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New treatment option for Sinusoidal Obstruction Syndrome
Official title Defibrotide Dose-escalation for SOS Post-HSCT
ClinicalTrials.gov ID: NCT05987124
What this study is testing
What is Defibrotide?
Defibrotide is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for sinusoidal obstruction syndrome.
Also referred to as Defitelio.
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 research study is being done to determine the safety and tolerability of increasing doses of defibrotide within a single patient with sinusoidal obstructive syndrome (SOS)/veno-occlusive disease (VOD) after hematopoietic cell transplantation (HCT) associated with either kidney and/or lung impairment that has not obtained a complete response (CR) or progressed in severity with standard doses of defibrotide.
- 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 1 to 75
You may be able to join if
- HCT recipients (Auto or Allograft)
- SOS/VOD as defined by Cairo/Cooke Diagnostic criteria (1) (Table 3) with either renal and/or pulmonary dysfunction as defined by Cairo/Cooke Grading...
- Unresponsive to standard defibrotide therapy as defined by at least one of the following:
- Patients with SOS/VOD failing to obtain a complete response (CR) defined by Grade I or less by Cairo/Cooke Grading criteria (1) (Appendix I). This...
- Progressive disease defined by progression of at least one grade or more from diagnostic grade as defined by Cairo/Cooke Grading criteria (1)...
You likely can't join if
- Patients who did not receive HCT.
- Concomitant systemic anticoagulation (excluding central venous line management, fibrinolytic instillation for central venous line occlusion...
- Active bleeding and/or hemorrhage of at least grade 2 and above.
- History of development of Grade III/IV anaphylaxis probably or directly secondary to defibrotide.
- Female patients who are pregnant or breast feeding.
See the full eligibility criteria
- HCT recipients (Auto or Allograft)
- SOS/VOD as defined by Cairo/Cooke Diagnostic criteria (1) (Table 3) with either renal and/or pulmonary dysfunction as defined by Cairo/Cooke Grading criteria (1) (Appendix I).
- Unresponsive to standard defibrotide therapy as defined by at least one of the following:
- Patients with SOS/VOD failing to obtain a complete response (CR) defined by Grade I or less by Cairo/Cooke Grading criteria (1) (Appendix I). This would therefore include patients with stable disease after at least 14...
- Progressive disease defined by progression of at least one grade or more from diagnostic grade as defined by Cairo/Cooke Grading criteria (1) (Appendix I) following at least 7 days of defibrotide (25mg/kg/day).
- Age 1 month - 75 years
- Patients who did not receive HCT.
- Concomitant systemic anticoagulation (excluding central venous line management, fibrinolytic instillation for central venous line occlusion, management of intermittent dialysis or ultrafiltration of CVVH).
- Active bleeding and/or hemorrhage of at least grade 2 and above.
- History of development of Grade III/IV anaphylaxis probably or directly secondary to defibrotide.
- Female patients who are pregnant or breast feeding.
The study team makes the final eligibility decision.
Where it's taking place
- Vallhala, New York, 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, 1 month to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Vallhala, New York, 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.