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Radiprodil

Radiprodil is an experimental drug being studied in clinical trials for various neurological conditions, including GRIN-related disorders, infantile spasms, tuberous sclerosis complex, and focal cortical dysplasia. These trials aim to evaluate the safety, effectiveness, and proper dosing of radiprodil in treating seizures and behavioral symptoms associated with these conditions. The drug works by modulating a specific receptor in the brain involved in neurological function.

Table of Contents

What is Radiprodil?

Radiprodil, also known as UCB3491, is a new investigational drug being studied for its potential to treat various neurological disorders[1]. It is currently undergoing clinical trials to evaluate its effectiveness and safety in treating conditions affecting the brain and nervous system.

How Radiprodil Works

Radiprodil is classified as a negative allosteric modulator of the NR2B subunit of the NMDA receptor[2]. To understand this in simpler terms:

  • NMDA receptor: This is a protein in brain cells that helps with communication between neurons (brain cells). It plays a crucial role in learning, memory, and brain development.
  • NR2B subunit: This is a specific part of the NMDA receptor.
  • Negative allosteric modulator: This means Radiprodil can reduce the activity of the NMDA receptor, specifically the part containing the NR2B subunit.

By modulating the activity of these receptors, Radiprodil may help in controlling excessive brain activity that can lead to seizures and other neurological symptoms[2].

Conditions Treated with Radiprodil

Radiprodil is being investigated for several neurological conditions, including:

  1. GRIN-related disorders: These are rare genetic conditions affecting brain development and function[2].
  2. Infantile Spasms (IS): A rare but serious type of seizure disorder that occurs in young children[3].
  3. Tuberous Sclerosis Complex (TSC): A genetic disorder causing non-cancerous tumors to grow in various parts of the body, including the brain[4].
  4. Focal Cortical Dysplasia (FCD) Type II: A condition where brain cells don’t develop properly, leading to seizures[4].

Current Clinical Trials

Several clinical trials are currently underway to evaluate Radiprodil:

  1. HONEYCOMB Study (NCT05818943): This trial is assessing Radiprodil in children with GRIN-related disorders[2].
  2. Infantile Spasms Study (NCT02829827): This study is evaluating Radiprodil in children with drug-resistant infantile spasms[3].
  3. ASTROSCAPE Study (NCT06392009): This trial is investigating Radiprodil in patients with Tuberous Sclerosis Complex (TSC) or Focal Cortical Dysplasia (FCD) Type II[4].

These trials aim to determine the safety, tolerability, and effectiveness of Radiprodil in treating these conditions.

How Radiprodil is Administered

Radiprodil is typically administered as a liquid suspension that can be taken orally or through a feeding tube[2][4]. The dosage is usually individualized for each patient, meaning the doctor will determine the right amount based on the patient’s specific needs and response to the medication.

In clinical trials, the drug is often given twice daily, with the dose gradually increased over time to find the most effective and safe amount for each patient[2][4].

Safety and Side Effects

As Radiprodil is still in the investigational stage, its full safety profile is not yet established. The ongoing clinical trials are carefully monitoring for any side effects or adverse reactions[2][3][4].

Some of the aspects being closely monitored include:

  • Treatment-Emergent Adverse Events (TEAEs)
  • Changes in vital signs (blood pressure, heart rate)
  • Changes in ECG readings (heart rhythm)
  • Changes in laboratory test results

It’s important to note that as an investigational drug, Radiprodil is only available through clinical trials at this time. Patients interested in this treatment should discuss with their healthcare providers about the possibility of participating in a clinical trial.

Aspect Details
Drug Name Radiprodil
Mechanism of Action Negative allosteric modulator of the NR2B subunit of the NMDA receptor
Conditions Studied GRIN-related disorders, Infantile spasms, Tuberous Sclerosis Complex, Focal Cortical Dysplasia Type II
Administration Oral suspension, twice daily
Primary Outcomes Safety, tolerability, pharmacokinetics, reduction in seizures, improvement in behavioral symptoms
Trial Phases Phase 1B/2A
Trial Duration Varies; initial phase up to 6 months, long-term treatment up to 1 year or more
Key Assessments Adverse events, plasma drug concentrations, seizure frequency, behavioral scales, quality of life measures

FAQ

  • What is radiprodil? Radiprodil is an experimental drug that acts as a negative allosteric modulator of the NR2B subunit of the NMDA receptor in the brain. It is being studied as a potential treatment for various neurological disorders, particularly those involving seizures and behavioral symptoms.
  • What conditions is radiprodil being studied for? Radiprodil is being investigated for several neurological conditions, including GRIN-related disorders, infantile spasms, tuberous sclerosis complex (TSC), and focal cortical dysplasia (FCD) type II. These conditions often involve difficult-to-treat seizures and behavioral issues.
  • How is radiprodil administered in clinical trials? In the clinical trials, radiprodil is typically given as a liquid suspension that can be taken orally or administered through a gastric or nasogastric tube. The dosage is usually given twice daily and is individually adjusted for each patient to find the most effective and safe dose.
  • What are the main goals of the radiprodil clinical trials? The primary objectives of these trials are to assess the safety and tolerability of radiprodil, determine its pharmacokinetics (how the drug moves through the body), and evaluate its potential effectiveness in reducing seizures and improving behavioral symptoms in patients with the targeted neurological disorders.
  • How long do the radiprodil clinical trials typically last? The duration of radiprodil trials varies, but they often include an initial phase lasting several months, followed by a longer-term treatment period that can last up to a year or more. Some trials also include an extended open-label phase to gather more data on long-term use and effects.

Ongoing Clinical Trials on Radiprodil

  • Study on the Safety and Effects of Radiprodil for Children with GRIN-related Disorder

    Recruiting

    1 1
    Investigated drugs:
    Belgium France Germany Italy The Netherlands Poland +2

  • Study on Radiprodil for Seizures and Behavioral Symptoms in Patients with Tuberous Sclerosis Complex or Focal Cortical Dysplasia Type II

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Italy The Netherlands Poland Spain

Glossary

  • NMDA receptor: A type of glutamate receptor in the brain that plays a crucial role in synaptic plasticity and memory function. Radiprodil targets a specific subunit of this receptor.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion. PK studies help determine proper dosing and administration of medications.
  • Open-label trial: A type of clinical trial where both the researchers and participants know which treatment is being administered, as opposed to a blinded study where this information is concealed.
  • Titration: The process of gradually adjusting the dose of a medication to find the optimal balance between effectiveness and side effects for each individual patient.
  • Adverse Event (AE): Any unfavorable and unintended sign, symptom, or disease temporarily associated with the use of a medical treatment or procedure, regardless of whether it is considered related to the medical treatment or procedure.
  • Hypsarrhythmia: An abnormal electroencephalogram (EEG) pattern characterized by high-voltage, irregular, and slow waves with spikes. It is often associated with infantile spasms.
  • Tuberous Sclerosis Complex (TSC): A genetic disorder characterized by the growth of benign tumors in various organs, often associated with seizures, developmental delays, and behavioral problems.
  • Focal Cortical Dysplasia (FCD): A congenital abnormality of brain development where neurons in an area of the brain failed to migrate and develop properly, often leading to seizures.
  • GRIN-related disorder: A group of neurological conditions caused by mutations in GRIN genes, which are involved in the function of NMDA receptors. These disorders can cause developmental delays, seizures, and behavioral issues.
  • Infantile spasms: A specific type of seizure disorder that typically begins in the first year of life and is characterized by sudden, brief contractions of the body, arms, and legs.

References

  1. https://clinicaltrials.gov/study/NCT02647697
  2. https://clinicaltrials.gov/study/NCT05818943
  3. https://clinicaltrials.gov/study/NCT02829827
  4. https://clinicaltrials.gov/study/NCT06392009