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Floatz Rating
B63/100
Confidence
Indicative
v0.2
Sponsor
City of Hope Medical Center
Modality
Small molecule
Development Phase
Phase 4 / Approved
Status
Active
Evidence ledger · v0.2

Clinical Evidence

Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
66High confidence
Detailed axis rationale is planned and will be published soon.
TrialPhaseStatusNPrimary endpointReadout
Total Marrow and Lymphoid Irradiation in Combination With Fludarabine and Melphalan as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplant in Older Patients With Refractory and Relapsed Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome
Not Yet Recruiting
Efficacy and Safety of the CloB2M (Clofarabine Combined With Busulfan and Melphalan) Conditioning Regimen in Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients With Acute Myeloid Leukemia in First Complete Remission
Not Yet Recruiting
Venetoclax or Placebo in Combination With Reduced-Intensity Conditioning Hematopoietic Cell (Bone Marrow/Blood Stem Cell) Transplant and as Maintenance Therapy After Transplant in Patients With Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)
Not Yet Recruiting
CD34+ Transplants for Leukemia and Lymphoma
Not Yet Recruiting
Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant
Recruiting
Fludarabine Plus Melphalan Versus Addition of Venetoclax to Fludarabine/Melphalan Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in AML/MDS Patients Aged > 50 Years: a Multicenter, Randomized, Phase 3 Trial
Recruiting
Haplo-Cord HSCT for AML/MDS
Recruiting
Evaluation of Treosulfan Versus Melphalan Conditioning Followed by PTCy in Patients With AML and MDS Undergoing Allogeneic Transplantation
Not Yet Recruiting
A Platform Protocol to Investigate Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis in Patients With Hematologic Malignancies Undergoing Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation
Recruiting
225Ac-DOTA-Anti-CD38 Daratumumab Monoclonal Antibody With Fludarabine, Melphalan and Total Marrow and Lymphoid Irradiation as Conditioning Treatment for Donor Stem Cell Transplant in Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome
Recruiting
Show 65 more trials
Vedolizumab Plus Post-transplant Cyclophosphamide and Short Course Tacrolimus for the Prevention of Graft Versus Host Disease in Patients Undergoing Allogeneic Hematopoietic Cell Transplantation After Reduced Intensity Conditioning
Recruiting
Pilot Study of Memory-like Natural Killer (ML NK) Cells After TCRαβ T Cell Depleted Haploidentical Transplant in AML
Recruiting
TMLI/Fludarabine/Melphalan Conditioning for Allogeneic Transplantation in High-risk Myelodysplastic Syndrome or Acute Myeloid Leukemia.
Not Yet Recruiting
Twice-per-weekSelinexor, 2 Days Melphalan
Not Yet Recruiting
RIC Allo-HSCT vs. Venetoclax-Based Consolidation in Elderly AML Patients After First CR
Recruiting
MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
Recruiting
Orca-T Following Chemotherapy and Total Marrow and Lymphoid Irradiation for the Treatment of Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia or Myelodysplastic Syndrome
Recruiting
HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis
Active Not Recruiting
Conditioning Regimen Containing Melphalan and Cladribine for Refractory / Relapsed AML
Not Yet Recruiting
Randomized Study of Conditioning of Fludarabine Combined With Single or Dual Alkylating Agents in Myeloid Malignancies
Recruiting
MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies
Recruiting
Mismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome
Active Not Recruiting
CD34+ Enriched Transplants From HLA-Compatible Patients With Hematologic Malignancies
Withdrawn
90Y-DOTA-anti-CD25 Basiliximab, Fludarabine, Melphalan, and Total Marrow and Lymphoid Irradiation for the Treatment of High-Risk Acute Leukemia or Myelodysplastic Syndrome
Active Not Recruiting
Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia
Withdrawn
Studying Conditioning Regimen In Pediatric Transplantation - AML , SCRIPT-AML
Recruiting
Off-the-shelf NK Cells + SCT for Myeloid Malignancies
Completed
HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide
Active Not Recruiting
A Modified Dose of Rabbit Anti-thymocyte Globulin (rATG) in Children and Adults Receiving Treatment to Help Prepare Their Bodies for a Bone Marrow Transplant
Active Not Recruiting
IS-free Treg HaploHCT
Recruiting
Aza-Ven Followed by Reduced Toxicity Conditioning Regimen (MBF) as Salvage Therapy for Refractory AML.
Unknown
Haplo Peripheral Blood Sct In GVHD Prevention
Completed
TCRαβ-depleted Progenitor Cell Graft With Additional Memory T-cell DLI, Plus Selected Use of Blinatumomab, in Naive T-cell Depleted Haploidentical Donor Hematopoietc Cell Transplantation for Hematologic Malignancies
Active Not Recruiting
Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy
Active Not Recruiting
Donor Stem Cell Transplantation Using α/β+ T-lymphocyte Depleted Grafts From HLA Mismatched Donors
Terminated
Umbilical Cord Blood Transplant With Added Sugar and Chemotherapy and Radiation Therapy in Treating Patients With Leukemia or Lymphoma
Completed
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
Lymphodepletion and Anti-PD-1 Blockade to Reduce Relapse in AML Patient Not Eligible for Transplant
Completed
Early Allogeneic Hematopoietic Cell Transplantation in Treating Patients With Relapsed or Refractory High-Grade Myeloid Neoplasms
Completed
Dose-Escalation Study of Ponatinib, a FLT3 Inhibitor, With and Without Combination of 5-Azacytidine, in Patients With FLT3-Mutated Acute Myeloid Leukemia (AML)
Withdrawn
Provision of TCRγδ T Cells and Memory T Cells Plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies Relapsed or Refractory Despite Prior Transplantation
Active Not Recruiting
Azacitidine in Haploidentical Donor Hematopoietic Cell Transplantation
Terminated
Personalized NK Cell Therapy in CBT
Recruiting
A Study of Vadastuximab Talirine Given Prior to or After Allogeneic Hematopoietic Stem Cell Transplant in AML Patients
Terminated
Ruxolitinib Phosphate, Tacrolimus and Sirolimus in Preventing Acute Graft-versus-Host Disease During Reduced Intensity Donor Hematopoietic Cell Transplant in Patients With Myelofibrosis
Withdrawn
Repeat Transplantation for Relapsed or Refractory Hematologic Malignancies Following Prior Transplantation
Terminated
Natural Killer Cells Before and After Donor Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelogenous Leukemia
Completed
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Active Not Recruiting
Chemo Sensitization Before Hematopoietic Stem Cell Transplantation in Patients With Acute Leukemia in Complete Remission
Unknown
Consolidation Therapy for Acute Myeloid Leukemia Guided by Leukemia Stem Cell Behavior
Terminated
NK Cells in Cord Blood Transplantation
Unknown
CPX-351 Salvage Therapy Followed by Haplo-Cord Transplant for Relapsed/Refractory Leukemia or Myelodysplastic Syndrome
Completed
Randomized HaploCord Blood Transplantation vs. Double Umbilical Cord Blood Transplantation for Hematologic Malignancies
Terminated
A Two-Step Approach to Bone Marrow Transplant Using Cells From Two Partially-Matched Relatives
Terminated
Reduced Intensity Double Umbilical Cord Blood Transplantation
Completed
Pooled Unrelated Donor Umbilical Cord Blood Transplant For Hematologic Malignancy Needing Allogeneic Stem Cell Transplant Without Related HLA-Match
Terminated
Reduced Intensity Regimen vs Myeloablative Regimen for Myeloid Leukemia or Myelodysplastic Syndrome (BMT CTN 0901)
Terminated
HLA-Compatible Related or Unrelated Donors With CD34+ Enriched, T-cell Depleted Peripheral Blood Stem Cells Isolated by the CliniMACS System in the Treatment of Patients With Hematologic Malignancies
Completed
A Phase Ib/IIb, Open-label, Multi-center, Study of Oral Panobinostat Administered With 5-Azacitidine (in Adult Patients With Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML).
Completed
Haploidentical Stem Cell Transplantation for Children With Therapy Resistant Leukemia
Completed
Pilot Study of Unrelated Cord Blood Transplantation
Terminated
Hematopoietic Stem Cell Transplantation (HSCT) Using CD34 Selected Mismatched Related Donor and One Umbilical Cord Unit
Terminated
Haploidentical Allogeneic Transplant With Post-transplant Infusion of Regulatory T-cells
Terminated
Haploidentical Stem Cell Transplantation With CD3/CD19 Depletion and Reduced Intensity Conditioning in Patients With Acute Leukemia
Terminated
Busulfan, Melphalan, and Fludarabine With Peri-transplant Palifermin, Followed by a T-Cell Depleted Hematopoietic Stem Cell Transplant From HLA Matched or Mismatched Related or Unrelated Donors in Patients With Advanced Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML)
Completed
High-Dose Chemotherapy With or Without Total-Body Irradiation Followed by Autologous Stem Cell Transplant in Treating Patients With Hematologic Cancer or Solid Tumors
Completed
Azacytidine With Valproic Acid Versus Ara-C in Acute Myeloid Leukemia (AML)/ Myelodysplastic Syndrome (MDS) Patients
Completed
Low Dose Melphalan and Bortezomib for AML and High-Risk MDS
Completed
Randomized Double Cord Blood Transplant Study
Completed
Early Allogeneic Blood Stem Cell Transplantation in High-risk Acute Myeloid Leukemia (AML)
Completed
Treatment of Children and Adolescents With Refractory or Relapsed Acute Myeloid Leukemia
Completed
T-Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Hematologic Malignancies
Completed
Allo Transplantation With Mylotarg, Fludarabine and Melphalan for AML, CML and MDS
Completed
Stem Cell Transplantation for Hematological Malignancies
Terminated
Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies
Completed

Competitive Position

Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
59High confidence
Detailed axis rationale is planned and will be published soon.

Same indication · Acute Myeloid Leukemia

AssetSponsorPhaseRating
Melphalan (this asset)City of Hope Medical CenterApprovedB · 63
GemtuzumabM.D. Anderson Cancer CenterP4A
AmsacrineUniversity Hospital, ToulouseP4BBB
PosaconazoleAstraZenecaP4BBB
(+)-VincristineJanssen Research & Development, LLCP3BBB
AclacinomycinChinese PLA General HospitalP4BB
AlvocidibM.D. Anderson Cancer CenterP2BB
PrednisoneChildren's Oncology GroupP3BB
Therapeutic Allogeneic LymphocytesUniversity of Southern CaliforniaP2BB

+42 more in the Acute Myeloid Leukemia cohort

Other indications for Melphalan

IndicationSponsorPhaseRating
Diffuse Large B-Cell LymphomaP4BBB · 76
Myelodysplastic/Myeloproliferative NeoplasmP2/3BBB · 76
Cancer Of Unknown Primary SiteP2/3BBB · 72

Scientific Foundation

Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR

Planned for methodology v0.2.

Development Feasibility

How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR

Planned for methodology v0.2.

Commercial Opportunity

Commercial prize: addressable population, unmet need, and the value case for the indication.
NR

Planned for methodology v0.2.

IP & Exclusivity

Exclusivity position, covering patent protection and freedom-to-operate runway.
NR

Planned for methodology v0.2.

Manufacturing & Supply

Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR

Planned for methodology v0.2.

Related assets

Citation

Floatz Terminal. Melphalan in Acute Myeloid Leukemia. Methodology v0.2.
Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026.
Accessed July 14, 2026.
https://terminal.floatz.ai/assets/melphalan-acute-myeloid-leukemia

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