NCT02675959 Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
| NCT ID | NCT02675959 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | New York Medical College |
| Condition | Sickle Cell Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 40 participants |
| Start Date | 2017-07-01 |
| Primary Completion | 2026-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 40 participants in total. It began in 2017-07-01 with a primary completion date of 2026-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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 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.
Eligibility Criteria
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 (or patients in Cohort 2 can meet other high risk criteria instead) * 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 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. * OR for Cohort #2 ONLY: Patient must be between 18 and 34.99 years of age, patients must demonstrate at least two of the following: * WBC \> 13,500 cells/microliter at baseline when not acutely ill (on two separate occasions) \> 2 weeks from a VOC event or hospitalization. * Tricuspid Regurgitant Jet Velocity (TRV) \> 3.0 m/s * Requiring Chronic Monthly Transfusions ( \> 12 transfusions in the 12 months) * History of sepsis * N-terminal pro-brain natriuretic peptide (NT-proBNP) \> 160 ng/L at clinical baseline when not acutely ill or hospitalized. * 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 * SCD Patients with documented uncontrolled infection at the time of study entry are not eligible. * SCD patients who have an unaffected HLA matched family 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
Contact & Investigator
Mitchell Cairo, MD
PRINCIPAL INVESTIGATOR
New York Medical College
Frequently Asked Questions
Who can join the NCT02675959 clinical trial?
This trial is open to participants of all sexes, aged 6 Months or older, up to 34 Years, studying Sickle Cell Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT02675959 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT02675959 currently recruiting?
Yes, NCT02675959 is actively recruiting participants. Contact the research team at Mitchell_Cairo@nymc.edu for enrollment information.
Where is the NCT02675959 trial being conducted?
This trial is being conducted at Los Angeles, United States, Gainesville, United States, Valhalla, United States, Milwaukee, United States.
Who is sponsoring the NCT02675959 clinical trial?
NCT02675959 is sponsored by New York Medical College. The principal investigator is Mitchell Cairo, MD at New York Medical College. The trial plans to enroll 40 participants.
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