Blood and Lymph Conditions

All Conditions

Neoplasms

Rare Diseases

Anemia

Thrombocytopenia

Polycythemia Vera

Polycythemia

Primary Myelofibrosis

Thrombocytosis

Thrombocythemia, Essential

Blood Platelet Disorders

Hematologic Diseases

Cytopenia

Myeloproliferative Disorders

Bone Marrow Diseases

Bone Marrow Neoplasms

Hematologic Neoplasms

Essential Thrombocythemia

Chronic Myeloproliferative Disorders

All Drugs and Chemicals

Antineoplastic Agents

Antibodies

Antibodies, Monoclonal

Immunoglobulins

Siltuximab

Immunologic Factors

Patients receive siltuximab IV over 60 minutes on day 1. Patients also undergo bone marrow biopsy and aspiration at baseline and at the end of treatment (within 30 days of last siltuximab dose) or as clinically indicated. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients who are responding after 6 courses may receive additional siltuximab treatment for up to 1 year at the discretion of the study doctor.

Undergo bone marrow aspiration and biopsy

Bone Marrow Aspiration and Biopsy

Laboratory Biomarker Analysis

Brady Stein, MD

The main purpose of this investigational research study is to determine how safe and tolerable the study drug siltuximab is in patients with myelofibrosis (MF). This medication has been approved by the FDA for another condition (multicentric castleman's disease (MCD), but not for myelofibrosis (MF). In MCD, siltuximab resulted in improvement in symptoms and anemia. While MCD and MF are different diseases, they share some common features including a protein call interleukin-6 (IL-6) that may be important in causing symptoms of MCD and MF.

PRIMARY OBJECTIVES:

I. To evaluate safety and tolerability of the drug siltuximab in the myelofibrosis patient population.

SECONDARY OBJECTIVES:

I. To determine clinical improvement (CI). II. To determine the overall response rate (ORR).

TERTIARY OBJECTIVES:

I. To determine if siltuximab results in improvement in iron dysregulation. II. To determine changes in inflammatory stress that may correlate with clinical response.

OUTLINE:

Patients receive siltuximab intravenously (IV) over 60 minutes on day 1. Patients also undergo bone marrow biopsy and aspiration at baseline and at the end of treatment (within 30 days of last siltuximab dose) or as clinically indicated. Treatment repeats every 21 days for up to 6 courses in the absence of disease progression, unacceptable toxicity, or patient decision to withdraw. Patients who are responding after 6 courses may receive additional siltuximab treatment for up to 1 year at the discretion of the study doctor.

After completion of study treatment, patients are followed up at 30 days.

Siltuximab in Treating Patients With Primary, Post-Polycythemia Vera, or Post-Essential Thrombocythemia Myelofibrosis

A Single-arm, Single Center, Pilot Study of Siltuximab, an Anti-IL6 Therapy, for Patients With Myelofibrosis

Peripheral blood will be taken to determine if siltuximab results in improvement in iron dysregulation.

To determine changes in inflammatory stress that may correlate with clinical response. To assess this, CRP will be measured.

To determine changes in inflammatory stress that may correlate with clinical response. To assess this, hepcidin will be measured.

To evaluate safety and tolerability of the drug siltuximab in the myelofibrosis patient population. Adverse events will be assessed by type, timing, frequency, and attribution and will be graded according to the NCI's common terminology criteria, version 4.03.

Changes in the baseline symptom burden using Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) questionnaire.

To determine spleen response, palpation will be used, using centimeters below the left costal margin, and if body habitus is prohibitive, by ultrasonography.

Baseline hemoglobin level and/or transfusion-dependence will be recorded at baseline and compared to results after cycle 3

The ORR will be assessed after 6 cycles of therapy using standard criteria published by the International Working Group for Myelofibrosis Research and Treatment which will evaluate for complete response, partial response, clinical improvement, or stable disease.

National Cancer Institute (NCI)

Northwestern University

Heart and Blood Diseases

Hemostatic Disorders

Blood Coagulation Disorders

Hemorrhagic Disorders

Ropeginterferon alfa-2b-njft (P1101)

Jewell Jessup, PhD

Mitch Shea

Jaime Chang

Anne Caldwell

Jennifer Steffen

Shannon Gleason

Melissa Parker

Barbie Warner

Leta Ko

Steven Schmitt

Morgan Johnson

Karyn Gordon

Layla Tapia

Stephanie Ortiz

Jason Brecher

Noelle Townsend

Zita Makselyte

Peggy Alton

Toria Wilson

Ines Hernandez

Suzhen Gong

Mary Ann Richie

Braxton Smith

Ellie Gorham

Tathiana Ruiz

Christopher Hillis

Nina Dimson

Ray Urbanski, MD, PhD

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of Ropeginterferon alfa-2b-njft (P1101) in Adult Patients with Essential Thrombocythemia

PharmaEssentia is developing a pegylated (PEG) IFN-α product, P1101, for the treatment of Essential Thrombocythemia (ET) as lack of disease modifying therapies in essential ET constitutes a serious issue in modern hematology.

Ropeginterferon alfa-2b-njft (P1101) may represent an effective, well-tolerated treatment with the ability to provide a deeper response and superior control of important blood parameters with the potential to alter the course of the disease and prevent progression to post-ET myelofibrosis (MF) and/or secondary acute myeloid leukemia (sAML). Ropeginterferon alfa-2b-njft (P1101) is currently being evaluated in comparison to ANA in the ongoing global Phase 3 clinical study, SURPASS ET.

Enrolled patients will receive P1101 over 13 months followed by an extension period.

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of Ropeginterferon Alfa-2b-njft (P1101) in Adult Patients With Essential Thrombocythemia

Efficacy will be based on peripheral blood count remission as defined by hematocrit (HCT) \<45%, white blood cell (WBC) count ≤10 × 109/L, and platelets (PLT) ≤ 400 × 109/L in at least 80% of bi-weekly measurements for a consecutive 32-wek period during the 52-week core study treatment period.

PharmaEssentia

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Siltuximab in Treating Patients With Primary, Post-Polycythemia Vera, or Post-Essential Thrombocythemia Myelofibrosis	NCT02805868	Myelofibrosis Polycythemia Ve... Primary Myelofi... Thrombocytopeni...	Bone Marrow Asp... Laboratory Biom... Siltuximab	18 Years -	Northwestern University
A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET	NCT05482971	Essential Throm...	Ropeginterferon...	18 Years -	PharmaEssentia

Spots Global Cancer Trial Database for essential

Every month we try and update this database with for essential cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for essential

Every month we try and update this database with for essential cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for essential cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.