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Poloxamer 407

This article explores the use of Poloxamer 407 (also known as Poloxamer 188) in clinical trials for the treatment of sickle cell disease. Poloxamer 407 is being investigated as a potential therapy to reduce the duration and severity of painful vaso-occlusive crises in patients with sickle cell anemia. The trials aim to assess the efficacy, safety, and impact on pain management and hospitalization duration for patients experiencing these crises.

Table of Contents

What is Poloxamer 407?

Poloxamer 407, also known as Poloxamer 188, is a drug that has shown promise in treating sickle cell disease, a genetic blood disorder that affects millions of people worldwide[1]. This medication is being studied to help patients who experience painful episodes called vaso-occlusive crises, which are a hallmark of sickle cell disease.

How Does It Work?

While the exact mechanism is not fully explained in the provided information, Poloxamer 407 is believed to help reduce the duration and severity of painful vaso-occlusive crises in sickle cell disease patients[1]. These crises occur when abnormally shaped red blood cells block small blood vessels, causing pain and potential organ damage.

Treating Sickle Cell Disease

Sickle cell disease is a condition where red blood cells become crescent or “sickle” shaped, leading to various complications. Poloxamer 407 is being investigated as a treatment specifically for the painful episodes associated with this disease[1]. The goal is to provide relief to patients during these crises and potentially reduce the need for other pain medications.

Clinical Trial Details

A Phase III clinical trial (NCT00004408) was conducted to assess the effectiveness of Poloxamer 407 in treating sickle cell disease[1]. Here are some key details about the study:

  • Study Type: The trial was randomized, double-blind, and placebo-controlled. This means that participants were randomly assigned to receive either Poloxamer 407 or a placebo (a substance with no active ingredients), and neither the patients nor the researchers knew who received which treatment during the study.
  • Administration: Poloxamer 407 was given to patients through continuous infusion (a steady flow into the bloodstream) for 48 hours when they presented with a vaso-occlusive crisis.
  • Timing: Treatment began within 12 hours of a patient experiencing a crisis.
  • Assessment: Pain levels were evaluated before, during, and after the treatment to determine the drug’s effectiveness.
  • Follow-up: Patients were monitored on days 7-14 and 28-35 after treatment to assess any long-term effects or benefits.

Potential Benefits

The clinical trial aimed to determine if Poloxamer 407 could provide the following benefits for sickle cell disease patients[1]:

  • Reduced Crisis Duration: The primary goal was to shorten the length of painful vaso-occlusive crises.
  • Pain Relief: Researchers assessed whether the drug could decrease both the duration and intensity of pain during crises.
  • Less Need for Pain Medication: The study looked at whether Poloxamer 407 could reduce the total amount of pain medication (analgesics) needed during a crisis.
  • Shorter Hospital Stays: Another potential benefit being investigated was whether the treatment could lead to shorter periods of hospitalization for patients experiencing crises.

It’s important to note that while these potential benefits were being studied, the results of the trial are not provided in the given information. Patients interested in this treatment should consult with their healthcare providers for the most up-to-date information on Poloxamer 407 and its effectiveness in treating sickle cell disease.

Aspect Details
Drug Name Poloxamer 407 (also known as Poloxamer 188)
Condition Studied Sickle Cell Anemia
Trial Phase Phase III
Study Design Randomized, double-blind, placebo-controlled, multicenter
Primary Objective Assess efficacy in reducing duration of painful vaso-occlusive crisis
Secondary Objectives Evaluate effects on pain intensity, analgesic use, and hospitalization length
Treatment Duration 48-hour continuous infusion
Follow-up Period Days 7-14 and 28-35 after treatment
Patient Stratification Based on hydroxyurea use
Treatment Initiation Within 12 hours of crisis presentation

FAQ

  • What is Poloxamer 407 and how is it being used in sickle cell disease trials? Poloxamer 407, also known as Poloxamer 188, is a drug being studied for its potential to reduce the duration of painful vaso-occlusive crises in patients with sickle cell disease. It is administered through continuous infusion for 48 hours when patients present with a crisis.
  • What are the main objectives of the clinical trial using Poloxamer 407? The main objectives are to assess the efficacy of Poloxamer 407 in reducing the duration of painful vaso-occlusive crises, evaluate its effect on pain intensity, measure total analgesic use, and determine its impact on the length of hospitalization for sickle cell disease patients.
  • How is the clinical trial designed? The trial is designed as a randomized, double-blind, placebo-controlled, multicenter study. Patients are stratified based on hydroxyurea use and randomly assigned to receive either Poloxamer 407 or a placebo. The treatment begins within 12 hours of presenting with a crisis.
  • What is the duration of treatment and follow-up in the trial? The treatment with Poloxamer 407 or placebo is administered as a continuous infusion for 48 hours. Patients are then followed up on days 7-14 and again on days 28-35 after the initial treatment.
  • How is pain assessed during the trial? Pain is assessed before, during, and after the treatment. This allows researchers to evaluate the effectiveness of Poloxamer 407 in managing pain associated with vaso-occlusive crises in sickle cell disease patients.

Ongoing Clinical Trials on Poloxamer 407

  • Study on UGN-103 (Mitomycin) for Treating Low Grade Nonmuscle Invasive Bladder Cancer in Patients at Intermediate Risk of Recurrence

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Bulgaria Estonia Latvia Romania Spain

Glossary

  • Sickle Cell Disease: 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.
  • Vaso-occlusive Crisis: A common and painful complication of sickle cell disease where sickled red blood cells block blood flow through tiny blood vessels to your bones and organs.
  • Poloxamer 407: Also known as Poloxamer 188, it is an experimental drug being studied for its potential to reduce the duration and severity of vaso-occlusive crises in sickle cell disease.
  • Randomized Trial: A type of clinical trial where participants are randomly assigned to different treatment groups, reducing bias in the study results.
  • Double-blind: A study design where neither the participants nor the researchers directly involved know who is receiving the experimental treatment and who is receiving a placebo.
  • Placebo: A substance with no active therapeutic effect, used as a control in testing new drugs to help determine the efficacy of the experimental treatment.
  • Hydroxyurea: A medication commonly used in sickle cell disease to reduce the frequency of painful crises and the need for blood transfusions.
  • Analgesic: A medication used to relieve pain, often referred to as a painkiller.
  • Continuous Infusion: A method of administering medication where the drug is given intravenously at a constant rate over a specified period.

References

  1. https://clinicaltrials.gov/study/NCT00004408