Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease (NYMC-571)

July 24, 2026 updated by: Mitchell Cairo, New York Medical College

The Safety and Efficacy of Prophylactic Defibrotide Administration in Children, Adolescents, and Young Adults With Sickle Cell Disease or Beta Thalassemia Following Myeloablative Conditioning (MAC) and Haploidentical or Matched Unrelated Donor (MUD) Stem Cell Transplantation Utilizing CD34 Enrichment and T-Cell (CD3) Addback

This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical or MUD allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS. The Funding Source is FDA OOPD.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

40

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Recruiting
        • University of California Los Angeles
        • Principal Investigator:
          • Theodore B Moore, MD
        • Contact:
        • Contact:
    • Florida
    • New York
      • Valhalla, New York, United States, 10595
        • Recruiting
        • New York Medical College
        • Contact:
        • Principal Investigator:
          • Mitchell S Cairo, MD
        • Contact:
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Medical College of Wisconsin
        • Contact:
        • Principal Investigator:
          • Julie A Talano, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 30 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Disease: Homozygous Hemoglobin S Disease, or Hemoglobin S B0/+ thalassemia, or Hemoglobin SC Disease, or Beta thalassemia intermedia/majora
  • Patients must demonstrate one or more of the following Sickle Cell Disease Complications
  • Clinically significant neurologic event (stroke) or any neurologic deficit lasting >24 hours that is accompanied by an infarct on cerebral MRI
  • Acute chest syndrome in the preceding two year period prior to enrollment that have failed, been non-compliant or declined hydroxyurea treatment, or prior to chronic RBC transfusion therapy, exchange transfusion or erythrocyte pheresis.
  • Recurrent painful events (at least 3 in the 2 years prior to enrollment or prior to chronic RBC transfusion therapy, exchange transfusion or erythrocyte pheresis).
  • Abnormal TCD study requiring starting on chronic transfusion therapy and/or exchange transfusions.
  • At least one silent infarct lesion on a MRI scan of the head. Or (directly or probably related to SCD)
  • Sickle Cell nephropathy;
  • Splenic sequestration requiring RBC transfusion;
  • Aplastic crisis requiring RBC transfusion;
  • Avascular necrosis of the hip diagnosed by MRI;
  • Two episodes or more of leg ulcerations;
  • Recurrent priapism .
  • Infant dactylitis.
  • all patients must meet disease, age, organ function and donor criteria;

Exclusion Criteria:

  • Patients who are receiving concomitant systemic anticoagulants and/or fibrinolytic therapies.
  • Patients with a previously known hypersensitivity reaction to defibrotide.
  • Females who are pregnant or breast-feeding are not eligible
  • Patients with documented uncontrolled infection at the time of study entry are not eligible.
  • Patients who have an unaffected HLA matched sibling donor willing to proceed to donation will not be eligible for this study.
  • Karnofsky or Lansky (age appropriate) Performance Score <50% (hemiplegia alone secondary to a previous stroke is not an exclusion)
  • Demonstrated lack of compliance with medical care.
  • Patients with clinically significant fibrosis or cirrhosis of the liver will not be eligible.
  • Patients who have previously received a HSCT will not be eligible.
  • Patients with contraindications to the use of defibrotide

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Defibrotide prophylaxis
defibrotide will be given prior to and during myeloablative immunotherapy conditioning (MAIC) followed by familial haploidentical (FHI) or matched unrelated donor (MUD) 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).
Drug: Defibrotide
defibrotide will be given prophylactically prior to AlloSCT to determine if it decreases the incidence of SOS in this high risk population, and determine that it is safe and feasible to give along with myeloimmunoablative therapy and allogeneic transplant.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Time Frame: 100 days
Patients will be given Defibrotide prophylaxis starting 10 days before the stem cell infusion at 6.25 mg/kg IV q6h and continue through Day +21.
100 days
All patients will be monitored for the development of SOS.
Time Frame: 1 year
All patients will get daily lab values while in patients and then as needed to monitor for elevation in liver function tests and other abnormal chemistry or hematology values. Imaging on the liver will be performed as needed to determine if they develop SOS with defibrotide.
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

New York Medical College

Collaborators

Johns Hopkins University
University of California, Los Angeles
Dana-Farber Cancer Institute
University of Florida
Medical College of Wisconsin
Children's Hospital Medical Center, Cincinnati
Children's Hospital Los Angeles

Investigators

  • Principal Investigator: Mitchell Cairo, MD, New York Medical College

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 1, 2017

Primary Completion (Estimated)

December 1, 2026

Study Completion (Estimated)

December 1, 2027

Study Registration Dates

First Submitted

January 22, 2016

First Submitted That Met QC Criteria

February 2, 2016

First Posted (Estimated)

February 5, 2016

Study Record Updates

Last Update Posted (Actual)

July 27, 2026

Last Update Submitted That Met QC Criteria

July 24, 2026

Last Verified

July 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NYMC 571
  • 7837 (Other Grant/Funding Number: OPD)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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