Ruxolitinib in Combination With Autotransplant

NCT02469974 | Withdrawn DMC

• 1 other identifier: GCO 14-2106
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Myelofibrosis; MF

Keywords

Myelofibrosis; RUXOLITINIB; Autologous stem cell transplant

Outcome Measures

Primary Outcomes (1)

• Safety of combining ruxolitinib with autologous HSCT measured by graft failure or death

  Safety of this approach as measured by graft failure or death

  2 years

Secondary Outcomes (11)

• CD34 cells

  4 years

• The regimen related mortality (RRM)

  day 100

• The regimen related mortality (RRM)

  day 365

• Rate of engraftment/graft failure

  4 years

• Time of engraftment for neutrophils and platelets

  4 years

Study Arms (1)

Ruxolitinib / INC 424

EXPERIMENTAL

Ruxolitinib, Jakafi ®, will be given orally at standard dose daily for 16 weeks pre ASCT and up to 3 months post-ASCT for 10 patients (allowing for 2 additional screen failures). Patients will restart ruxolitinib at 100 days after the ASCT as long as their platelet count is at least 50 x103. For patients whose platelet count is below 50 x103 at day 100, ruxolitinib should be restarted once platelet count reaches 50 x103. The dose of ruxolitinib can be titrated up as per clinical guidelines. PBSC mobilization will include G-CSF 10 mcg/kg/day. HDC for ASCT will consist of IV busulfan 2.0 mg/KBW once daily x 4 for days -5 to -2.

Drug: RUXOLITINIB / INC 424; Drug: Filgrastim; Drug: Busulfan

Interventions

RUXOLITINIB / INC 424 DRUG

Administered orally 5-20 mg twice daily x 16 weeks of therapy prior to attempted peripheral blood stem cells (PBSC) collection, during the collection and rest period and 3 months of therapy after high dose chemotherapy (HDC).

Also known as: RUX, Jakafi®

Ruxolitinib / INC 424

Filgrastim DRUG

Peripheral blood stem cells (PBSC) will be mobilized with filgrastim 10 mcg/kg/day IV

Also known as: Neupogen®

Ruxolitinib / INC 424

Busulfan DRUG

Conditioning for autologous Hematopoeitic Stem Cell Transplantation (HSCT) will consist of IV busulfan 2.0 mg/KBW once daily x 4 for days -5 to -2

Also known as: Busulfex®

Ruxolitinib / INC 424

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Red cell transfusion dependency
• unfavorable Karyotype
• platelet count \<100 x 109/L
• symptomatic splenomegaly
• PB blasts \> 1%
• Blasts in PB \<20% prior to study enrollment
• 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
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Marina Kremyanskaya: lead
• Incyte Corporation: collaborator

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

ruxolitinib; Filgrastim; Busulfan

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds

Study Officials

• Marina Kremyanskaya, MD, PhD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: May 28, 2015
• First Posted: June 12, 2015
• Study Start: May 1, 2015
• Primary Completion: August 1, 2016
• Study Completion: August 1, 2016
• Last Updated: August 12, 2016

Record last verified: 2016-08