Melphalan for Hematologic Malignancies

Stanford University, Stanford, CA
Hematologic Malignancies+5 More ConditionsMelphalan - Drug
Eligibility
18 - 75
All Sexes
What conditions do you have?
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Study Summary

This trial found that RIC followed by a match aploidentical donor peripheral blood stem cell transplantation can be a feasible and effective treatment for older adults or those patients that are otherwise unfit for myeloablative conditioning.

Eligible Conditions
  • Advanced Hematologic Malignancies
  • Allogeneic Bone Marrow Transplant
  • Leukemia
  • Myelodysplastic Syndrome
  • Myeloproliferative Disorder
  • Acute Leukemia

Treatment Effectiveness

Study Objectives

5 Primary · 8 Secondary · Reporting Duration: 2 years

12 months
Determine the GVHD-free relapse-free survival (GRFS) post-HCT ( Arm-A)
Disease-free survival (DFS)
GVHD-relapse-free survival
Non-relapse mortality (NRM)
Overall survival
2 years
Determine the overall survival (OS) post-HCT ( Arm-B)
Year 2
The incidence and timing of primary graft failure
Year 2
Donor CD3 chimerism at Day+60 post-HCT
Year 2
Incidence of Grade III-IV acute GVHD
Day 100
Acute GVHD (all grades)
Incidence of serious infections (grade 2 and greater)
Secondary graft failure
Time to Neutrophil engraftment
Time to Platelet engraftment
Treatment-emergent adverse events (TEAs)
Day 5
Steroid-refractory acute GVHD

Trial Safety

Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.
Approved for 20 Other Conditions
This treatment demonstrated efficacy for 20 other conditions.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

2 Treatment Groups

Arm A: Matched related/matched unrelated donor transplantation
1 of 2
Arm B: Haploidentical transplantation
1 of 2

Experimental Treatment

24 Total Participants · 2 Treatment Groups

Primary Treatment: Melphalan · No Placebo Group · Phase 1

Arm A: Matched related/matched unrelated donor transplantationExperimental Group · 8 Interventions: Purified regulatory T-cells (Treg) plus CD34+ HSPC, Fludarabine, Melphalan, CliniMACS CD34 Reagent System, Tacrolimus, Cyclophosphamide, Plerixafor, Filgrastim granulocyte colony-stimulating factor (G-CSF) or equivalent · Intervention Types: Drug, Drug, Drug, Device, Drug, Drug, Drug, Drug
Arm B: Haploidentical transplantationExperimental Group · 8 Interventions: Purified regulatory T-cells (Treg) plus CD34+ HSPC, Fludarabine, Melphalan, CliniMACS CD34 Reagent System, Tacrolimus, Cyclophosphamide, Plerixafor, Filgrastim granulocyte colony-stimulating factor (G-CSF) or equivalent · Intervention Types: Drug, Drug, Drug, Device, Drug, Drug, Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Fludarabine
FDA approved
Melphalan
FDA approved
Tacrolimus
FDA approved
Cyclophosphamide
FDA approved
Plerixafor
FDA approved

Trial Logistics

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 2 years

Who is running the clinical trial?

Stanford UniversityLead Sponsor
2,220 Previous Clinical Trials
34,902,580 Total Patients Enrolled
Orca Biosystems, Inc.Industry Sponsor
5 Previous Clinical Trials
673 Total Patients Enrolled
Everett Meyer, MD,PhDPrincipal InvestigatorStanford Universiy

Eligibility Criteria

Age 18 - 75 · All Participants · 0 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:

Frequently Asked Questions

Are there other peer-reviewed trials that utilize Filgrastim granulocyte colony-stimulating factor (G-CSF)?

"At this moment, there are 1140 Filgrastim granulocyte colony-stimulating factor (G-CSF) or equivalent clinical trials underway with 206 in Phase 3. Many of these medical studies are based in Philadelphia, Pennsylvania, but there are 31564 locations running trials for Filgrastim granulocyte colony-stimulating factor (G-CSF) or equivalent." - Anonymous Online Contributor

Unverified Answer

Does this research project have age restrictions?

"This study requires that participants are over 18 and younger than 75." - Anonymous Online Contributor

Unverified Answer

Has the FDA given their thumbs up to Filgrastim granulocyte colony-stimulating factor (G-CSF)?

"Given that Phase 1 trials have limited data to support safety and efficacy, our team rates Filgrastim granulocyte colony-stimulating factor (G-CSF) or equivalent as a 1." - Anonymous Online Contributor

Unverified Answer

Why is Filgrastim granulocyte colony-stimulating factor (G-CSF) or equivalent a common medication?

"Filgrastim granulocyte colony-stimulating factor (G-CSF) is an effective treatment for dermatitis, atopic, multiple sclerosis, and leukemia, myelocytic, acute." - Anonymous Online Contributor

Unverified Answer

What are the benefits that researchers hope to achieve with this study?

"The primary outcome of this clinical trial, which will be measured over the course of baseline, day +30, 60, 90, 180, year 1 and year 2 is to The incidence and timing of primary graft failure. Secondary objectives include Steroid-refractory acute GVHD which is defined as Steroid refractory acute GVHD will be defined as per the EBMT-NIH-CIBMTR Task Force position statement, Acute GVHD (all grades) which is defined as Acute GVHD (all grades) will be reported, and Non-relapse mortality (NRM)" - Anonymous Online Contributor

Unverified Answer

Are researchers currently looking for participants in this trial?

"The official website for this clinical trial shows that it is still recruiting patients. This trial was first announced on September 7th, 2021 and was edited October 19th, 2022." - Anonymous Online Contributor

Unverified Answer

Could I be a part of this research project if I qualify?

"This study is seeking 24 participants aged 18-75 who have myelodysplastic syndromes. Eligible participants must also meet the following criteria: histopathologically-confirmed acute myeloid, lymphoid, or mixed phenotype leukemia in complete remission (CR) or CR with incomplete hematologic recovery (CRi) or beyond first complete remission (CR1) without the presence of minimal residual disease; not in morphologic CR with bone marrow infiltration by leukemic blasts of ≤10%; or in morphologic CR with evidence of minimal residual disease positivity by either multiparametric flow cytometric analysis or by" - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.