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)

NCT00629798 | Completed Phase 2 Results

• 1 other identifier: 08-008
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 1

Brief Summary

This study will see if the researchers can lower that risk by giving the patient Palifermin. This drug helps protect the lining of the mouth, throat, and stomach. These areas typically get sores or ulcers while the blood cell counts are very low. The patient can get infections in or from these sores. Palifermin might also help the immune system recover faster. It is currently approved for patients who receive their own stem cells. That is called an autologous transplant. This study will test the use of Palifermin for T-cell depleted allogeneic stem cell transplants.

Conditions

Acute Myeloid Leukemia; Advanced Myelodysplastic Syndromes

Keywords

Busulfan; Melphalan; Fludarabine; 08-008

Outcome Measures

Primary Outcomes (1)

• To Reduce the Early Transplant-related Mortality.

  conclusion of study

Secondary Outcomes (2)

• To Improve the Quality of Immune Reconstitution Following Transplantation.

  conclusion of study

• To Reduce the Incidence Rate of Fatal Post Transplant Infectious Complications.

  conclusion of study

Study Arms (1)

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EXPERIMENTAL

This is a single arm phase II trial to assess the efficacy (decrease the transplant related mortality) and safety of peri-transplant Palifermin in combination with a preparative regimen with busulfan, melphalan, fludarabine, and anti-thymocyte globulin (ATG), and a T cell depleted stem cell transplant from a histocompatible related or unrelated donor in patients with advanced MDS and AML evolved from MDS. The addition of Palifermin is to decrease the toxicity and the infection rate associated with this regimen and transplant type and to foster earlier immune reconstitution.

Drug: Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant

Interventions

Busulfan, Melphalan, Fludarabine, Anti-Thymocyte Globulin, Palifermin, Stem cell transplant DRUG

Patients will receive Palifermin 60 mcg/kg/day IV on three consecutive days with the last dose administered no less than 24 and no more than 48 hr prior to start of cytoreduction. The preparative regimen to be used for transplants will consist: of busulfan administered in 12 doses over three days of 0.8 mg/kg IV for patients \> or = to 4 years of age or 1.0 mg/kg IV for patients \< 4 years of age; melphalan 70 mg/m2 IV x 2 days; and, fludarabine 25 mg/m2 IV x 5 days

Also known as: Patients will receive ATG for two doses prior to transplant., G-CSF mobilized CD34+E- PBSCs (or BM if donors are unwilling or unable, to donate PBSC) obtained from the HLA compatible donor will be infused on, day 0. Patients will receive three additional daily doses of Palifermin,, the first approximately 6 hours after the stem cell infusion on day 0,, followed by two daily doses given on d+1 and d+2. Supportive care will be administered as per the BMT Service, guidelines.

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Eligibility Criteria

• Age: Up to 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients should be \< 65 years. Patients \> or equal to 65 years will be accrued on a case by case basis after discussion and approval by the BMT Service.
• Patients may be of either gender or any ethnic background.
• Patients must have a Karnofsky or Lansky Performance Status \> or equal to 70%.
• Patients must have adequate organ function measured by:
• \* Cardiac: asymptomatic or if symptomatic then LVEF at rest must be \> or equal to 50% and must improve with exercise.
• Hepatic: \< 3x ULN ALT and \< 1.5 total serum bilirubin, unless there is congenital benign hyperbilirubinemia.
• Renal: serum creatinine \< than or equal to 1.2 mg/dl or if serum creatinine is outside the normal range, then CrCl \> 60-ml/min/1.73 m2
• Pulmonary: asymptomatic or if symptomatic, DLCO \> 50% of predicted (corrected for hemoglobin)
• Each patient must be willing to participate as a research subject and must sign an informed consent form.
• Parent or legal guardians of patients who are minors will sign the informed consent form.

You may not qualify if:

• Active CNS or skin leukemic involvement
• Female patients who are pregnant or breast-feeding
• Active viral, bacterial or fungal infection
• Patient seropositive for HIV-I/II; HTLV -I/II
• Patients who have undergone a prior allogeneic or autologous stem cell transplant within the previous six months.
• Patients who have had a previous malignancy that is not in remission.

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead

Study Sites (1)

Memorial Sloan-Kettering Cancer Center

New York, New York, 10065, United States

Location

Related Links

• Memorial Sloan-Kettering Cancer Center

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

Busulfan; Melphalan; fludarabine; Antilymphocyte Serum; Fibroblast Growth Factor 7; Stem Cell Transplantation; Guidelines as Topic

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures; Fibroblast Growth Factors; Intercellular Signaling Peptides and Proteins; Peptides; Biological Factors; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative; Quality Assurance, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Results Point of Contact

• Title: Dr. Roni Tamari, MD
• Organization: Memorial Sloan Kettering Cancer Center

tamarir@mskcc.org

646-608-3738

Study Officials

• Roni Tamari, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 14, 2008
• First Posted: March 6, 2008
• Study Start: February 12, 2008
• Primary Completion: October 20, 2020
• Study Completion: October 20, 2020
• Last Updated: September 24, 2021
• Results First Posted: September 24, 2021

Record last verified: 2020-10

Locations

US (1)