Study of Canakinumab in Patients With Myelofibrosis

NCT05467800 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: GCO 22-0543MPN-RC 122
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 7

Brief Summary

This is an open label, multicenter, phase 2 trial of Canakinumab in patients with primary myelofibrosis (PMF), post essential thrombocythemia/polycythemia vera related MF (Post ET/PV MF). Eligible patients will receive Canakinumab administered as a subcutaneous injection on day 1 of a 21 day cycle for a core study period of 8 cycles. Canakinumab will be given by subcutaneous injection (SC) injection at a starting dose of 200 mg (one 150 mg/mL syringe and one 50 mg/0.5 mL syringe) every 3 weeks. The interim analysis will be performed when the number of enrolled patients reaches 10. If no responses OR 4 or more patients have unacceptable toxicity, the study will not proceed to the second stage. If the total number of patients reaches the maximum sample size of 26, the treatment is deemed acceptable if the number of responses in the efficacy endpoint are greater than 3, and the number of toxicities are less than 7.

Conditions

ET-MF; Primary Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis; Post-polycythemia Vera Related Myelofibrosis; PV-MF

Outcome Measures

Primary Outcomes (1)

• Number of participant with response based on IWG-MRT criteria

  Efficacy of Canakinumab as measured by number of participant with response based on the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) criteria. A response is considered any one of the following: complete response, partial response, or clinical improvement (inclusive of anemia response, spleen response, or symptom response).

  24 weeks

Secondary Outcomes (10)

• Number of Adverse Events

  24 weeks

• Response using IWG-MRT

  12 weeks

• Response using IWG-MRT

  24 weeks

• Number of participants with clinical improvement

  12 weeks

• Number of participants with clinical improvement

  24 weeks

Study Arms (1)

Canakinumab

EXPERIMENTAL

Canakinumab will be given by subcutaneous injection (SC) injection at a starting dose of 200 mg (one 150 mg/mL syringe and one 50 mg/0.5 mL syringe) every 3 weeks.

Drug: Canakinumab

Interventions

Canakinumab DRUG

Canakinumab administered as a subcutaneous injection on day 1 of a 21 day cycle for a core study period of 8 cycles.

Canakinumab

Eligibility Criteria

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

You may qualify if:

• Parts 1 and 2:
• Participants must voluntarily sign informed consent form (ICF) and be willing and able to adhere to the study visit schedule and all protocol requirements.
• Participants must be ≥ 18 years of age at the time of signing the ICF.
• Participants must have a pathologically confirmed diagnosis of PMF as per the World Health Organization (WHO) diagnostic criteria46 or post-ET MF or post-PV MF according to IWG-MRT criteria.47
• Participants must have at least one of the following:
• Hemoglobin \< 10 g/dL
• Transfusion-dependency (at least 6 units of packed red blood cells (PRBC) in the 12 weeks prior to study enrollment, for a hemoglobin \< 8.5 g/dL, in the absence of bleeding or treatment-induced anemia with the most recent transfusion having occurred in the 28 days prior to study enrollment)
• Splenomegaly palpated ≥ 5 cm below the left costal margin (LCM) or spleen volume ≥ 450 cc
• MFSAF v4.0 TSS ≥ 10
• A bone marrow biopsy must be performed within the 30-day screening period; however, a bone marrow biopsy obtained within 90 days of signing ICF without intervening treatments and approved by the study chair may suffice.
• Participants must have an Eastern Cooperative Oncology Group (ECOG) Performance Status of 0-2.
• Life expectancy of at least 6 months.
• Recovery to ≤ Grade 1 or baseline of any toxicities due to prior systemic treatments excluding alopecia.
• Women of childbearing potential (WCBP) must have a negative urine or serum pregnancy test within 28 days of starting the study drug. Men and women of childbearing potential must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence, tubal ligation, vasectomy) prior to Cycle 1 Day 1 and for 130 days after stopping study treatment. Vasectomy must be performed a minimum of 3 months before study start.
• Must have adequate organ function as demonstrated by the following:

You may not qualify if:

• Prior malignancy active within the previous ≤ 1 year except for locally curable cancers that have been apparently cured, such as basal or squamous cell skin cancer, superficial bladder cancer, or carcinoma in situ of the prostate, cervix, or breast.
• Any of the following cardiac abnormalities
• Uncontrolled, symptomatic congestive heart failure as designated by the treating physician
• Myocardial infarction ≤ 6 months prior to enrollment
• Unstable angina pectoris designated by treating physician
• Serious uncontrolled cardiac arrhythmia as designated by treating physician
• Uncontrolled hypertension as designated by treating physician
• Known history of human immunodeficiency virus (HIV) (no laboratory testing is required), or active infection with hepatitis B or Hepatitis C.
• Active TB infection or documented, untreated latent TB infection (all participants should undergo TB risk evaluation prior to enrollment with TB screening performed as per local guidelines.)
• Active, uncontrolled infection at the time of enrollment, except in cases of localized infections that are unlikely to lead to a systemic infection such as onychomycoses or dental caries.
• a. Participants with a new fever (T\>38.0o C) or respiratory symptoms are required to undergo laboratory screening for COVID-19.
• Have undergone prior allo-HSCT for treatment of any hematological disorder or prior solid organ transplant
• Any serious or uncontrolled psychiatric or medical disorder that, in the opinion of the investigator, may increase the risk associated with the study participation or study drug administration, impair the ability of the participants to receive protocol therapy, or interfere with the interpretation of study results.
• Women who are pregnant or breastfeeding.
• Participants undergoing concurrent treatment with agents targeting TNF-α or IL-1 within 28 days of study enrollment.

Sponsors & Collaborators

• John Mascarenhas: lead

Study Sites (7)

Cedars-Sinai Medical Center, Samuel Oschin Comprehensive Cancer Institute

Los Angeles, California, 90048, United States

Location

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

The University of Kansas Cancer Center-Westwood

Westwood, Kansas, 66205, United States

Location

Ruttenberg Treatment Center

New York, New York, 10029, United States

Location

Atrium Health Levine Cancer Institute

Charlotte, North Carolina, 28204, United States

Location

Wake Forest Baptist Health Comprehensive Cancer Center

Winston-Salem, North Carolina, 27157, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

canakinumab

Condition Hierarchy (Ancestors)

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

Study Officials

• John Mascarenhas, MD

  MOUNT SINAI HOSPITAL

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: July 18, 2022
• First Posted: July 21, 2022
• Study Start: August 2, 2022
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): July 1, 2027
• Last Updated: May 7, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)