Blood and Lymph Conditions

All Conditions

Heart and Blood Diseases

Rare Diseases

Myeloproliferative Disorders

Thrombocytosis

Thrombocythemia, Essential

Blood Platelet Disorders

Hematologic Diseases

Bone Marrow Diseases

Hemostatic Disorders

Blood Coagulation Disorders

Hemorrhagic Disorders

Chronic Myeloproliferative Disorders

Essential Thrombocythemia

Gastrointestinal Agents

All Drugs and Chemicals

Antineoplastic Agents

Antisickling Agents

Fibrinolytic Agents

Platelet Aggregation Inhibitors

Calcium Carbonate

Hydroxyurea

Anagrelide

Ropeginterferon alfa-2b (P1101) dosage: from 250 mcg to 500 mcg

Ropeginterferon alfa-2b

Anagrelide dosage: 0.5 mg per capsule, according to label and physician's judgement

Toshiaki Sato, MD/PhD

Craig Zimmerman, PhD

This is a Phase 3 open-label, multicenter, randomized, active-controlled study designed to compare the efficacy and safety and tolerability of P1101 compared with ANA after 12 months of treatment as second-line therapy for subjects with ET who have had a suboptimal or failed response to HU.

PharmaEssentia Corporation is developing a pegylated (PEG) IFN-α product, P1101, for the treatment of ET.

Available clinical data and experience with P1101 in PV shows that the compound, with proper dose modifications, is effective in controlling disease in a significant proportion of subjects with ET. Further, its increased serum half-life presents distinct advantages for ET treatment over that of standard IFN-α and other available PEG IFN-α therapy. This pivotal Phase 3 study will establish the efficacy and safety of P1101 in ET subjects.

In core study phase, the enrolled subjects will be randomized into two arms, the test arm is P1101, the control arm is ANA. The overall duration for each eligible patient is 14 months, including screening (1 month), treatment (12 months) and follow-up (1 month) period. Efficacy evaluations, safety assessments, and PK and immunogenicity evaluations of P1101 will be performed.

Evaluation of efficacy will include clinical laboratory assessments, allelic burden measurements of CALR, JAK-2, and MPL, spleen size measurements, bone marrow sampling, EQ-5D-3L, and MPN-SAF TSS completion.

Evaluation of safety will include assessing vital signs, clinical safety laboratory tests, physical examinations, ECG evaluation, heart ECHO, lung X-ray, ECOG performance status, ocular examination, and AEs.

Subjects who completed the end of treatment (EoT) and safety follow-up (EoS) visits of the SURPASS ET study and may benefit from P1101 therapy have the opportunity to receive P1101 treatment.

Experimental Drug (Biological): Ropeginterferon alfa-2b (P1101), Q2W, SC injection Control Drug: Anagrelide, capsules, daily, p.o.

Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance

A Phase 3, Open-Label, Multicenter, Randomized, Active-controlled Study to Assess PK and Compare the Efficacy, Safety, and Tolerability of P1101 vs Anagrelide as 2nd Line Therapy for Essential Thrombocythemia (SURPASS ET): The Core Study and Its Extension Study.

platelets ≤400 x 10\^9/L AND white blood cells (WBC) \<9.5 x 10\^9/L

based on the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)

absence of hemorrhagic or thrombotic events

PharmaEssentia

Neoplasms

Symptoms and General Pathology

Leukemia

Leukemia, Myeloid

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Neoplasms by Histologic Type

Chronic Disease

Disease Attributes

Myeloid Leukemia

Chronic Graft Versus Host Disease

Chronic Myeloid Leukemia

Anti-Infective Agents

Amino Acids

Bosutinib

Interferons

Tyrosine Kinase Inhibitors

Protein Kinase Inhibitors

Enzyme Inhibitors

Tyrosine

Bosutinib, provided by Pfizer, starting dose of 200mg QD and stepwise dose escalation (\> 300 mg/d \> 400 mg/d) during the first three months. A pharmacological study will be performed in the French cohort (BOSUSTEP Substudy). BOS residual plasma concentration (Cmin) will be checked after initiation, before each dose step in the French cohort, and at M3 also for Nordic patients in ancillary studies.

Ro-Peg-Interferon α2b will be supplied by AOP Orphan to be administered by subcutaneous injections from prefilled injection pens. RoPegIFN will be given in an open-label fashion. Patients assigned to RoPegIFN will start with 50 μg injected subcutaneously every 14 days, in combination with Bosutinib.

Ropeginterferon

Henrik Hjorth-Hansen, md phd

Lydia Roy, md phd

Rie Sander Bech

Jesper Stentoft

Christen Lykkegård Andersen

Andreja Dimitrijevic

Marianne Augustenborg

Perttu M Koskenvesa

Bjørn Tore Gjertsen

Øystein Sefland

Kristin H Låstad

Eivind Galteland

Tobias Gedde-Dahl

Margrete Friestad

Mohammed Waleed Majeed

Mats I Olsen

Anders Vik

Kourosh Lotfi

Arta Dreimane

Johan Richter

Anna Lubking

Leif Stenke

Anders Själander

Berit Markevärn

Ulla Olsson-Strömberg

Erik Ahlstrand

Tom Christian Martinsen, md phd

To study the efficacy and safety of combination of Ro-Peg-interferon-α2b (RoPegIFN) with Bosutinib (BOS) in comparison to BOS monotherapy, as frontline therapy for newly diagnosed chronic myeloid leukemia patients, and to estimate efficacy of the addition of RoPegIFN to BOS in terms of deep molecular response with the aim of increasing the proportion of patients who may achieve treatment free remission. (NCMLSG study #NordCML012)

Long-acting Low Dose Ropeginterferon for Chronic Myeloid Leukemia Treated With Bosutinib From Diagnosis

A Study of Efficacy and Safety of Long-acting Low Dose Ropeginterferon in Patients With Chronic Myeloid Leukemia Treated With Bosutinib From Diagnosis: a Randomized Prospective Trial

Molecular response 4 (MR4) is defined by either a positive BCR-ABL/ABL ratio ≤ 0.01% on the international scale (IS) or by undetectable BCR-ABL with the analysis of at least 10000 copies of ABL or 24000 copies of GUS (according to the ELN recommendations by N. Cross et al., Leukemia 2015)

type and grade according to the NCI CTCAE v4.03

Sustained deep molecular response (MR) criteria will be defined according updated data and ELN guidelines before the first patient will achieve month 48 (at least a MR4 over a 12 months period and confirmed on the last centralized measurement at month 48

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance	NCT04285086	Essential Throm...	Ropeginterferon... Anagrelide	18 Years -	PharmaEssentia
Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance	NCT04285086	Essential Throm...	Ropeginterferon... Anagrelide	18 Years -	PharmaEssentia
Long-acting Low Dose Ropeginterferon for Chronic Myeloid Leukemia Treated With Bosutinib From Diagnosis	NCT03831776	Chronic Myeloid...	Bosutinib Ropeginterferon	18 Years - 75 Years	St. Olavs Hospital

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