Increasing doses of defibrotide for treating sinusoidal obstruction syndrome after transplant
A Phase II Intrapatient Open-Label Dose Escalation Trial of Defibrotide in Hematopoietic Cell Transplantation (HCT) Recipients With Sinusoidal Obstructive Syndrome (SOS) Post-HCT Associated With Either Renal and/or Pulmonary Dysfunction With Either Refractory or Progressive Disease Following Defibrotide Therapy
This study is testing whether higher doses of defibrotide can help people with sinusoidal obstruction syndrome after a transplant who haven't improved with standard doses.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 1 Month to 75 Years |
| Sex | All |
| Sponsor | New York Medical College Academic / other |
| Locations | 1 site (Vallhala, New York) |
| Trial ID | NCT05987124 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the safety and tolerability of escalating doses of defibrotide in patients suffering from sinusoidal obstruction syndrome (SOS) or veno-occlusive disease (VOD) following hematopoietic cell transplantation (HCT). The study focuses on patients who have not achieved a complete response or have shown disease progression with standard defibrotide doses. Participants will receive higher doses of the medication to assess its effectiveness in improving their condition. The trial is designed to provide insights into alternative dosing strategies for patients with severe symptoms.
Who should consider this trial
Good fit: Ideal candidates include HCT recipients aged 1 month to 75 years with SOS/VOD who have not responded to standard defibrotide therapy.
Not a fit: Patients who have not undergone hematopoietic cell transplantation or are on systemic anticoagulation may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could offer a new treatment option for patients with SOS/VOD who do not respond to standard therapy.
How similar studies have performed: While there is limited information on similar studies, the approach of dose escalation in this context is relatively novel and may provide new insights into treatment options.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion 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 days of defibrotide or partial response after at least 21 days of defibrotide (25mg/kg/day). * 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 Exclusion Criteria: * 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.
Where this trial is running
Vallhala, New York
- New York Medical College — Vallhala, New York, United States (Recruiting)
Study contacts
- Principal investigator: Mitchell Cairo, MD — New York Medical College
- Study coordinator: Mitchell Cairo, MD
- Email: mitchell_cairo@nymc.edu
- Phone: 914-594-2150
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.