Ruxolitinib in Combination With Autotransplant

• Status: Withdrawn
• Trial end date: 2016-08-01
• Target enrollment: 0
• Participant gender: All

Summary

To determine the safety of the approach of giving RUXOLITINIB before and after an autologous stem cell transplant, as measured by graft failure or death.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Marina Kremyanskaya
• Collaborator: Incyte Corporation
• Treatments: Busulfan; Lenograstim

Criteria

Inclusion Criteria:

• Histologically documented diagnosis of MF (idiopathic or post PV/ET)

• Age 18-75 years

• Intermediate-2/ high-risk disease as per Dynamic IPSS (DIPSS) criteria or Intermediate-1 risk disease with one of the following features within one year from screening:

1. Red cell transfusion dependency

2. unfavorable Karyotype

3. platelet count <100 x 109/L

4. symptomatic splenomegaly

5. PB blasts > 1%

6. Blasts in PB <20% prior to study enrollment

7. No available suitable matched related (6/6 or 5/6) or unrelated donor (8/8 or 7/8 allele matched) or unwilling or unable to pursue allogeneic stem cell transplant

8. WBC <50,000/ml at screening

• Able to give informed written consent

• ECOG Performance status of 0-2

• Life expectancy >6 months

• Off all myelofibrosis-related investigational or standard agents (except for ruxolitinib) for at least 4 weeks prior to study enrollment and recovered from all toxicities. If patient is already on ruxolitinib for a minimum of 16 weeks prior to study enrollment, patient can proceed to mobilization and collection

• Adequate organ function defined as the following (*unless clearly disease related):

1. Adequate renal function - creatinine <2 x ULN

2. Adequate hepatic function - AST/ALT <3 x ULN, Total Bilirubin <3 x ULN, exception is elevated indirect bilirubin attributed to Gilbert's syndrome or hemolysis

3. Adequate hematopoietic function - Platelet ≥50 x 109/L (without transfusion) and ANC ≥1.0 x 109/L

4. LVEF >40% (MUGA or echocardiogram)

5. Adequate pulmonary function with DLCO >40%

Exclusion Criteria:

• Hypersensitivity to JAK inhibitor

• Clinical evidence of cirrhosis

• Leukemic transformation (>20% blasts in PB or BM any time prior to HCT)

• Platelet count <50 x 109/L

• Active uncontrolled infection

• History of another malignancy within 5-years of date of HCT except history of basal cell or squamous cell carcinoma of skin or PV or ET

• Known HIV positive

• Woman of childbearing potential unwilling or unable to use adequate contraception Pregnant or nursing females Known active infection with hepatitis A, B or C virus