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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 below

Select...

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.

NYMC-571 Trial 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

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 • 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

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

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Baylor College of MedicineOTHER

1,001 Previous Clinical Trials

6,002,172 Total Patients Enrolled

Johns Hopkins UniversityOTHER

2,264 Previous Clinical Trials

14,823,137 Total Patients Enrolled

Children's Hospital Los AngelesOTHER

232 Previous Clinical Trials

5,076,484 Total Patients Enrolled

Media Library

Defibrotide (Anticoagulant) Clinical Trial Eligibility Overview. Trial Name: NCT02675959 — Phase 2

Sickle Cell Disease Clinical Trial 2023: Defibrotide Highlights & Side Effects. Trial Name: NCT02675959 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many participants may join this trial at its peak?

"This clinical trial necessitates the active participation of 40 willing participants that meet the eligibility criteria. These individuals can join from University of California in Los Angeles and New york Medical College, based out of Valhalla respectively."

Answered by AI

Are there any available vacancies for this clinical experiment?

"According to the information housed on clinicaltrials.gov, this study is recruiting participants. It was initially listed on July 1st 2017 and has been modified as recently as June 15th 2022."

Answered by AI

Does the age requirement for this study extend beyond octogenarians?

"As per the requirements for enrolment, individuals must be between 6 months and 34 years of age to qualify for this clinical trial."

Answered by AI

Do I meet the qualifications to participate in this research endeavor?

"This clinical trial is enrolling forty individuals who are between 6 months and 34 years old, have anemia or sickle cell. Qualified candidates must have experienced one of the following: a clinically relevant neurologic event (e.g., stroke) that lasted for more than 24 hours with MRI evidence to corroborate; Sickle Cell nephropathy; Homozygous Hemoglobin S Disease, Hemoglobin S B0/+ thalassemia, Hemoglobin SC Disease, Beta thalassemia intermedia/majora; three painful episodes in two years prior to enrollment without hydroxyurea treatment or chronic RBC transfusions"

Answered by AI

Has Defibrotide been officially certified by the FDA?

"Given the Phase 2 status of this drug, there is limited evidence backing its safety profile which our team at Power assigned a score of 2. Unfortunately, efficacy data for Defibrotide are not yet available."

Answered by AI

Is Defibrotide an established remedy for any conditions as per past clinical trials?

"Presently, 4 clinical trials for Defibrotide are underway with no Phase 3 initiatives. With multiple of these studies running out of Boston, Massachusetts, 9 distinct sites have opened themselve up to hosting this research."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease

Mitchell Cairo 2017. "Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02675959.

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