Sickle Cell DiseasePhase 2NYMC-571NCT02675959
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
A Phase 2 study testing Defibrotide in people with Sickle Cell Disease, run by New York Medical College at 4 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 months to 21 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Defibrotide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 4 sitesUnited States
- Study dates
- 1 Jul 2017to Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
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- 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;
Who cannot join
- 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)
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- 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
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
University of Florida
Gainesville, Florida, 32610-0278
Jordan Milner, MD, jordan.milner@peds.ufl.edu
Giselle Moore-Higgs, PhD, mooregj@ufl.edu
- Recruiting
University of California Los Angeles
Los Angeles, California, 90095
Theodore B Moore, MD, (310) 825-6708, tbmoore@mednet.ucla.edu
Ricardo Ortega, 310-794-8929, RicardoOrtega@mednet.ucla.edu
- Recruiting
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Julie A Talano, MD, 414-955-4185, jtalano@mcw.edu
Meredith Beversdorf, BSN, RN, mbeversdorf@childrenswi.org
- Recruiting
New York Medical College
Valhalla, New York, 10595
Megan Campbell, RN, PNP, (914) 594-2154, Megan.Campbell@wmchealth.org
Erin Morris, RN, 714-964-5359, erin_morris@nymc.edu
About this study
Official title: 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.
- Conditions
- Sickle Cell Disease
- Being tested
- Defibrotide
- Keywords
- stem cell transplantation, sickle cell disease, haploidentical, defibrotide, matched unrelated donor
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DefibrotideDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Defibrotide prophylaxis | Experimental | 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). Receives: Drug: Defibrotide |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
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.
Measured: 100 days
All patients will be monitored for the development of SOS.
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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2016 (estimated)
- Study start1 Jul 2017
- Primary completionDec 2026 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- New York Medical College
- Sponsor type
- University, hospital or other
- Collaborators
- University of California, Los Angeles, Medical College of Wisconsin, Johns Hopkins University, Dana-Farber Cancer Institute, Children's Hospital Los Angeles, Children's Hospital Medical Center, Cincinnati, University of Florida
- Responsible party
- Principal investigator, Mitchell Cairo, New York Medical College
- Investigators
- Mitchell Cairo, MD, Principal investigator, New York Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02675959
- Sponsor's protocol ID
- NYMC 571
- Other IDs
- 7837
- Acronym
- NYMC-571
- Status last verified
- Jul 2026
- First submitted
- 22 Jan 2016
- Last updated
- 27 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Chu Y, Talano JA, Baxter-Lowe LA, Verbsky JW, Morris E, Mahanti H, Ayello J, Keever-Taylor C, Johnson B, Weinberg RS, Shi Q, Moore TB, Fabricatore S, Grossman B, van de Ven C, Shenoy S, Cairo MS. Donor chimerism and immune reconstitution following haploidentical transplantation in sickle cell disease. Front Immunol. 2022 Dec 9;13:1055497. doi: 10.3389/fimmu.2022.1055497. eCollection 2022.
PubMed 36569951
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT02675959
- Acronym / sponsor ID
- NYMC-571 / NYMC 571
- Conditions
- Sickle Cell Disease
- Keywords
- stem cell transplantation, sickle cell disease, haploidentical, defibrotide, matched unrelated donor
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- New York Medical CollegeUniversity, hospital or other
- Collaborators
- University of California, Los Angeles, Medical College of Wisconsin, Johns Hopkins University, Dana-Farber Cancer Institute, Children's Hospital Los Angeles, Children's Hospital Medical Center, Cincinnati, University of Florida
- Study start
- 1 Jul 2017 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 5 Feb 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Defibrotide prophylaxis | Experimental | Drug: Defibrotidedefibrotide 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). |
Who may qualify
- Ages
- 6 months to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (15 listed)
- 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).
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Defibrotidedefibrotide 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. | Drug | Defibrotide prophylaxis |
Primary outcome
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.
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2026.
- Derived from this study: PubMed 36569951Chu Y, Talano JA, Baxter-Lowe LA, Verbsky JW, Morris E, Mahanti H, Ayello J, Keever-Taylor C, Johnson B, Weinberg RS, Shi Q, Moore TB, Fabricatore S, Grossman B, van de Ven C, Shenoy S, Cairo MS. Donor chimerism and immune reconstitution following haploidentical transplantation in sickle cell disease. Front Immunol. 2022 Dec 9;13:1055497. doi: 10.3389/fimmu.2022.1055497. eCollection 2022.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Mitchell Cairo, MD principal investigator
New York Medical College
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Defibrotide in people with Sickle Cell Disease, run by New York Medical College at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 27 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mitchell S Cairo, MD (914-594-2150, Mitchell_Cairo@nymc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02675959, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jul 2026.