Phase 2
N=66
Study of Fludarabine Based Conditioning for Allogeneic Stem Cell Transplantation for Myelofibrosis
Myelofibrosis
Bottom Line
View on ClinicalTrials.gov: NCT00572897 ↗Enrolled (actual)
66
Serious AEs
47.0%
Results posted
May 2016
Primary outcome: Primary: The Primary Endpoint is Progression-free Survival. — 24; 11 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Fludarabine, Melphalan +/- ATG (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- John Mascarenhas
- Primary completion
- Aug 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Primary Endpoint is Progression-free Survival. |
24; 11 | — |
| SECONDARY Response Outcomes |
7; 6; 8; 1; 11; 5 | — |
| SECONDARY Overall Survival |
25; 11 | — |
| SECONDARY Absolute Neutrophil Count (ANC) |
31; 26; 1; 8 | — |
| SECONDARY PLT |
28; 20; 4; 14 | — |
| SECONDARY Transplant-related Mortality |
3; 20; 29; 14 | — |
Summary
Stem cell transplantation is used to treat may types of diseases. There a 2 types of transplants, conventional (very intense) and reduced intensity-non-myeloablative, also called mini-transplants.
This study proposes to use a conditioning regimen for allogeneic transplantation along with a reduced intensity transplant. Conditioning regiment is the name for the combination of chemotherapy drugs that is given to patients before receiving a transplantation of donor stem cells. It is hoped that the regimen designed for this study proves to be less toxic and has an equal or better anticancer effect than the regimens that are normally used. The regimen being used is a combination of two chemotherapy drugs, fludarabine and melphalan. This regimen has been studied in recipients of matched sibling transplants and in recipients of alternative donor stem cells in other hematologic malignancies. Those subjects, who receive stem cells from an unrelated donor, will also receive and additional drug called ATG or anti thymocyte globulin. ATG suppresses the immune system, thus reducing the chances for the recipient rejecting the transplant (graft).
The purpose of this study is to observe if reduced intensity transplants can be used to allow engraftment or "take" of the donor's bone marrow. Studies conducted in the past show this type of transplant is much less toxic than traditional bone marrow transplants. Reduced intensity transplants may be better tolerated by patients who may experience serious side effects from standard (very intense) stem cell transplant.
The study has been recently amended to follow all subjects for survival.
Eligibility Criteria
Inclusion Criteria
- Patients with the following disease: Idiopathic myelofibrosis, or spent PV-, or ET-related myelofibrosis in chronic phase ( 1 at any time, or platelet 3 months.
- Adequate cardiac function, normal LVEF ≥ 45% by MUGA or echocardiogram and adequate pulmonary function DLCO ≥ 50% of predicted.
- Serum creatinine 50 ml/min.
- Serum bilirubin 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
Data sourced from ClinicalTrials.gov (NCT00572897). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.