BridgeMD
New treatment option for Sickle Cell Disease
Official title Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
ClinicalTrials.gov ID: NCT02675959
What this study is testing
What is Defibrotide?
Defibrotide is an investigational medicine, being studied as a potential treatment for sickle cell disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 to 34
You may be able to join if
- 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...
- 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...
- Recurrent painful events (at least 3 in the 2 years prior to enrollment or prior to chronic chronic RBC transfusion therapy, exchange transfusion or...
You likely can't join if
- 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)
See the full eligibility 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...
- 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;
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Gainesville, Florida, United States
- Valhalla, New York, United States
- Milwaukee, Wisconsin, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 months to 34 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Gainesville, Florida, United States; Valhalla, New York, United States; Milwaukee, Wisconsin, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.