Elotuzumab for the Treatment of JAK2-Mutated Myelofibrosis

NCT04517851 | Active Not Recruiting Phase 2 FDA Drug

• 2 other identifiers: 2020-0522NCI-2020-05712
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

This phase II trial investigates how well elotuzumab works in treating patients with JAK2-mutated myelofibrosis. Elotuzumab may help to control myelofibrosis and/or help to improve blood cell count and bone marrow function.

Conditions

Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase; Myelofibrosis Transformation in Essential Thrombocythemia; Primary Myelofibrosis

Outcome Measures

Primary Outcomes (1)

• Overall response (OR)

  OR is defined as CR (complete response) +PR (partial response) + CI (clinical improvement), where CI includes clinical improvements in anemia, splenomegaly and/or symptoms. Will estimate the OR rate, along with the exact 95% confidence interval.

  Up to completion of cycle 36 (1 cycle is 28 days)

Secondary Outcomes (8)

• Incidence of adverse events

  Up to 30 days post-treatment

• Duration of response

  Up to 5 years

• Time to next treatment

  Up to 5 years

• Rates of complete response

  Up to 5 years

• Rates of partial response

  Up to 5 years

Other Outcomes (5)

• Biomarker analysis

  Up to 5 years

• Percentage of circulating SLAMF7high/CD16neg monocytes

  Baseline and over time up to 5 years

• Serum IL-1Ralpha levels

  Baseline and over time up to 5 years

Study Arms (1)

Treatment (elotuzumab)

EXPERIMENTAL

Patients receive elotuzumab IV over 1-4 hours on days 1, 8, 15, and 22 of cycles 1-2. Beginning in cycle 3, patients receive elotuzumab IV over 1-4 hours on day 1. Treatment repeats every 28 days for up to 36 cycles in the absence of disease progression or unacceptable toxicity.

Biological: Elotuzumab; Other: Questionnaire Administration

Interventions

Elotuzumab BIOLOGICAL

Given IV

Also known as: BMS-901608, Empliciti, HuLuc-63, HuLuc63, PDL-063, PDL063

Treatment (elotuzumab)

Questionnaire Administration OTHER

Ancillary studies

Treatment (elotuzumab)

Eligibility Criteria

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

You may qualify if:

• Adults with JAK2 V617F+ primary myelofibrosis (PMF) or post-polycythemia vera (PV)/essential thrombocythemia myelofibrosis (ET-MF) who require treatment and have intermediate or higher risk disease (as assessed by the International Prognostic Scoring System for Myelodysplastic Syndrome \[IPSS\], Dynamic International Prognostic Scoring System \[DIPSS\], DIPSS-plus, Mutation-Enhanced Prognostic System for Transplant Age Patients with Primary Myelofibrosis \[MIPSS70\], MIPSS70-plus version \[v\] 2.0, or MYelofibrosis SECondary to PV and ET-Prognostic Model \[MYSEC-PM\]). The MYSEC-PM is to only be used for patients with post-PV/ET MF
• Patients must not be candidates for JAK inhibitor therapy in the opinion of the treating physician
• Bone marrow (BM) fibrosis grade 2 or 3 according to the European classification
• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2 (Karnofsky performance status \>= 60%)
• Absolute neutrophil count \>= 0.5 x 10\^9/L
• Direct bilirubin =\< 1.5 x institutional upper limit of normal
• Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 2.5 x institutional upper limit of normal unless felt to be due to liver involvement by MF/extramedullary hematopoiesis, in which case =\< 5 x institutional upper limit of normal is permissible
• Creatinine =\< 2 x institutional upper limit of normal OR creatinine clearance \>= 30 mL/min/1.73 m\^2 for patients with creatinine levels above institutional normal
• Ability to understand and the willingness to sign a written informed consent document
• Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation until 6 months after the last administration of elotuzumab. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Women of child-bearing potential must have a negative pregnancy test. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 6 months after the last administration of elotuzumab

You may not qualify if:

• Splenic irradiation within the preceding 4 months
• Chemotherapy (other than hydroxyurea), interferons, IMiDs, danazol or other androgens, erythroid stimulating agents, or other MF-directed commercially available agents within 4 weeks prior to entering the study or those who have not recovered to baseline from adverse events due to agents administered more than 4 weeks earlier
• Other investigational agents within 4 half-lives prior to study entry
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to elotuzumab
• Patients with known central nervous system (CNS) involvement
• Prior allogeneic hematopoietic cell transplantation (allo-HCT) for MF
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
• Known pregnancy or lactation
• Known human immunodeficiency virus (HIV), hepatitis B virus (HBV) or hepatitis C virus (HCV) positivity

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• MD Anderson Cancer Center Website

MeSH Terms

Conditions

Polycythemia Vera; Primary Myelofibrosis

Interventions

elotuzumab

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Myeloproliferative Disorders

Study Officials

• Prithviraj Bose

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

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: August 14, 2020
• First Posted: August 18, 2020
• Study Start: February 10, 2021
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: January 13, 2026

Record last verified: 2026-01

Locations

US (1)