Recruiting

Phase 2

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

Sponsor:

New York Medical College

Code:

NCT02675959

Conditions

Sickle Cell Disease

Eligibility Criteria

Sex: All

Age: 0 - 21

Healthy Volunteers: Not accepted

Interventions

Defibrotide

Study Details

Brief summary:

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.

Conditions

Sickle Cell Disease

Study ID

NCT02675959

Start date

Jul 1, 2017

Status verified date

Jul, 2026

Completion date

Dec, 2027

Anticipated

Primary completion date

Dec, 2026

Anticipated

Eligibility Criteria

Sex: All

Age: 0 - 21

Healthy Volunteers: Not accepted

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 Design

Enrollment

40 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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).

Interventions

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.

Primary outcome measure

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 ]
All patients will be monitored for the development of SOS. [ Time Frame: 1 year ]

Central Contacts and Locations

Central contacts

Mitchell S Cairo, MD

914-594-2150 Mitchell_Cairo@nymc.edu

Erin Morris, RN

714-964-5359 erin_morris@nymc.edu

Locations

University of California Los Angeles

Recruiting

Los Angeles, California, United States, 90095

Contacts

Theodore B Moore, MD

(310) 825-6708 tbmoore@mednet.ucla.edu

Ricardo Ortega

310-794-8929 RicardoOrtega@mednet.ucla.edu

Principal Investigator:

Theodore B Moore, MD

University of Florida

Recruiting

Gainesville, Florida, United States, 32610-0278

Contacts

Jordan Milner, MD

jordan.milner@peds.ufl.edu

Giselle Moore-Higgs, PhD

mooregj@ufl.edu

New York Medical College

Recruiting

Valhalla, New York, United States, 10595

Contacts

Megan Campbell, RN, PNP

(914) 594-2154 Megan.Campbell@wmchealth.org

Erin Morris, RN

714-964-5359 erin_morris@nymc.edu

Principal Investigator:

Mitchell S Cairo, MD

Medical College of Wisconsin

Recruiting

Milwaukee, Wisconsin, United States, 53226

Contacts

Julie A Talano, MD

414-955-4185 jtalano@mcw.edu

Meredith Beversdorf, BSN, RN

mbeversdorf@childrenswi.org

Principal Investigator:

Julie A Talano, MD

More Information

Sponsor

New York Medical College

Last update posted

Jul 27, 2026

Last verified

Jul, 2026

Keywords

stem cell transplantation
sickle cell disease
haploidentical
defibrotide
matched unrelated donor

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-09. This information was provided to ClinicalTrials.gov by New York Medical College on 2026-07-27. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.