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

Immune System Diseases

Skin and Connective Tissue Diseases

Mastocytosis

Urticaria

Urticaria Pigmentosa

Neoplasms, Connective Tissue

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Mast Cell Activation Disorders

Melody C Carter, M.D.

This study will evaluate children with mastocytosis, a disease of excessive mast cells in tissues such as skin and bone marrow, to identify the cause of the disease and describe its course. Mast cells can release chemicals that cause itching, blisters, flushing, bone pain, and abdominal pain. Usually, mastocytosis in children involves the skin only and is of limited duration. This study, however, will focus on children with more severe disease that more closely resembles adult-onset mastocytosis.

Patients up to 21 years of age with childhood-onset mastocytosis may be eligible for this study. Candidates must have one or more of the following abnormalities, which indicate severe disease: enlarged liver or spleen; diffuse skin involvement; history of gastrointestinal bleeding or peptic ulcer; bone marrow biopsy with abnormal mast cells either in number or shape; elevated blood levels of the enzyme tryptase; or abnormal hemoglobin, white blood cells, platelets, or clotting factors.

Participants will have a medical history and physical examination; various blood tests, including studies to identify genetic changes that are important in the growth, development, and functioning of human mast cells; and bone marrow aspiration and biopsy. For the bone marrow procedure, the skin over the hipbone and the outer surface of the bone itself are numbed with an injection of local anesthesia. Then, a special needle is inserted into the hipbone and about 2 tablespoons of bone marrow are drawn into a syringe. Another needle is then inserted through the first needle to collect a small piece of the bone marrow. Pain will be managed according to the individual patient s needs. Additional procedures, such as a gastroenterology consultation, colonoscopy to examine the colon, or computerized axial tomography (CT) or ultrasound of the abdomen to assess the liver and spleen, may be done if medically indicated. Standard medical treatment, including antihistamines for itching or steroids for abdominal cramping or diarrhea, will be recommended as appropriate.

Patients biologic parents may also be enrolled to provide a blood sample for genetic analysis and a bone marrow aspirate and biopsy for clinical and research purposes.

Patients will return to NIH once a year for follow-up evaluations until their disease is stable or until the 5-year study ends.

...

Mastocytosis in infants and children is an unusual disease characterized by an excess of mast cells in tissues. In pediatric onset mastocytosis, disease is usually localized to the skin and disease is considered to be of limited duration. However, a subset of children appear to develop a clinical picture resembling that observed in adults who have adult-onset disease. This study will focus on children with more severe mastocytosis in an attempt to define its pathogenesis, focusing on mutations and polymorphisms in genes regulating mast cell proliferation and survival to determine if they might contribute to this disease pattern. If severe pediatric-onset disease does not fit within the existing classification, new criteria of diagnosis must be proposed for the pediatric age group.

This study will examine children and young adults age birth to 21 years with pediatric-onset disease previously enrolled on NIAID mastocytosis protocols, or as a result of physician referral, which have disease more consistent with adult-onset disease or parameters associated with increased morbidity. The evaluation may include serum tryptase, blood count, bone marrow biopsy and aspirate, morphology and mutational analysis. Subjects may be asked to return in 12-24 months for assessment. If relevant mutation effecting mast cell growth and function are identified, such mutations will be sought in the biologic parents as appropriate, but only when such mutations are believed to be germ line (not somatic) mutations. This study will aid in the understanding of the characteristics of severe mastocytosis in the pediatric age group.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
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
Cause and Natural Course of Pediatric-Onset Mastocytosis	NCT00050193	Mastocytosis		- 21 Years	National Institutes of Health Clinical Center (CC)
Cause and Natural Course of Pediatric-Onset Mastocytosis	NCT00050193	Mastocytosis		- 21 Years	National Institutes of Health Clinical Center (CC)

Spots Global Cancer Trial Database for severe

Every month we try and update this database with for severe 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 severe

Every month we try and update this database with for severe 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 severe 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.