{"entity":{"id":"study-2005-01","kind":"trial","name":"Study 2005-01","aka":["2005-01","Defitelio Study 1"],"tldr":"Study 2005-01 showed that defibrotide helped more people survive to day 100 after a stem-cell transplant when blocked liver veins had also led to kidney or lung failure, and it was the main evidence for the US approval of Defitelio.","summary":"Study 2005-01 was a multicentre, open-label, historically controlled phase 3 trial of defibrotide in adults and children with established hepatic veno-occlusive disease (sinusoidal obstruction syndrome) and advanced multi-organ failure after haematopoietic stem-cell transplant. One hundred and two patients received defibrotide at 25 mg per kilogram per day (6.25 mg per kilogram every six hours) for at least 21 days. They were compared with 32 historical controls, chosen by blinded independent reviewers from 6867 medical charts of transplant patients, and baseline characteristics were well balanced. The primary endpoint was survival at day 100 after transplant, using a propensity-adjusted analysis.\n\nDay-100 survival was 38.2 percent with defibrotide and 25 percent in the historical controls (estimated difference 23 percent, 95.1% CI 5.2 to 40.8, P = .0109). Complete response by day 100 was 25.5 percent against 12.5 percent (difference 19 percent, 95.1% CI 3.5 to 34.6, P = .0160). Bleeding and low blood pressure were the related adverse events, and common bleeding events (pulmonary alveolar 11.8 against 15.6 percent, gastrointestinal 7.8 against 9.4 percent) occurred at similar rates in the two groups (Blood 2016). The registry results add day-180 survival of 32.4 against 25.0 percent.\n\nThe US label describes the same 102 patients as Study 1, reports their day-100 survival as 38 percent (95% CI 29 to 48) and bases the approval on it. The registry counts 134 participants (102 treated plus 32 controls) and records the study as completed. The FDA approved defibrotide (Defitelio) in March 2016 for hepatic veno-occlusive disease with renal or pulmonary dysfunction after transplant. The trial treats a complication of transplant rather than a cancer, so it carries no cancer ids.","status":"positive","asOf":"2026-09-24","links":[{"label":"ClinicalTrials.gov NCT00358501","url":"https://clinicaltrials.gov/study/NCT00358501"},{"label":"Blood 2016","url":"https://doi.org/10.1182/blood-2015-10-676924"},{"label":"Defitelio label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2c3db989-d7ad-41ed-9ebf-698dcf6c24ec"}],"tags":[],"related":[],"cancers":[],"sections":[],"technologies":["allogeneic-hsct","autologous-stem-cell-transplant"],"targets":[],"drugs":["defibrotide"],"companies":["jazz"],"institutions":[],"pathways":[],"terms":["allogeneic-transplant"],"trials":[],"people":["paul-richardson"],"bottlenecks":[],"keyPapers":["paper-defibrotide-2005-01-blood-2016"],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT00358501","phase":"3","setting":"Severe hepatic veno-occlusive disease with multi-organ failure after stem-cell transplant: defibrotide in treated patients, compared with historical controls chosen by blinded review","sponsor":"Jazz Pharmaceuticals","result":"Day-100 survival 38.2% with defibrotide against 25% in historical controls (propensity-adjusted difference 23%, P = .0109); complete response by day 100 25.5% against 12.5%.","yearReported":2016,"enrolled":134,"enrolledBasis":"registry","outcomes":[{"endpoint":"Survival at day 100 after transplant","primary":true,"unit":"%","arms":[{"name":"Defibrotide","n":102,"value":38.2,"note":"Propensity-adjusted difference against historical control 23% (95.1% CI 5.2 to 40.8)"},{"name":"Historical control","n":32,"value":25}],"p":"0.0109","source":"https://doi.org/10.1182/blood-2015-10-676924"},{"endpoint":"Complete response by day 100 after transplant","unit":"%","arms":[{"name":"Defibrotide","n":102,"value":25.5,"note":"Difference against historical control 19% (95.1% CI 3.5 to 34.6), same propensity-adjusted method"},{"name":"Historical control","n":32,"value":12.5}],"p":"0.0160","source":"https://doi.org/10.1182/blood-2015-10-676924"},{"endpoint":"Survival at day 180 after transplant","unit":"%","arms":[{"name":"Defibrotide","n":102,"value":32.4},{"name":"Historical control","n":32,"value":25}],"source":"https://clinicaltrials.gov/study/NCT00358501?tab=results"}]},"route":"/trials/study-2005-01/","neighbours":{"technology":[{"id":"allogeneic-hsct","kind":"technology","name":"Allogeneic stem cell transplantation","route":"/technologies/allogeneic-hsct/"},{"id":"autologous-stem-cell-transplant","kind":"technology","name":"Autologous stem cell transplant (high-dose therapy)","route":"/technologies/autologous-stem-cell-transplant/"}],"drug":[{"id":"defibrotide","kind":"drug","name":"Defibrotide","route":"/drugs/defibrotide/"}],"company":[{"id":"jazz","kind":"company","name":"Jazz Pharmaceuticals","route":"/companies/jazz/"}],"term":[{"id":"allogeneic-transplant","kind":"term","name":"Allogeneic stem cell transplant (allo-SCT)","route":"/terms/allogeneic-transplant/"}],"person":[{"id":"paul-richardson","kind":"person","name":"Paul G. Richardson","route":"/people/paul-richardson/"}],"paper":[{"id":"paper-defibrotide-2005-01-blood-2016","kind":"paper","name":"Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure","route":"/key-papers/paper-defibrotide-2005-01-blood-2016/"}]}}