What side effects are associated with this type of intervention?

Common treatments for Sickle Cell Disease (SCD) include L-glutamine, hydroxyurea, and red blood cell transfusions. L-glutamine is generally well-tolerated with mild gastrointestinal symptoms being the most common side effect. Hydroxyurea can cause bone marrow suppression, leading to lower blood cell counts, and should be monitored for long-term side effects. Red blood cell transfusions carry risks such as iron overload and alloimmunization. Defibrotide, used prophylactically in high-risk SCD patients undergoing stem cell transplantation, is primarily aimed at preventing Sinusoidal Obstructive Syndrome (SOS) and has shown efficacy in this context.

What prior FDA approvals exist?

The FDA has approved several treatments for Sickle Cell Disease (SCD). Hydroxyurea is one of the most established treatments, known for reducing vaso-occlusive pain and other complications. L-glutamine has also been approved to reduce acute complications of SCD. While Defibrotide is being studied for the prevention of Sinusoidal Obstructive Syndrome (SOS) in high-risk SCD patients undergoing stem cell transplantation, it is not yet approved for this specific use in SCD. Other investigational therapies and disease-modifying treatments are also being explored to improve patient outcomes.

What other types of research exist?

While the context does not provide specific novel alternatives to Defibrotide for the prevention of Sinusoidal Obstructive Syndrome in Sickle Cell Disease patients, hydroxyurea remains a cornerstone therapy for SCD due to its efficacy in reducing complications. Additionally, ongoing research into other disease-modifying therapies, such as decitabine, butyrate derivatives, and new medications targeting vaso-occlusion, may offer future alternatives. Consultation with a healthcare provider is essential to explore the most current and suitable treatment options.

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Anticoagulant

Defibrotide Prophylaxis + Stem Cell Transplant for Sickle Cell Disease (NYMC-571 Trial)

Phase 2

Recruiting

Led By Mitchell Cairo, MD

Research Sponsored by New York Medical College

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients with Homozygous Hemoglobin S Disease, Hemoglobin S B0/+ thalassemia, Hemoglobin SC Disease, or Beta thalassemia intermedia/majora

Patients in Cohort 2 must be between 18 and 34.99 years of age and demonstrate at least two specified criteria

Must not have

Patients with contraindications to the use of defibrotide

SCD patients with an unaffected HLA matched family donor willing to proceed to donation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial tests if Defibrotide can safely and effectively prevent liver damage in high-risk sickle cell or beta thalassemia patients undergoing a special stem cell transplant. The medication works by improving blood flow in the liver to prevent blockages.

Who is the study for?

This trial is for high-risk sickle cell or beta thalassemia patients who have had severe complications like acute chest syndrome, stroke, or organ damage. It's open to those with specific genetic forms of the disease and certain health indicators like elevated NT-proBNP levels. Adults up to age 34.99 can join if they meet additional criteria such as a history of frequent blood transfusions or leg ulcers.

What is being tested?

The study tests Defibrotide prophylaxis in patients undergoing haploidentical allogeneic stem cell transplantation (a type of bone marrow transplant). The goal is to prevent sinusoidal obstructive syndrome (SOS), a serious complication that can occur after transplants in these patients.

What are the potential side effects?

Defibrotide may cause bleeding problems, low blood pressure, diarrhea, vomiting, nausea and possible allergic reactions. Since it affects blood clotting, there's an increased risk of hemorrhage especially when used with other anticoagulants.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a specific type of blood disorder related to hemoglobin.

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I am between 18 and 34 years old and meet at least two specified criteria.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I cannot take defibrotide due to health reasons.

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I have SCD and a family member who matches my HLA and is willing to donate.

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I have had a stem cell transplant before.

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I am currently on blood thinners or clot-dissolving drugs.

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I need considerable assistance and medical care.

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I have severe liver scarring.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

All patients will be monitored for known and unknown side effects of defibrotide with daily physical exams while in the hospital and then as needed in addition to daily laboratory values including chemistries, hematology labs as needed

All patients will be monitored for the development of SOS.

Side effects data

From 2018 Phase 4 trial • 20 Patients • NCT02876601

81%

Headache

38%

Flu-like Symptoms

19%

Chills

13%

Nausea

13%

Arthralgia

Precollapse

Dizziness

Vertigo

100%

80%

60%

40%

20%

Study treatment Arm

Defibrotide Plus LPS

Placebo Plus LPS

Defibrotide Plus Placebo

Placebo/Placebo

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Defibrotide prophylaxisExperimental Treatment1 Intervention

defibrotide will be given prior to and during myeloablative immunotherapy conditioning (MAIC) followed by familial haploidentical (FHI) allogeneic stem cell transplantation (AlloSCT) with CD34 enrichment and t-cell addback in patients with high-risk sickle cell disease or beta thalassemia to reduced the risk and rate of the development of sinusoidal obstructive syndrome (SOS).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Defibrotide

2018

Completed Phase 4

~2490

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Sickle Cell Disease (SCD) include hydroxyurea, red blood cell transfusions, and investigational therapies like Defibrotide. Hydroxyurea works by increasing fetal hemoglobin (HbF) production, which reduces the sickling of red blood cells and decreases vaso-occlusive episodes. Red blood cell transfusions help by diluting the sickle hemoglobin (HbS) with normal hemoglobin, thereby reducing the risk of complications such as stroke and acute chest syndrome. Defibrotide, although primarily studied for preventing SOS in stem cell transplantation, has anti-inflammatory and anti-thrombotic properties that may benefit SCD patients by protecting the vascular endothelium. These treatments are crucial for SCD patients as they help manage pain, prevent complications, and improve overall quality of life.

Sickle cell dehydration: Pathophysiology and therapeutic applications.The controversial role of red cell transfusions for sickle cell pain.Drugs for preventing red blood cell dehydration in people with sickle cell disease.