Chemo/Radiation Therapy + Stem Cell Transplant for Myelofibrosis

Recruiting at 1 trial location

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: City of Hope Medical Center

Disqualifiers: HIV, Hepatitis B/C, Liver cirrhosis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

22 Participants Needed

Trial Summary

What is the purpose of this trial?

This pilot phase I trial studies the side effects of combination chemotherapy, total body irradiation, and donor blood stem cell transplant in treating patients with secondary myelofibrosis. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. Giving combination chemotherapy and total body irradiation before a donor blood stem cell transplant helps to stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets.

Research Team

Monzr M. Al Malki

Principal Investigator

City of Hope Medical Cancer Center

Eligibility Criteria

This trial is for patients with secondary myelofibrosis who are fit (Karnofsky performance >=70%), have adequate organ function, and no severe pulmonary or liver conditions. Donors must be a genetic half-match without certain infections or health risks, and not pregnant if female of childbearing potential.

Inclusion Criteria

DONOR: Is approved and completed evaluation prior to recipient initiation of the preparative regimen per institutional guidelines

Left ventricular ejection fraction (LVEF) >= 50%

My donor is a relative who is not my mother and matches half of my HLA markers.

Exclusion Criteria

I have severe liver issues with symptoms like bleeding, fluid buildup, or confusion.

I have had a transplant involving solid organ, stem cell, bone marrow, or blood.

I have liver cirrhosis.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Chemotherapy and Radiation

Participants receive melphalan and fludarabine chemotherapy, followed by total body irradiation before hematopoietic cell transplantation

7 days

Daily visits for chemotherapy and radiation administration

Hematopoietic Cell Transplantation

Participants undergo hematopoietic cell transplantation and receive post-transplant cyclophosphamide

5 days

Daily visits for transplantation and post-transplant medication

Post-Transplant Treatment

Participants receive tacrolimus, mycophenolate mofetil, and G-CSF to support recovery and prevent complications

6 months

Regular visits for medication administration and monitoring

Follow-up

Participants are monitored for safety, effectiveness, and long-term outcomes after treatment

Up to 2 years

Periodic visits for follow-up assessments

Treatment Details

Interventions

• Allogeneic Hematopoietic Stem Cell Transplantation
• Cyclophosphamide
• Fludarabine
• Melphalan
• Mycophenolate Mofetil
• Tacrolimus
• Total-Body Irradiation

Trial Overview The trial tests a treatment combining chemotherapy drugs (Melphalan, Mycophenolate Mofetil, Tacrolimus, Cyclophosphamide, Fludarabine), total body irradiation, and donor blood stem cell transplant to see how well it treats secondary myelofibrosis.

Participant Groups

Group I: Treatment (combination chemotherapy, TBI, HCT) Experimental Treatment 9 Interventions

Patients receive melphalan IV over 30 minutes on day -5, fludarabine IV over 30-60 minutes on days -5 to -2. Patients undergo TBI on day -1 and HCT on day 0. Patients receive cyclophosphamide IV over 1-2 hours on days 3 and 4. Starting on day 5, patients receive tacrolimus IV then PO for 6 months followed by a taper, mycophenolate mofetil PO TID until day 35, and G-CSF IV daily until absolute neutrophil count \> 1,500/mm\^3 for 3 consecutive days. Treatment continues in the absence of disease progression or unexpected toxicity.

Allogeneic Hematopoietic Stem Cell Transplantation is already approved in European Union, United States, Canada, Japan for the following indications:

Approved in European Union as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Approved in United States as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Approved in Canada as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Approved in Japan as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Who Is Running the Clinical Trial?

City of Hope Medical Center

Lead Sponsor

Trials

614

Recruited

1,924,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+