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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
NYMC-571 Trial Summary
This trial will assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation.
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.Check my eligibility
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.See study design
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.
NYMC-571 Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have a specific type of blood disorder related to hemoglobin.
Select...
I am between 18 and 34 years old and meet at least two specified criteria.
NYMC-571 Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
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 • NCT0287660181%
Headache
38%
Flu-like Symptoms
19%
Chills
13%
Nausea
13%
Arthralgia
6%
Precollapse
6%
Dizziness
6%
Vertigo
100%
80%
60%
40%
20%
0%
Study treatment Arm
Defibrotide Plus LPS
Placebo Plus LPS
Defibrotide Plus Placebo
Placebo/Placebo
NYMC-571 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
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.
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.
Find a Location
Who is running the clinical trial?
Baylor College of MedicineOTHER
1,004 Previous Clinical Trials
6,003,243 Total Patients Enrolled
Johns Hopkins UniversityOTHER
2,275 Previous Clinical Trials
14,840,598 Total Patients Enrolled
Children's Hospital Los AngelesOTHER
236 Previous Clinical Trials
5,077,056 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have a specific type of blood disorder related to hemoglobin.I have complications from Sickle Cell Disease.I am between 18 and 34 years old and meet at least two specified criteria.I cannot take defibrotide due to health reasons.I have SCD and a family member who matches my HLA and is willing to donate.I have had a stem cell transplant before.I am currently on blood thinners or clot-dissolving drugs.I need considerable assistance and medical care.I have severe liver scarring.
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
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