving the actual treatment and who is receiving a placebo.
  • Placebo: An inactive substance that looks like the drug being tested but has no medical effect.
  • Efficacy: The ability of a treatment to produce the desired beneficial effect.
  • Biomarker: A measurable indicator of a biological state or condition, often used to track the progress of a disease or the effects of treatment.
  • CGI-S: Clinical Global Impression of Severity, a scale used by healthcare providers to assess the severity of a patient's illness.
  • PGI-S: Patient Global Impression of Severity, a scale used by patients to rate the severity of their own condition.
  • FARS-ADL: Friedreich's Ataxia Rating Scale – Activities of Daily Living, a measure of how ataxia affects a person's ability to perform everyday tasks.

References

  1. http://clinicaltrials.eu/trial/study-on-trehalose-dihydrate-for-treating-spinocerebellar-ataxia-in-adults/