Neuroblastoma Clinical Trial
Official title:
Defibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy in Patients With High-Risk Neuroblastoma Receiving Tandem Transplants
This phase II trial tests how well defibrotide works in preventing transplant-associated thrombotic microangiopathy (TA-TMA) in patients with high-risk neuroblastoma undergoing tandem transplants (hematopoietic stem cell transplant [HSCT]). TMA is a potential life-threatening complication of stem cell transplant. TMA is a possible side effect of the chemotherapy (conditioning regimen) patients receive to help treat high-risk neuroblastoma, because these medicines can sometimes damage the blood vessel walls in the body. This damage leads to formation of tiny blood clots in organs, especially the kidney. This then causes organ damage and leads to problems with how they function. This study may help researchers learn how defibrotide may help prevent TMA before it starts, or help treat it once it starts among patients with high-risk neuroblastoma undergoing tandem transplants.
Status | Not yet recruiting |
Enrollment | 15 |
Est. completion date | March 31, 2027 |
Est. primary completion date | March 31, 2027 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Year to 18 Years |
Eligibility | Inclusion Criteria: 1. Age: 1 - < 18 years old. 2. Participants must have high-risk neuroblastoma according to Children's Oncology Group (COG) risk classification at the time of initial diagnosis. Participants who were initially considered low or intermediate risk, but then reclassified as high risk are also eligible. 3. Participants may have had salvage therapies (i.e., meta-iodobenzylguanidine (MIBG), dinutuximab) after induction but cannot have progressive disease at start of 4. Planned tandem transplants with conditioning with cyclophosphamide-thiotepa and carboplatin-etoposide-melphalan on, or as per, NCT03126916 (ANBL1531) or other tandem-containing COG high-risk neuroblastoma trials. 4. Participants may be enrolled in upfront neuroblastoma protocol, but this is not required. 5. Upfront MIBG or other therapies is not a contraindication. 6. Organ function per institutional standard of care (SOC) guiding clearance for autologous HSCT. 7. Written informed consent (and assent when applicable) obtained from participant or participant's legal representative and ability for subject to comply with the requirements of the study. Exclusion Criteria: 1. Life expectancy < 6 months. 2. Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study. 3. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data. 4. Known bleeding diathesis or bleeding risk deemed by the treating physician to be a contraindication to administration of defibrotide or on concomitant therapeutic anticoagulation. Administration of heparin and/or alteplase for central line maintenance is allowed. |
Country | Name | City | State |
---|---|---|---|
United States | University of California, San Francisco | San Francisco | California |
Lead Sponsor | Collaborator |
---|---|
University of California, San Francisco | Jazz Pharmaceuticals |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Cumulative incidence of transplant-associated thrombotic microangiopathy (TA-TMA) | The cumulative incidence of TA-TMA in participants will be compared to historical controls. The historic controls will be participants with high-risk neuroblastoma who underwent planned tandem transplant at University of California San Francisco (UCSF) between 2017 and 2023. | Up to1 year | |
Secondary | Cumulative incidence of severe TA-TMA | The cumulative incidence of severe TA-TMA in the patients enrolled on study compared to historical controls. | Up to 1 year |
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