Melphalan Hydrochloride

Phase 3Small molecule

SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Melphalan Hydrochloride is one of 48 small molecule programs targeting Ganglioneuroblastoma. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

GanglioneuroblastomaRecruiting

Graft Versus Host DiseaseActive, not recruiting

GanglioneuroblastomaPhase 3

Recruiting

Graft Versus Host DiseasePhase 2

Active, not recruiting

Competition

Competitive Landscape

42 competitors in Graft Versus Host Disease

#DrugCompanyPhaseTrials

Trexall

Jazz Pharmaceuticals

Approved

15 2

Neoral

Novartis

Approved

8 3

Medrol

Pfizer

Approved

4 4

Selzentry

GSK

Approved

1 5

Defitelio

Nipent

Olumiant

Gleevec

Qvar

3 10

Cellcept

Roche

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2027

Phase 1/2Active, not recruiting

Graft Versus Host Disease

NCT03192397

Q3 2030

Phase 3Recruiting

Ganglioneuroblastoma

NCT03126916

Clinical

Trials by Indication[1]

Graft Versus Host Disease2 trials

High Dose Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil in Preventing Graft Versus Host Disease in Patients With Hematological Malignancies Undergoing Myeloablative or Reduced Intensity Donor Stem Cell Transplant

Completed·—·Phase 2·NCT03128359·38 enrolled

Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant

Active, not recruiting·RRoswell Park Cancer Institute·Phase 1/2·NCT03192397·35 enrolled

Myeloid Neoplasm1 trial

Early Allogeneic Hematopoietic Cell Transplantation in Treating Patients With Relapsed or Refractory High-Grade Myeloid Neoplasms

Completed·UUniversity of Washington·Phase 2·NCT02756572·30 enrolled

Completed·MM.D. Anderson Cancer Center·Phase 1/2·NCT03417284·62 enrolled

2 more indications

Ganglioneuroblastoma1 trial

A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or ALK Inhibitor Therapy Added to Intensive Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma (NBL)

Recruiting·CChildren's Oncology Group·Phase 3·NCT03126916·750 enrolled

Unspecified1 trial

Study of Personalized Melphalan Dosing in the Setting of Autologous Transplant

Withdrawn·OOhio State University Comprehensive Cancer Center·Phase 2·NCT03328936

Sources

References

[1]
ClinicalTrials.gov — 8 trial records for Melphalan Hydrochloride. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Melphalan Hydrochloride compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Melphalan Hydrochloride. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Melphalan Hydrochloride

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q2 2027

Q3 2030

Trials by Indication[1]

Graft Versus Host Disease2 trials

Myeloid Neoplasm1 trial

Diffuse Intrinsic Pontine Glioma1 trial

Retinoblastoma1 trial

Multiple Myeloma1 trial

Ganglioneuroblastoma1 trial

Unspecified1 trial

References

Drug Info

Quick Facts

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