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Berotralstat

Berotralstat, also known as BCX7353 or Orladeyo®, is a drug being studied in clinical trials for the treatment of hereditary angioedema (HAE). These trials aim to evaluate its safety, effectiveness, and proper dosing in various patient populations, including children. The studies focus on using berotralstat as a preventive treatment to reduce the frequency and severity of HAE attacks.

Table of Contents

What is Berotralstat?

Berotralstat, also known by its brand name Orladeyo® or research code BCX7353, is a medication designed to prevent attacks of hereditary angioedema (HAE)[1]. HAE is a rare genetic condition that causes sudden episodes of swelling in various parts of the body, including the hands, feet, face, and airway. These swelling attacks can be painful, disfiguring, and even life-threatening if they affect the throat.

What Does Berotralstat Treat?

Berotralstat is specifically used for the prophylaxis (prevention) of HAE attacks in both adults and children[1][2]. This means it’s taken regularly to reduce the frequency and severity of HAE episodes, rather than treating attacks when they occur. It’s important to note that while berotralstat helps prevent attacks, patients may still need other medications to treat acute attacks if they do occur.

How is Berotralstat Administered?

Berotralstat is taken orally, which means it’s a medication you can take by mouth. For adults, the typical dose is one 150mg capsule taken once daily[2][3]. This makes it a convenient option for many patients, as it doesn’t require injections or infusions like some other HAE treatments.

For children, the dosing is weight-based, meaning the amount of medication will depend on how much the child weighs[1]. In some cases, berotralstat may be available as oral granules for children, which might be easier for younger patients to take.

Clinical Trials and Research

Berotralstat has been and continues to be studied in various clinical trials to ensure its safety and effectiveness. Here are some key points about the ongoing research:

  • Pediatric Studies: A Phase 3 study is evaluating the safety and pharmacokinetics (how the drug moves through the body) of berotralstat in children aged 2 to less than 12 years old[1]. This study aims to determine the appropriate weight-based dosing for children.
  • Long-term Safety: Another study is looking at the long-term safety and tolerability of berotralstat in patients who have previously participated in berotralstat studies[2]. This study will last up to 240 weeks (about 5 years) to gather data on the medication’s effects over an extended period.
  • Effectiveness Measures: Researchers are tracking various outcomes to assess how well berotralstat works, including the frequency and severity of HAE attacks[1].

Safety and Side Effects

As with any medication, it’s important to understand the potential side effects of berotralstat. The clinical trials are closely monitoring for adverse events (side effects) and serious adverse events. Some of the safety measures being tracked include:

  • The number and proportion of patients experiencing treatment-related side effects[2]
  • Any serious adverse events that occur
  • Grade 3 or 4 side effects (which are considered severe)
  • Any abnormal changes in blood chemistry related to the treatment
  • The number of patients who stop taking the medication due to side effects

It’s important to note that while these potential side effects are being monitored, it doesn’t mean every patient will experience them. Your doctor will discuss the potential risks and benefits of berotralstat with you based on your individual health situation.

Access to Berotralstat

Berotralstat is available through different means depending on your location and circumstances:

  • Commercial Availability: In some countries, berotralstat is already commercially available and can be prescribed by your doctor[2].
  • Clinical Trials: If berotralstat is not yet approved in your country, you may be able to access it through participation in a clinical trial[1][2].
  • Expanded Access Program: In the United States, there is an expanded access program that provides berotralstat to eligible patients with HAE who have an unmet medical need[3]. This program is available until the medication becomes commercially available. If you’re interested in this program, you should discuss it with your doctor, who can contact the program coordinators for more information.

Remember, the decision to use berotralstat should be made in consultation with your healthcare provider, who can assess your individual situation and determine if this medication is appropriate for you.

Aspect Details
Drug Name Berotralstat (BCX7353, Orladeyo®)
Condition Treated Hereditary Angioedema (HAE)
Administration Oral, once daily
Age Groups Studied Children (2 to <12 years), Adults
Primary Objectives Safety, Efficacy, Pharmacokinetics
Study Durations Up to 144 weeks (pediatric), Up to 240 weeks (adult)
Key Outcomes Measured Adverse events, HAE attack frequency and severity, Drug concentrations in blood
Special Programs Expanded Access Program for eligible patients

FAQ

  • What is hereditary angioedema (HAE)? Hereditary angioedema is a rare genetic condition that causes sudden swelling in various parts of the body, including the face, hands, feet, and airways. These swelling attacks can be painful and potentially life-threatening.
  • How is berotralstat administered in these clinical trials? Berotralstat is administered orally once daily. The dosage may vary depending on the specific trial and patient group, with some studies using weight-based dosing for children.
  • What age groups are being studied in these clinical trials? The clinical trials are studying berotralstat in various age groups. One study focuses on children aged 2 to less than 12 years old, while others include adult patients with HAE.
  • What are the main goals of these clinical trials? The main goals of these trials are to evaluate the safety, effectiveness, and proper dosing of berotralstat for preventing HAE attacks. They also aim to assess the drug's pharmacokinetics (how the body processes the drug) and long-term tolerability.
  • How long do these clinical trials last? The duration of the trials varies. Some studies last up to 144 weeks (about 3 years), while others may continue for up to 240 weeks (approximately 5 years) to assess long-term safety and effectiveness.

Ongoing Clinical Trials on Berotralstat

  • Long-Term Safety Study of Berotralstat for Patients with Hereditary Angioedema from Previous Trials

    Not yet recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia France Germany Italy Poland Slovakia +1

  • Study on the Safety and Effects of Berotralstat for Children Aged 2 to 12 with Hereditary Angioedema (HAE)

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria France Germany Italy Poland Spain

Glossary

  • Hereditary Angioedema (HAE): A rare genetic disorder characterized by recurrent episodes of severe swelling in various parts of the body, including the limbs, face, intestinal tract, and airways.
  • Berotralstat: An oral medication being studied for the prevention of hereditary angioedema attacks. It is also known by its drug code BCX7353 or brand name Orladeyo®.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including how it's absorbed, distributed, metabolized, and eliminated.
  • Prophylaxis: A preventive treatment used to stop something from happening. In the context of HAE, it refers to treatments that aim to prevent attacks from occurring.
  • Open-label study: A type of clinical study where both the researchers and participants know which treatment is being administered.
  • Adverse Event (AE): Any unfavorable and unintended sign, symptom, or disease that occurs during a clinical study, whether or not it is related to the treatment being studied.
  • Serious Adverse Event (SAE): An adverse event that results in death, is life-threatening, requires hospitalization, causes a significant incapacity, or is otherwise considered medically significant.
  • Treatment-Emergent Adverse Event (TEAE): An adverse event that appears or worsens during the course of a clinical study.
  • Expanded Access Program: A program that allows patients with serious or life-threatening conditions to access investigational drugs outside of clinical trials when no comparable or satisfactory alternative therapy options are available.

References

  1. https://clinicaltrials.gov/study/NCT05453968
  2. https://clinicaltrials.eu/trial/long-term-safety-study-of-berotralstat-for-patients-with-hereditary-angioedema-from-previous-trials/
  3. https://clinicaltrials.gov/study/NCT04428632