Melphalan clinical trials
Melphalan is named as an intervention in 32 current clinical trials in our China, Canada, Israel, Spain, Australia, Germany, Italy, France, Mexico, USA, UK, Netherlands, Czechia, Denmark, Norway, Brazil, Egypt, Belgium, Sweden, Switzerland, Turkey, Poland, Japan, South Korea, Taiwan, Singapore and Austria registers. 19 are recruiting patients now. The most common phase is Phase 2. The sponsor with the most trials is National Cancer Institute (NCI) (4). The most-studied condition is Multiple Myeloma. The EU clinical trials register (CTIS) separately lists 1 authorised or ongoing trial.
Where Melphalan is approved
All regulators →Of the three regulators tracked here, the first to approve Melphalan was the US, on 17 Jan 1964. It is approved in 2 of the 3 regulators tracked here.
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Melphalan patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Melphalan, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 18 listed patents; the last listed expires 29 Jun 2036 (US 10537520).
- Exclusivity ODE-438: Orphan drug exclusivity: as a liver-directed treatment for adult patients with uveal melanoma with unresectable hepatic metastases affectin…, until 14 Aug 2030.
Reproduced from the FDA Orange Book and Purple Book (checked 8 Nov 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
What health systems spend on Melphalan
All medicines →From NHSBSA Prescription Cost Analysis (Open Government Licence v3.0) and CMS State Drug Utilization Data. Both are gross amounts: neither system publishes its discounts or rebates per drug. Products combining several substances are not counted here.
How Melphalan works
All drug targets →- AL amyloidosisPhase 3
- Ewing sarcomaPhase 3
- Acute lymphoblastic leukemiaPhase 3
- AmyloidosisPhase 3
- Breast cancerPhase 3
- Chronic myeloid leukemiaPhase 3
- Classic Hodgkin lymphomaPhase 3
- Colorectal cancerPhase 3
- Diffuse large B-cell lymphomaPhase 3
- Follicular lymphomaPhase 3
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL852, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 7620, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
NICE appraisals of Melphalan
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 1 technology appraisal of Melphalan. 1 is current.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Melphalan for haematological diseases before allogeneic haematopoietic stem cell transplant (terminated appraisal) NICE TA822 | Terminated (no company submission) | 14 Sep 2022 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Melphalan
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 3 products on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: PhelinunEMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Reference identifiers for Melphalan
- ATC code
- L01AA03
- ChEMBL
- CHEMBL852
- DrugBank
- DB01042
- PubChem CID
- 6918992
- CAS number
- 148-82-3
- FDA UNII
- Q41OR9510P
- Wikidata
- Q2298283
- Wikipedia
- Melphalan
Codes that identify this medicine in other databases, from Wikidata (CC0), matched by its ChEMBL ID. The ATC code is the WHO classification of what the medicine is used for.
Trials by status
Trials by phase
Clinical trials of Melphalan
32 trialsRegisters list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Melphalan in the EU trials register (CTIS)
1 trialAuthorised or ongoing trials in the EU Clinical Trials Information System that list a product containing Melphalan. Many trials are registered in both CTIS and ClinicalTrials.gov, so these are shown separately rather than added to the count above. Trials with a site in Ireland are listed first.
| Study | Phase | Status | Sponsor | Countries |
|---|---|---|---|---|
|
A Phase III, Open-label, Multicentre, Randomised Study Comparing Consolidation Treatment with AZD0120, an Autologous Dual Targeting Chimeric Antigen Receptor T-cell (CAR-T) Therapy Directed Against BCMA and CD19, versus Autologous Stem Cell Transplant (ASCT) in Participants with Newly Diagnosed Multiple Myeloma who are Transplant Eligible (TE NDMM) (DURGA-6)
2026-525367-40-00 · CTIS ↗ · Neoplasms
|
Phase 3 | Authorised | AstraZeneca AB | Germany, Spain, Norway + 4 more |
Melphalan across our data
Ireland · EU · USThe same active substance in our other registers, matched by its exact name. Each entry links to the register record it comes from.
Licensed in Ireland (HPRA)
3 single-substance products containing Melphalan on the HPRA medicines list.
- Alkeran 2 mg Film-coated TabletsAspen Pharma Trading Limited · PA1691/004/002 · Marketed
- Alkeran 50 mg Powder and Solvent for Solution for InfusionAspen Pharma Trading Limited · PA1691/004/001 · Marketed
- Melphalan Tillomed 50 mg powder and solvent for solution for injection/infusionTillomed Pharma GmbH · PA22720/006/001 · Not marketed
Supply shortages
- Melphalan Hydrochloride InjectionUS (FDA) · Resolved · updated 16 Sep 2026
- Melphalan-ratiopharm 50 mg Pulver und Lösungsmittel zur Herstellung einer Injektions- bzw. InfusionslösungGermany (BfArM) · Production problem · 5 Jun 2026 to 31 Dec 2026
Conditions studied with Melphalan
50 conditionsAll 50 conditions
| Condition (as registered) | Trials |
|---|---|
| Multiple Myeloma | 5 |
| Retinoblastoma | 3 |
| Neuroblastoma | 3 |
| Acute Myeloid Leukemia | 3 |
| Ganglioneuroblastoma, Nodular | 2 |
| Refractory Metastatic Colorectal Cancer | 1 |
| Newly Diagnosed Multiple Myeloma (NDMM) | 1 |
| Pancreatic Adenocarcinoma Metastatic | 1 |
| BRCA1 Mutation | 1 |
| BRCA2 Mutation | 1 |
| Pancreatic Acinar Cell Carcinoma | 1 |
| Chemotherapy Effect | 1 |
| Aplastic Anemia Idiopathic | 1 |
| Retinoblastoma, Extraocular | 1 |
| MDS | 1 |
| Primary Plasma Cell Leukemia | 1 |
| Extramedullary Multiple Myeloma | 1 |
| Toxicities | 1 |
| Survivorship | 1 |
| AML - Acute Myeloid Leukemia | 1 |
| MDS (Myelodysplastic Syndrome) | 1 |
| High-Risk Neuroblastoma | 1 |
| Patient With Insufficient Response Chemoimmunotherapy | 1 |
| Children | 1 |
| Retinal Neoplasm | 1 |
| Smoldering Multiple Myeloma | 1 |
| Newly Diagnosed Multiple Myeloma | 1 |
| Acute Myeloid Leukemia in Remission | 1 |
| Myelodysplastic Syndromes | 1 |
| Chronic Myeloid Leukemia in Remission | 1 |
| Myeloproliferative Syndrome | 1 |
| Myeloma Multiple | 1 |
| Sickle Cell Disease | 1 |
| Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm | 1 |
| Acute Myeloid Leukemia Post Cytotoxic Therapy | 1 |
| Acute Myeloid Leukemia, Myelodysplasia-Related | 1 |
| Bilateral Retinoblastoma | 1 |
| Childhood Intraocular Retinoblastoma | 1 |
| Group D Retinoblastoma | 1 |
| Stage I Retinoblastoma | 1 |
| Acute Lymphoblastic Leukemia | 1 |
| Mixed Phenotype Acute Leukemia | 1 |
| Myelodysplastic Syndrome | 1 |
| Uveal Melanoma | 1 |
| DS Stage I Multiple Myeloma | 1 |
| DS Stage II Multiple Myeloma | 1 |
| DS Stage III Multiple Myeloma | 1 |
| Refractory Multiple Myeloma | 1 |
| AL Amyloidosis | 1 |
| Ganglioneuroblastoma | 1 |
Sponsors
lead sponsor per trial+ 9 more sponsors in the trial list above.
By country register
A trial with sites in more than one country appears in each of those registers. CTIS does not publish a recruiting flag; its trials may also appear in the registers above.
Related compounds
Questions about Melphalan trials
How many clinical trials are testing Melphalan?
32 current trials in our China, Canada, Israel, Spain, Australia, Germany, Italy, France, Mexico, USA, UK, Netherlands, Czechia, Denmark, Norway, Brazil, Egypt, Belgium, Sweden, Switzerland, Turkey, Poland, Japan, South Korea, Taiwan, Singapore and Austria registers name Melphalan (also registered as L01AA03) as a drug or biological intervention, as of the registry sync on 9 November 2026. Completed and withdrawn studies are not counted.
Are Melphalan trials recruiting patients?
Yes. 19 of the 32 trials are recruiting: 5 with sites in China, 5 with sites in Canada, 5 with sites in Israel, 3 with sites in Spain, 4 with sites in Australia, 4 with sites in Germany, 3 with sites in Italy, 3 with sites in France, 3 with sites in Mexico, 3 with sites in USA, 2 with sites in UK, 2 with sites in Netherlands, 2 with sites in Czechia, 2 with sites in Denmark, 2 with sites in Norway, 1 with sites in Brazil, 2 with sites in Egypt, 2 with sites in Belgium, 2 with sites in Sweden, 2 with sites in Switzerland, 1 with sites in Turkey, 1 with sites in Poland, 1 with sites in Japan, 1 with sites in South Korea, 1 with sites in Taiwan, 1 with sites in Singapore and 1 with sites in Austria. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Melphalan clinical trials?
National Cancer Institute (NCI) (4), Children's Oncology Group (3), Institute of Hematology & Blood Diseases Hospital, China (2) and Institut Curie (2), among 24 lead sponsors in total. The number in brackets is how many trials each organisation leads.
Is Melphalan licensed in Ireland?
Yes. The HPRA medicines list includes 3 single-substance products containing Melphalan, for example Alkeran 2 mg Film-coated Tablets (Aspen Pharma Trading Limited). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Melphalan shortage?
The registers we track list 2 entries for Melphalan: US FDA: resolved; 1 German notice. Shortages are product- and country-specific; check with a pharmacist.
Where does this information come from?
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Melphalan works or whether it is effective, and it is not medical advice.
How this page is built
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Melphalan works or whether it is effective. Each trial links to its own record so you can check it yourself.
- ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, intervention names and synonyms, phase, overall status, lead sponsor and conditions for every trial on this page
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Melphalan (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 9 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.