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Study of Fludarabine Based Conditioning for Allogeneic Stem Cell Transplantation for Myelofibrosis


N/A
18 Years
65 Years
Open (Enrolling)
Both
Myelofibrosis

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Trial Information

Study of Fludarabine Based Conditioning for Allogeneic Stem Cell Transplantation for Myelofibrosis


This study is designed as a single arm Phase II clinical trial in patients with
myelofibrosis who are eligible for transplantation from a related donor or from an unrelated
donor source. Patients will be accrued into two separate strata defined by donor type. Each
of the two strata will be analyzed separately.

Patients will be followed yearly from time of enrollment into the study to assess clinical
response and overall, progression and event free survival, as well as incidence and degree
of acute and chronic GVHD. We will estimate cumulative survival and transplant related
mortality in patients enrolled in each of the two strata.


Inclusion Criteria:



- Patients with the following disease: Idiopathic myelofibrosis, or spent PV-, or
ET-related myelofibrosis in chronic phase (<20% blast cells in the bone marrow) with
Lille score >1 at any time, or platelet <100K.

- Age 18-65 years.

- ECOG performance status < 3.

- Life expectancy >3 months.

- Adequate cardiac function, normal LVEF ≥ 45% by MUGA or echocardiogram and adequate
pulmonary function DLCO ≥ 50% of predicted.

- Serum creatinine < 1.1 x the upper limit of normal (ULN) or Creatinine Clearance >50
ml/min.

- Serum bilirubin < 2.0 mg/dl, SGPT <2.5 x upper limit of normal

- No evidence of chronic active hepatitis or cirrhosis

- HIV-negative

- Patient is not pregnant

- Patient or guardian able to sign informed consent.

- Patients with >20% myeloblasts in the blood or marrow, extramedullary blast cell
proliferation or large foci of blasts in bone marrow biopsy specimens are not
eligible.

- Pretransplant splenectomy: MMM patients with variable degrees of splenomegaly, or
splenectomized, are eligible to be enrolled. Any decision of having a patient
splenectomized prior to transplant will be made in each center prior to enrolling the
patient in the study.

- Patients should be off treatment with investigational for at least 4 weeks and have
recovered from all toxicities.

Exclusion Criteria:

- Pregnancy

- HIV positive

- > 20% myeloblasts in the peripheral blood or bone marrow

- LVEF < 45%

- DLCO < 50% of predicted

- ECOG performance status ≥ 3

- Chronic active hepatitis or cirrhosis

- Chronic renal insufficiency

Type of Study:

Interventional

Study Design:

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

The primary endpoint is progression-free survival.

Outcome Time Frame:

2 years

Safety Issue:

No

Principal Investigator

John Mascarenhas, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Icahn School of Medicine at Mount Sinai

Authority:

United States: Institutional Review Board

Study ID:

GCO 07-0548-00101

NCT ID:

NCT00572897

Start Date:

August 2007

Completion Date:

August 2020

Related Keywords:

  • Myelofibrosis
  • Myelofibrosis
  • Myeloproliferative disease
  • Stem Cell Transplant
  • Allogeneic Transplant
  • Conditioning Regimen
  • Reduced Intensity Regimen
  • Fludarabine
  • Melphalan
  • ATG
  • Primary Myelofibrosis

Name

Location
Roswell Park Cancer Institute Buffalo, New York  14263
University of Illinois at Chicago Chicago, Illinois  60612
University of Utah Salt Lake City, Utah  
Johns Hopkins Baltimore, Maryland  21231
Weill Cornell Medical College New York, New York  10021
Ohio State Univesity Columbus, Ohio  43210
Icahn School of Medicine at Mount Sinai New York, New York  10029