Symptoms and General Pathology

All Conditions

Blood and Lymph Conditions

Neoplasms

Heart and Blood Diseases

Rare Diseases

Fibrosis

Myeloproliferative Disorders

Polycythemia Vera

Polycythemia

Primary Myelofibrosis

Thrombocytosis

Thrombocythemia, Essential

Splenomegaly

Bone Marrow Diseases

Hematologic Diseases

Bone Marrow Neoplasms

Hematologic Neoplasms

Blood Platelet Disorders

Hemostatic Disorders

Blood Coagulation Disorders

Hemorrhagic Disorders

Chronic Myeloproliferative Disorders

Essential Thrombocythemia

intravenous in dose cohorts of 70mg/m2 or 180 mg/m2

AVID200

John Mascarenhas, MD

Ruben Mesa, MD

Ronald Hoffman, MD

Increased levels of TGF-β1 were detected in serum, plasma and BM and positively correlated with both grade of BMF and extent of leukemic cell infiltration in the marrow. TGF-β likely plays a dual role in promoting myelofibrosis and myeloproliferation, both of which are the bone marrow morphologic hallmark of MF. AVID200 is a drug that targets TGF-β1 and TGF-β3. The study team hypothesizes that inhibiting the TGF-β signaling pathway in MF will decrease the fibrogenic stimuli leading to myelofibrosis and concomitantly interrupt myeloproliferation and restore normal hematopoiesis.

This is a first in human, open-label, multicenter, Phase I/Ib trial of AVID200. Patients must have intermediate-2 or higher primary myelofibrosis (PMF), post-essential thrombocythemia or polycythemia-vera related MF (Post ET/PV MF). This study will enroll up to 24 patients. AVID200 is delivered by IV infusion on day 1 of each 3 week cycle.

This is a first in human, open-label, multicenter, Phase I/Ib trial of AVID200. To date, there is no therapy for MF evaluated in the clinic that clearly demonstrates the ability to target the malignant HSC and result in effective and reproducible bone marrow morphologic, cytogenetic and molecular responses. Medicinal therapies that result in disease course modification are urgently needed in this chronic and progressive myeloid malignancy. TGF-β likely plays a dual role in promoting myelofibrosis and myeloproliferation, both of which are the bone marrow morphologic hallmark of MF. The study team proposes that inhibiting the TGF-β signaling pathway in MF will decrease the fibrogenic stimuli leading to myelofibrosis and concomitantly interrupt myeloproliferation and restore normal hematopoiesis. AVID200 is a fusion protein containing TGF-β receptor ectodomains fused to a human Fc IgG domain. AVID200 is a potent TGFβ trap with antibody-like properties which has pM potency against two of the three TGFβ ligands, TGFβ1 and β3.

MPN-RC 118 AVID200 in Myelofibrosis

Phase I Study of AVID200 in Patients With Myelofibrosis (Myeloproliferative Neoplasms Research Consortium [MPN-RC] 118)

Cohorts of 3 patients with a maximum evaluable sample size of 12 patients and a target toxicity rate of 30% to estimate the MTD. Each cycle is 21 days.

Subjects attaining at least a CI (clinical improvement) by IWG/ELN criteria, or a decrease in bone marrow fibrosis by ≥1 grade with otherwise stable disease, will be allowed to continue AVID200 in the extension phase of the trial.

response by IWG/ELN criteria at the end of Cycle 6 and Cycle 12. The IWG/ELN criteria: CR (complete remission), PR (partial remission), Clinical improvement, Anemia response, Spleen response, Symptoms response, PD (progressive disease), SD (stable disease), Relapse, Cytogenetic remission, and Molecular remission

bone marrow fibrosis grade at the end of Cycle 6 and 12. Bone marrow fibrosis (MF) is graded as MF-0 to MF-3, with higher number indicating more disease.

MF-SAFv4.0,each of the items are scored 0 to 10, with total summation from 0 to 100, with higher score indicating more symptoms.

EORTC QLQ-C30, 28 item scored from 1 (not at all) to 4 (very much), and 2 items scored 1 (very poor) to 7 (excellent). Total score from 28 - 126, with higher score indicating poorer health

Formation Biologics

National Cancer Institute (NCI)

Myeloproliferative Neoplasm Research Consortium (MPN-RC)

John Mascarenhas

Associate Professor

All Drugs and Chemicals

Janus Kinase Inhibitors

JAK-inhibitor-naive patients, treatment start with ruxolitinib less than 14 days prior to the baseline visit

Patients pretreated with a JAK-inhibitor for more than 14 days prior to the baseline visit

Prospective observational study. There is no treatment allocation. Patients administered Jakavi by prescription and administered according to the SmPC.

Jakavi

Novartis Pharmaceuticals

This was a prospective, two-arm, non-interventional study of JAKAVI® (Ruxolitinib) in patients with myelofibrosis

The purpose of this NIS was to gather data from the daily clinical practice of the Jakavi®-treatment in a broad patient population. In order to evaluate the direct effect of Jakavi®, only JAK inhibitor naive patients were documented in the first study arm; patients pretreated with JAK inhibitors were documented in the second study arm to evaluate the long-term efficacy of Jakavi® in this subpopulation.

The documentation of all patients was carried out prospectively and began after the baseline visit. The medical decision on which therapeutic and diagnostic measures to take was made solely by the responsible physician. The observational period per patient was 36 months. The visit schedule after the baseline visit was set by the responsible physician according to standard clinical care, the clinical condition of the respective patients and the SmPC.

A Prospective, Two-arm, Non-interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis

A Prospective, Two-arm, Non Interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis

Evaluation of all occurring adverse events, serious adverse events and serious and non-serious adverse drug reactions

Spleen size (or volume) reduction was measured by palpation

The ECOG performance status is a scale used to assess how a patient's disease is progressing, assess how the disease affects the daily living abilities of the patient, and determine appropriate treatment and prognosis.

The grade ranges from 0 (fully active, able to carry on all pre-disease performance without restriction) to 5 (dead).

Number of patients with change in constitutional symptoms was collected

The MPN-SAF questionnaire contains important questions that cover MF-specific symptoms whose analysis is part of the standard of care. It includes disease related symptoms each scored from 0 (absent) to 10 (worst imaginable). Total Scores range from 0-100, with higher scores indicating a greater number of symptoms and severity.

This questionnaire consists of questions measuring physical function, physical role limitation, pain, general health, vitality, social function, emotional role limitations, and mental health status. The scores that can be obtained from the scale vary between 0 and 100 and the increase in the scores indicates that the quality of life is high.

Overall survival for JAK inhibitor naive and pretreated patients

Ruxolitinib start and end dose was collected

Number of participants with therapy discontinuation and dose adjustments was collected

Number of patients with co-morbidities was collected

Number of patients with blood transfusion dependency was collected

Number of patients with concomitant medications prescribed for myelofibrosis therapy and for the management of side effects was collected

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
MPN-RC 118 AVID200 in Myelofibrosis	NCT03895112	Primary Myelofi... Post-essential ... Post-polycythem... Post ET MF Post PV MF	AVID200	18 Years -	Icahn School of Medicine at Mount Sinai
A Prospective, Two-arm, Non-interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis	NCT05044026	Primary Myelofi... Post-polycythem... Post-essential ...	Jakavi	18 Years - 120 Years	Novartis

Spots Global Cancer Trial Database for post et mf

Every month we try and update this database with for post et mf cancer trials from around the world to help patients and their families find trials that might be right for them.
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Spots Global Cancer Trial Database for post et mf

Every month we try and update this database with for post et mf 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 post et mf 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.