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(S)-2-Hydroxy-6-((4-(2-(2-Hydroxyethyl)Nicotinoyl)Morpholin-3-Yl)Methoxy)Benzaldehyde

This article discusses the ongoing clinical trials of GBT021601, also known as (S)-2-Hydroxy-6-((4-(2-(2-Hydroxyethyl)Nicotinoyl)Morpholin-3-Yl)Methoxy)Benzaldehyde, a potential new treatment for Sickle Cell Disease (SCD). The trials aim to evaluate the drug’s effectiveness, safety, and impact on patients’ quality of life across different age groups, from infants to adults.

Table of Contents

Introduction to GBT021601

GBT021601, also known as (S)-2-hydroxy-6-((4-(2-(2-hydroxyethyl)nicotinoyl)morpholin-3-yl)methoxy)benzaldehyde, is a new medication being developed to treat Sickle Cell Disease (SCD). This drug is currently undergoing clinical trials to evaluate its effectiveness and safety in both adults and children with SCD[1].

What is Sickle Cell Disease?

Sickle Cell Disease is a group of inherited red blood cell disorders. In SCD, the red blood cells become crescent or “sickle” shaped, which can cause them to break down early, block blood flow, and lead to complications such as pain, organ damage, and anemia (low red blood cell count)[1].

How GBT021601 Works

GBT021601 is designed to help improve the function of hemoglobin, the protein in red blood cells that carries oxygen. By doing so, it aims to reduce the sickling of red blood cells and improve their ability to carry oxygen throughout the body[1].

Current Clinical Trial

The ongoing clinical trial for GBT021601 is a Phase 2/3 study that includes both adults and children with Sickle Cell Disease. The study is divided into three parts[1]:

  • Part A: Assesses the effects of GBT021601 in adult participants, focusing on changes in hemoglobin levels.
  • Part B: Compares GBT021601 to a placebo in adults and adolescents, looking at hemoglobin response and the rate of vaso-occlusive crisis (VOC) events. VOCs are painful episodes caused by blocked blood flow.
  • Part C: Evaluates how the drug behaves in the bodies of pediatric participants (pharmacokinetics).

Who Can Participate in the Trial?

The trial has specific criteria for who can participate. Some key points include[1]:

  • Participants must have confirmed Sickle Cell Disease (genotype HbSS or HbSB).
  • For Part B, participants must be 12 years or older and have experienced between 2 and 10 VOCs in the past year.
  • For Part C, different age groups are being studied, ranging from 6 months to 18 years old.
  • Participants must have a hemoglobin level between 5.5 and 10.5 g/dL.

There are also several factors that would exclude someone from participating, such as pregnancy, recent blood transfusions, or certain medical conditions[1].

Potential Benefits of GBT021601

The researchers are hoping to see several potential benefits from GBT021601, including[1]:

  • Increased hemoglobin levels
  • Reduced rate of vaso-occlusive crisis events
  • Improved measures of hemolysis (breakdown of red blood cells)
  • Better quality of life for patients
  • Improved neurocognitive function

Safety Considerations

As with any new medication, safety is a crucial aspect of the clinical trial. The researchers will be closely monitoring[1]:

  • Any adverse events (side effects) that occur during the trial
  • Changes in laboratory test results
  • Changes in ECG (heart rhythm) readings
  • Changes in vital signs

It’s important to note that while GBT021601 shows promise, it is still in the testing phase. More research is needed to fully understand its effectiveness and safety profile.

Aspect Details
Drug Name GBT021601 ((S)-2-Hydroxy-6-((4-(2-(2-Hydroxyethyl)Nicotinoyl)Morpholin-3-Yl)Methoxy)Benzaldehyde)
Condition Sickle Cell Disease (SCD)
Trial Phase Phase 2/3
Age Groups 6 months to adults
Primary Objectives Assess effects on hemoglobin levels, VOC events, and pharmacokinetics
Secondary Objectives Evaluate safety, quality of life, and measures of hemolysis
Administration Oral tablet
Maximum Daily Dose 300 mg
Treatment Duration Up to 48 weeks

FAQ

  • What is GBT021601? GBT021601 is a new drug being tested for the treatment of Sickle Cell Disease. It's also known by its chemical name (S)-2-Hydroxy-6-((4-(2-(2-Hydroxyethyl)Nicotinoyl)Morpholin-3-Yl)Methoxy)Benzaldehyde.
  • What are the main goals of the clinical trials? The main goals are to assess the effects of GBT021601 on hemoglobin levels, rate of vaso-occlusive crisis (VOC) events, and to study its safety and effectiveness in both adult and pediatric patients with Sickle Cell Disease.
  • Who can participate in these clinical trials? The trials include participants with Sickle Cell Disease, ranging from 6 months to adults. Different parts of the study have specific age requirements and other eligibility criteria, such as hemoglobin levels and history of VOC events.
  • What are the primary outcomes being measured? The primary outcomes include changes in hemoglobin levels, hemoglobin response (increase from baseline), rate of VOC events, and pharmacokinetic parameters of the drug in different age groups.
  • How long do the trials last? The duration varies for different parts of the study, but some participants may receive treatment for up to 48 weeks, with follow-up assessments to evaluate long-term effects and safety.

Ongoing Clinical Trials on (S)-2-Hydroxy-6-((4-(2-(2-Hydroxyethyl)Nicotinoyl)Morpholin-3-Yl)Methoxy)Benzaldehyde

  • Study of GBT021601 for Adults and Children with Sickle Cell Disease

    Not yet recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    France Germany

Glossary

  • Sickle Cell Disease (SCD): A group of inherited red blood cell disorders where red blood cells become hard and sticky, taking on a C-shape (like a sickle). These cells die early, causing a constant shortage of red blood cells.
  • Hemoglobin (Hb): A protein in red blood cells that carries oxygen throughout the body. In SCD, abnormal hemoglobin causes the sickle shape of red blood cells.
  • Vaso-occlusive Crisis (VOC): A common and painful complication of SCD where sickled red blood cells block blood vessels, restricting blood flow to body tissues.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Hemolysis: The destruction of red blood cells, which occurs at a faster rate in people with SCD.
  • Reticulocyte: Immature red blood cells; a high count can indicate increased production of red blood cells to compensate for anemia.
  • Bilirubin: A yellow compound produced when hemoglobin breaks down. High levels can indicate increased destruction of red blood cells.
  • Lactate Dehydrogenase (LDH): An enzyme released when cells are damaged or destroyed. Elevated levels in SCD can indicate increased hemolysis.

References

  1. http://clinicaltrials.eu/trial/study-of-gbt021601-for-adults-and-children-with-sickle-cell-disease/