Defibrotide

Summary

Defibrotide (CHEMBL2108396) is a phase-3 clinical-stage unknown (ATC B01AX01); indicated across 8 conditions including thrombotic disease and hepatic veno-occlusive disease.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Unknown
• ATC class: B01AX01
• Indications: 8 conditions
• Clinical trials: 22

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2108396
Name	Defibrotide
Type	Unknown
Max phase	3
ATC	B01AX01

Also known as: Bos taurus lung defibrotide, Defibrotida, Defibrotide, Defibrotide (bovine lung), Defibrotide (bovine), DEFIBROTIDE

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

8 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
thrombotic disease	3	MONDO:0000831	HP:0004419
hepatic veno-occlusive disease	3	MONDO:0019514	MONDO:0019514
graft versus host disease	2	MONDO:0013730	EFO:0004599
Kawasaki disease	2	MONDO:0012727	EFO:0004246
severe acute respiratory syndrome	2	MONDO:0005091	MONDO:0100096
sickle cell disease	2	MONDO:0011382	MONDO:0011382
plasma cell myeloma	1	MONDO:0009693	EFO:0001378

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 22.

Phase distribution

Phase	Trials
PHASE2	13
PHASE3	3
Not specified	2
PHASE4	1
PHASE2/PHASE3	1
PHASE1/PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02876601	PHASE4	COMPLETED	Defibrotide in the Human Endotoxemia Model — an Exploratory Trial Investigating the Effects and the Mechanisms of Defibrotide
NCT00272948	PHASE2/PHASE3	COMPLETED	Pediatric Trial Investigating the Incidence & Outcome of Veno-Occlusive Disease With the Prophylactic Use of Defibrotide
NCT00358501	PHASE3	COMPLETED	Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients
NCT00628498	PHASE3	COMPLETED	Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study
NCT02851407	PHASE3	COMPLETED	Study Comparing Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients
NCT02675959	PHASE2	RECRUITING	Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
NCT04652115	PHASE2	RECRUITING	Defibrotide for the Treatment of Severe COVID-19
NCT05987124	PHASE2	RECRUITING	Defibrotide Dose-escalation for SOS Post-HSCT
NCT00003966	PHASE2	COMPLETED	Defibrotide in Treating Patients With Liver Damage Following Peripheral Stem Cell Transplantation
NCT00406978	PHASE1/PHASE2	COMPLETED	Melphalan, Prednisone, Thalidomide and Defibrotide in Relapsed Multiple Myeloma Patients
NCT03339297	PHASE2	COMPLETED	An Open-Label Study of Defibrotide for the Prevention of Acute Graft-versus-Host-Disease (AGvHD)
NCT03384693	PHASE2	COMPLETED	Defibrotide TMA Prophylaxis Pilot Trial
NCT03805581	PHASE2	COMPLETED	Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome
NCT03954106	PHASE2	TERMINATED	A Safety and Efficacy Study of Defibrotide in the Prevention of Chimeric Antigen Receptor-T-cell-associated Neurotoxicity
NCT04313036	PHASE2	UNKNOWN	5-day Defibrotide Treatment for Hepatic SOS/VOD
NCT04335201	PHASE2	COMPLETED	Defibrotide in COVID-19 Pneumonia
NCT04348383	PHASE2	COMPLETED	Defibrotide as Prevention and Treatment of Respiratory Distress and Cytokine Release Syndrome of Covid 19.
NCT04777422	PHASE2	UNKNOWN	Defibrotide in Children With High Risk Kawasaki Disease
NCT06182410	PHASE2	WITHDRAWN	Defibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy for Neuroblastoma
NCT04530604	PHASE1	COMPLETED	Defibrotide Therapy for SARS-CoV2 (COVID-19) Acute Respiratory Distress Syndrome (ARDS)
NCT00143546	Not specified	NO_LONGER_AVAILABLE	Compassionate Use of Defibrotide for Patients With Veno-occlusive Disease of the Liver
NCT00263978	Not specified	NO_LONGER_AVAILABLE	Defibrotide Compassionate Use for Patients With Life Threatening Veno-Occlusive Disease of the Liver

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).