Summary
Arsenic Trioxide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Multiple Myeloma would be drug repurposing rather than first-in-human development. This does not mean it is approved for Multiple Myeloma. ClinicalTrials.gov lists 8 registered trials linking Arsenic Trioxide to Multiple Myeloma: 3 have completed; 5 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT00085345). The largest enrolment is 60 participants (NCT00469209). Registration activity spans 1999 to 2008. No published literature item is linked to Arsenic Trioxide and Multiple Myeloma in the OSMF database yet, so the record rests on registry entries alone. Registry entries describe intent to study, not outcomes.
The RepurpOS disease-intelligence file for Multiple Myeloma ranks 50 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: Essentially zero; MGUS (precursor) progresses to myeloma in 1%/year; median survival without treatment <1 year once symptomatic.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 33.5 (trials 13.5, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 0 active / not yet recruiting, 3 completed, 5 other |
| Linked publications | 0 |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | CCND1 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT00085345 | Melphalan, Arsenic Trioxide, and Ascorbic Acid in Treating Patients With Relapsed or Refractory Multiple Myeloma | withdrawn | Phase 2 | — |
| NCT00112879 | Arsenic Trioxide, Ascorbic Acid, Dexamethasone, and Thalidomide in Treating Patients With Multiple Myeloma | withdrawn | Phase 2 | — |
| NCT00258245 | Arsenic Trioxide and Ascorbic Acid Combined With Bortezomib, Thalidomide, and Dexamethasone in Treating Patients With Relapsed or Refractory Multiple Myeloma or Plasma Cell Leukemia 2005 | completed | Phase 1 | 5 |
| NCT00590603 | Trisenox, Ascorbic Acid and Bortezomib in Patients With Relapsed/Refractory Multiple Myeloma 2008 | terminated | Phase 1 | 25 |
| NCT00504101 | Bortezomib, Arsenic Trioxide, and Melphalan in Treating Patients Undergoing an Autologous Stem Cell Transplant For Multiple Myeloma 2007 | withdrawn | Phase 1 | — |
| NCT00006021 | Arsenic Trioxide Plus Vitamin C in Treating Patients With Recurrent or Refractory Multiple Myeloma 2000 | completed | PHASE1, PHASE2 | 22 |
| NCT00469209 | Velcade, Trisenox, Vitamin C and Melphalan for Myeloma Patients 2006 | completed | PHASE1, PHASE2 | 60 |
| NCT00006220 | Arsenic Trioxide With or Without Tretinoin in Treating Patients With Hematologic Cancer That Has Not Responded to Previous Therapy 1999 | terminated | PHASE1, PHASE2 | 5 |
Published literature
No publication is linked to this pair yet.
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
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 CCND1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Arsenic Trioxide approved for Multiple Myeloma?
Arsenic Trioxide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Multiple Myeloma would be drug repurposing rather than first-in-human development. This does not mean it is approved for Multiple Myeloma. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Arsenic Trioxide in clinical trials for Multiple Myeloma?
ClinicalTrials.gov lists 8 registered trials linking Arsenic Trioxide to Multiple Myeloma: 3 have completed; 5 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT00085345). The largest enrolment is 60 participants (NCT00469209). Registration activity spans 1999 to 2008.
What does the evidence show for Arsenic Trioxide in Multiple Myeloma?
Arsenic Trioxide has 3 completed registered trials for Multiple Myeloma but no linked publication, which usually means results are unpublished, pending, or not yet matched to this record. 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 CCND1. 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 2026-07-06. Page built 2026-10-07.