Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease (NYMC-571)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02675959|
Recruitment Status : Recruiting
First Posted : February 5, 2016
Last Update Posted : June 21, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: Defibrotide||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Safety and Efficacy of Prophylactic Defibrotide in Children, Adolescents, and Young Adults With Sickle Cell Disease or Beta Thalassemia Following MAC and Haploidentical Stem Cell Transplantation Utilizing CD34 Enrichment and T-Cell (CD3) Addback|
|Actual Study Start Date :||July 1, 2017|
|Estimated Primary Completion Date :||December 2023|
|Estimated Study Completion Date :||December 2024|
Experimental: Defibrotide prophylaxis
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).
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.
- 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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||6 Months to 34 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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 .
- 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;
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02675959
|Contact: Mitchell S Cairo, MD||914-594-2150||Mitchell_Cairo@nymc.edu|
|Contact: Erin Morris, RNemail@example.com|
|United States, California|
|University of California Los Angeles||Recruiting|
|Los Angeles, California, United States, 90095|
|Contact: Theodore B Moore, MD 310-825-6708 firstname.lastname@example.org|
|Contact: Andres Vargas 310-794-8929 AndresVargas@mednet.ucla.edu|
|Principal Investigator: Theodore B Moore, MD|
|United States, Michigan|
|University of Michigan||Withdrawn|
|Ann Arbor, Michigan, United States, 48109-1274|
|United States, New York|
|New York Medical College||Recruiting|
|Valhalla, New York, United States, 10595|
|Contact: Sandra Fabricatore, RN, PNP 914-594-2152 email@example.com|
|Contact: Erin Morris, RN 714-964-5359 firstname.lastname@example.org|
|Principal Investigator: Mitchell S Cairo, MD|
|United States, Wisconsin|
|Medical College of Wisconsin||Recruiting|
|Milwaukee, Wisconsin, United States, 53226|
|Contact: Julie A Talano, MD 414-955-4185 email@example.com|
|Contact: Isabella Puls firstname.lastname@example.org|
|Principal Investigator: Julie A Talano, MD|
|Principal Investigator:||Mitchell Cairo, MD||New York Medical College|
|Responsible Party:||Mitchell Cairo, Principal Investigator, New York Medical College|
|Other Study ID Numbers:||
4090 ( Other Grant/Funding Number: OPD )
|First Posted:||February 5, 2016 Key Record Dates|
|Last Update Posted:||June 21, 2022|
|Last Verified:||June 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Undecided|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
stem cell transplantation
sickle cell disease
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Platelet Aggregation Inhibitors