Summary
Defibrotide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Peripheral Artery Disease (PAD) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Peripheral Artery Disease (PAD). ClinicalTrials.gov lists 1 registered trial linking Defibrotide to Peripheral Artery Disease (PAD): 1 has completed. The most advanced is Phase 4 (NCT01718288). It plans or enrolled 150 participants. Registered activity dates to 2006. The literature layer holds 3 publications for this pair: 3 clinical trial publications. Publication years run from 1991 to 1996. These are primary trial reports rather than syntheses, so results have not yet been pooled or graded independently.
The RepurpOS disease-intelligence file for Peripheral Arterial Disease ranks 292 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Progressive disease; spontaneous improvement rare; limb ischaemia worsens in 25-30% over 5 years; major adverse limb events (MALE) in 10-15%/year in advanced PAD; major amputation in 5-10% at 5 years without revascularisation.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 30.5 (trials 4.5, literature 6.0, tier 15, approved bonus 5.0) |
| Registered trials | 1 total: 0 recruiting, 0 active / not yet recruiting, 1 completed, 0 other |
| Linked publications | 3 (3 clinical trial publications) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | agonist |
| Data sources | DGIdb |
| Linked via biomarker / target | ADORA2A |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT01718288 | Optimization of Treatment in Patients With Severe Peripheral Ischemia (Fontaine Stage IIb) 2006 | completed | Phase 4 | 150 |
Published literature
- Clinical trial publication Plasma levels of the molecular markers of coagulation and fibrinolysis in patients with peripheral arterial diseaseStrano A, Hoppensteadt D, Walenga JM et al. · Seminars in thrombosis and hemostasis · 1996 · PMID 8807727
- Clinical trial publication Effects of defibrotide on physical performance and hemorheologic picture in patients with peripheral arteriopathyMarrapodi E, Leanza D, Giordano S et al. · Clinical trials and meta-analysis · 1994 · PMID 10150182
- Clinical trial publication A double-blind, multicenter, placebo-controlled, dose comparison study of orally administered defibrotide: preliminary results in patients with peripheral arterial diseaseStrano A, Fareed J, Sabbá C et al. · Seminars in thrombosis and hemostasis · 1991 · PMID 1948094
Mechanism and notes
Recorded mechanism or class: agonist.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target ADORA2A. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Defibrotide approved for Peripheral Artery Disease (PAD)?
Defibrotide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Peripheral Artery Disease (PAD) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Peripheral Artery Disease (PAD). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Defibrotide in clinical trials for Peripheral Artery Disease (PAD)?
ClinicalTrials.gov lists 1 registered trial linking Defibrotide to Peripheral Artery Disease (PAD): 1 has completed. The most advanced is Phase 4 (NCT01718288). It plans or enrolled 150 participants. Registered activity dates to 2006.
What does the evidence show for Defibrotide in Peripheral Artery Disease (PAD)?
Defibrotide has 1 completed trial and 3 linked publications for Peripheral Artery Disease (PAD). Completed trials may or may not have posted results; follow the NCT links to check. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target ADORA2A. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-05. Page built 2026-10-07.