Study Comparing Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients

Summary

This study is to compare the efficacy and safety of defibrotide prophylaxis in addition to best supportive care versus best supportive care alone in the prevention of hepatic veno- occlusive disease (VOD) in adult and pediatric patients undergoing hematopoietic stem cell transplant who are at high risk or very high risk of developing VOD.

Timeline

Start

2016-09-01

Primary completion

2020-10-20

Completion

2020-10-20

Outcome

Missed primary endpoint

paper HR 1·27 [95% CI 0·84-1·93]; p=0·85). PMID 37001534 ↗

paper Defibrotide did not show a benefit in the prophylaxis of sinusoidal obstruction syndrome. PMID 37001534 ↗

release “highly unlikely that the study would reach its primary endpoint” jazzpharma.com ↗

Publications

• Corbacioglu S, Grupp SA, Richardson PG, Duarte R, Pagliuca A, Ruutu T, Mahadeo K, Carreras E. Prevention of veno-occlusive disease/sinusoidal obstruction syndrome: a never-ending story and no easy answer. Bone Marrow Transplant. 2023 Aug;58(8):839-841. doi: 10.1038/s41409-023-02007-2. Epub 2023 May 25. No abstract available.
• Grupp SA, Corbacioglu S, Kang HJ, Teshima T, Khaw SL, Locatelli F, Maertens J, Stelljes M, Stepensky P, Lopez P, Amber V, Pagliuca A, Richardson PG, Mohty M. Defibrotide plus best standard of care compared with best standard of care alone for the prevention of sinusoidal obstruction syndrome (HARMONY): a randomised, multicentre, phase 3 trial. Lancet Haematol. 2023 May;10(5):e333-e345. doi: 10.1016/S2352-3026(23)00011-X. Epub 2023 Mar 28.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Defibrotide	Oligonucleotide (other)	6.25 mg/kg	Intravenous

Indications