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

Digestive System Diseases

Symptoms and General Pathology

Infections

Carcinoma

Carcinoma, Hepatocellular

Recurrence

Hepatitis A

Hepatitis

Hepatitis B

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Adenocarcinoma

Liver Neoplasms

Gastrointestinal Neoplasms

Digestive System Neoplasms

Gastrointestinal Diseases

Liver Diseases

Pharmaceutical Solutions

Nivolumab

Antineoplastic Agents, Immunological

Immune Checkpoint Inhibitors

Phase I of Study : To determine the safety, tolerability, DLT, and potential phase 2 dose of sequential administration of P1101 and anti-PD1

:Sequential administration 6 doses (450mcg) of P1101 and 3 doses of anti-PD1 (Escalating from 0.3, 0.75, 1.5, 3 mg/kg) for Phase I Study

Phase II Study Group I: anti-PD1 arm 3mg/kg 3 doses

Phase II Study Group II: P1101 arm 450mcg 12 doses

Phase II Study GroupIII:Sequential administration of 6 doses of 450mcg P1101 and followed by 3 doses of anti-PD1 dosage (base on Phase I study result)

solution for injection in prefilled syringe, 500 µg/ mL , 450μg /time, subcutaneous injection every 2 weeks

P1101 (Ropeginterferon alfa-2b)

Phase I study will use 0.3, 0.75, 1.5, 3mg/kg, Q2W for 3 doses after 6 doses of P1101.

Phase II study : Group I will use 3mg/kg, Q2W for 3 doses; Group III will use the dosage that determine from Phase I study 3 doses after 6 doses of P1101

Pei-Jer Chen

Shu-Fen Chang

The main purpose of this trial is to evaluate the safety of the new adjuvant treatment of curative HCC, or the treatment of long-acting interferon P1101 alone, or the use of long-acting interferon P1101 and subsequent treatment of anti-PD1, and any efficacy in reducing the recurrence rate of patients after surgery.

secondary end-point: P1101 and anti-PD1 sequential therapy on hepatitis B (especially on HbsAg).

Phase I : Dose escalation study with Anti-PD1 dossages 4 cohorts Phase II : 3 parallel arms

P1101 and Anti-PD1 for After Curative Surgery of Hepatitis B-related Hepatocellular Carcinoma

A Phase I/II Open Label Study to Evaluate Safety and the Prophylactic Effect on Recurrence of Anti-PD1 Monotherapy, P1101 Monotherapy, and Sequential Administration of P1101 and Anti-PD1 After Curative Surgery of HBV-related HCC

To determine the potential phase 2 dose of sequestial administration of P1101 and anti-PD1. The MTD is determine by the prior dose level below the dose level at which ≥2/3 or ≥2/6 subjects suffer dose-limiting toxicity (DLT).

To evaluate safety(assessment of AE, SAE and unanticipated problem) and the recurrence-free survival (defined as the time from randomization to HCC recurrence or death from any cause, whichever occured first) at 48 weeks after randomization of anti-PD 1 monotherapy, P1101 monotherapy, and sequential administration of P1101 and anti-PD 1 therapy arms

To assess the effect of anti-PD1 monotherapy, P1101 monotherapy, and sequential administration of P1101 and anti-PD1 in inhibiting the recurrence, using disease-free survival (defined as the time from randomization to HCC recurrence, death from any cause, or onset of secondary tumor, whichever occurred first) at 48 weeks after randomization as the endpoint

To assess the treatment effect of anti-PD1 monotherapy, P1101 monotherapy, or sequential administration of P1101 and anti-PD1 in inhibiting the recurrence, using recurrence-free survival (defined as the time from randomization to HCC recurrence or death from any cause, whichever occurred first) at 96 weeks after randomization as the endpoint

To assess the change in mean HBsAg level from baseline at the end of treatment (EOT), 24 weeks and 48 weeks after randomization

National Taiwan University Hospital

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.

Leukemia

Leukemia, Myeloid

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Leukemia, Myeloid, Chronic-Phase

Chronic Disease

Disease Attributes

Myeloid Leukemia

Chronic Graft Versus Host Disease

Chronic Myeloid Leukemia

Anti-Infective Agents

Amino Acids

Interferons

Peginterferon alfa-2b

Interferon-alpha

Imatinib Mesylate

Interferon alpha-2

Antiviral Agents

Immunologic Factors

Proline

P1101 50µg s.c. will be administered every 2 weeks in addition to preexisting imatinib treatment. In the absence of dose limiting toxicities after 12 weeks, the dose will be escalated to 100µg every 2 weeks.

Maximum treatment duration will not expand 18 months.

P1101

Josef Thaler, MD

In this phase I pilot study, it is planned to investigate the feasibility and safety of adding an interferon therapy to an preexisting imatinib treatment in patients with chronic phase chronic myeloid leukaemia. The participating patients have already reached a response during their imatinib therapy (CCyR) but have still a detectable disease (no molecular response MR 4.5 or better).

Addition of P1101 to Imatinib Treatment in Patients With Chronic Phase Chronic Myeloid Leukaemia Not Achieving a Complete Molecular Response

Phase 1 Study to Evaluate the Feasibility and Efficacy of the Addition of P1101 (PEG-Proline-Interferon Alpha-2b) to Imatinib Treatment in Patients With Chronic Phase Chronic Myeloid Leukaemia Not Achieving a Complete Molecular Response (MR 4.5 or BCR-ABL Transcripts Not Detectable)

The primary objective is to determine the safety and tolerability of the addition of P1101 to the pre-study established dose of imatinib.

Secondary objective is to determine the rate of achievement of ≥ 1 log reduction from the initial BCR-ABL transcript level at study entry and the achievement of molecular remission 4.5 or undetectable BCR-ABL transcripts.

AOP Orphan Pharmaceuticals AG

Arbeitsgemeinschaft medikamentoese Tumortherapie

Polycythemia Vera

Polycythemia

Bone Marrow Neoplasms

Hematologic Neoplasms

Ropeginterferon alfa-2b subcutaneously (SC) every two weeks (± 3 days), target optimal dose of 500 µg. Phlebotomy should be conducted when Hct ≥ 45%.

Phlebotomy only and should be conducted when Hct ≥ 45%.

Ropeginterferon alfa-2b subcutaneously (SC) every two weeks (± 3 days), 250 µg at Day 1, 350 µg at Week 2, and target optimal dose of 500 µg at Week 4.

Control group patients will receive phlebotomy only

Phlebotomy

Ting-Fang Wang

Evan Huang

This is a randomized, open-label, multicenter, two-arm study to assess the efficacy and safety of ropeginterferon alfa-2b for patients with low-risk PV. Approximately 110 patients with low-risk PV will be enrolled. The whole study period is 108 weeks, including a main treatment phase (56 weeks), an extension treatment phase (48 weeks), and a safety follow-up phase (four weeks).

Efficacy and Safety of Ropeginterferon Alfa 2b (P1101) for Patients With Low-Risk Polycythemia Vera

Efficacy and Safety of Ropeginterferon Alfa 2b (P1101) for Patients With Low-Risk Polycythemia Vera - A Randomized Open Label Multicenter Study

Antipyretics

Anti-Inflammatory Agents

Antirheumatic Agents

Analgesics

Aspirin

Anti-Inflammatory Agents, Non-Steroidal

Analgesics, Non-Narcotic

Cyclooxygenase Inhibitors

Enzyme Inhibitors

Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks.

P1101 (ropeginterferon alfa-2b) will be administered subcutaneously every 2 weeks at the starting dose of 100 μg every two weeks (or 50 μg in patients under another cytoreductive therapy). The dose should be gradually increased by 50 μg every two weeks (in parallel, other cytoreductive therapy should be decreased gradually, as appropriate) until stabilization of the hematological parameters is achieved (hematocrit \<45%, platelets \<400 x 10\^9/L and leukocytes \<10 x 10\^9/L). The maximum recommended single dose is 500 μg injected every two weeks. The dose will be maintained at the highest level which can be tolerated and delivers best possible disease response.

Low-dose aspirin (acetylsalicylic acid) (75-150 mg/day) will be given as background therapy during the 12 months of study treatment, unless contraindicated.

Low-dose aspirin

Phlebotomy is performed aiming at a hematocrit \< 45%. When the hematocrit value is 45% or higher, phlebotomy is performed. The volume of phlebotomy per procedure should be 200 to 400 mL while monitoring the circulatory dynamics such as blood pressure and pulse. In the elderly and patients with cardiovascular disorders, a small volume (100-200 mL) should be considered to avoid rapid changes in hemodynamics.

This is a Phase 2 single arm study to investigate efficacy and safety of P1101 for adult Japanese patients with PV.

Eligible patients will be treated with P1101, starting at 100 μg (or 50 μg in patients under another cytoreductive therapy). The dose should be gradually increased by 50 μg every two weeks (in parallel, other cytoreductive therapy should be decreased gradually, as appropriate) until stabilization of the hematological parameters is achieved (hematocrit \<45%, platelets \<400 x 10\^9/L and leukocytes \<10 x 10\^9/L). The maximum recommended single dose is 500 μg injected every two weeks.

At week 36 (month 9) and week 52 (month 12), the primary study endpoint, phlebotomy-free CHR, will be analyzed. After completion of the 52-week study duration, provision and administration of P1101, collection of the long-term follow up information (blood parameters, molecular and cytogenetic data, safety parameters and as also the optional bone marrow data) will be continued until the drug becomes commercially available for all study subjects..

Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of P1101 (ropeginterferon alfa-2b) once every 2 weeks

Efficacy and Safety of P1101 in Polycythemia Vera Patients for Whom the Standard of Treatment is Difficult to Apply

Phase 2 Single Arm Study of Efficacy and Safety of P1101 for Polycythemia Vera (PV) Patients for Whom the Current Standard of Treatment is Difficult to Apply

Status of bone marrow histological remission defined as the disappearance of hypercellularity (age-adjusted), trilineage growth (panmyelosis) and absence of \>grade 1 reticulin fibrosis

The primary efficacy outcome measure is the proportion of subjects achieving durable phlebotomy-free complete hematologic response (CHR) at Month 12.

Durable phlebotomy-free CHR is defined as any subject achieving phlebotomy-free CHR at Month 9 and maintaining the response up to Month 12.

A responder in sense of a primary outcome measure is a subject who has met all the following criteria at the time points:

* Hematocrit \<45% phlebotomy-free (absence of phlebotomy during the previous 3 months)
* Platelet count ≤400 x 10\^9/L,
* WBC count ≤10 x 10\^9/L

WBC count will be recorded every 3 months.

Plt count will be recorded every 3 months.

Spleen size will be recorded every 3 months.

Time to requiring no phlebotomy is recorded.

Time required to first response is defined as time to achieve complete hematological response (CHR) without phlebotomy.

Duration of maintained complete hematologic response (CHR) since first achievement of CHR after administration of the study drug will be calculated.

Thrombotic or hemorrhagic events will be recorded any time during the study. The proportion of subjects without thrombotic or hemorrhagic events is defined as the proportion of subjects experienced no thrombotic and hemorrhagic events during the study period (12 months).

Quantitative JAK2 measurements at screening, Months 3, 6, 9 and 12 (central laboratory) for subjects who signed consent form. Change of JAK2 allelic burden over time will be assessed.

Trough concentration is the measured concentration of a drug at the end of a dosing interval at steady state every 2 weeks.

Additionally, serum concentration is measured at hours 0, 24, 48, 96 and 168 after administration at Week 0 and Week 28.

PharmaEssentia Japan K.K.

Pre-filled Syringe, Q2W starting at 250-350-500, SC injection

Pre-filled Syringe, Q2W starting at 100 up to 500 (50mcg increases), SC injection

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
P1101 and Anti-PD1 for After Curative Surgery of Hepatitis B-related Hepatocellular Carcinoma	NCT04233840	Hepatocellular ...	P1101 (Ropegint... Nivolumab	20 Years -	National Taiwan University Hospital
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
Addition of P1101 to Imatinib Treatment in Patients With Chronic Phase Chronic Myeloid Leukaemia Not Achieving a Complete Molecular Response	NCT01933906	Chronic Phase C...	P1101	18 Years -	Arbeitsgemeinschaft medikamentoese Tumortherapie
Efficacy and Safety of Ropeginterferon Alfa 2b (P1101) for Patients With Low-Risk Polycythemia Vera	NCT06290765	Polycythemia Ve... Myeloproliferat...	Ropeginterferon... Phlebotomy	18 Years - 59 Years	PharmaEssentia
P1101 and Anti-PD1 for After Curative Surgery of Hepatitis B-related Hepatocellular Carcinoma	NCT04233840	Hepatocellular ...	P1101 (Ropegint... Nivolumab	20 Years -	National Taiwan University Hospital
Efficacy and Safety of P1101 in Polycythemia Vera Patients for Whom the Standard of Treatment is Difficult to Apply	NCT04182100	Polycythemia Ve...	P1101 Low-dose aspiri... Phlebotomy	20 Years -	PharmaEssentia Japan K.K.
A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients With PV	NCT05481151	Polycythemia Ve...	P1101 (Ropegint... Ropeginterferon...	18 Years -	PharmaEssentia
P1101 and Anti-PD1 for After Curative Surgery of Hepatitis B-related Hepatocellular Carcinoma	NCT04233840	Hepatocellular ...	P1101 (Ropegint... Nivolumab	20 Years -	National Taiwan University Hospital
A Study to Access Efficacy and Safety of P1101 in Chinese PV Patients Who Are Intolerant or Resistance to HU	NCT05485948	Polycythemia Ve...	Ropeginterferon...	18 Years -	PharmaEssentia
A Study to Evaluate P1101 in Japanese PV Patients	NCT06002490	Polycythemia Ve...	P1101 Low-dose aspiri... Phlebotomy	18 Years -	PharmaEssentia Japan K.K.

Spots Global Cancer Trial Database for p1101

Every month we try and update this database with for p1101 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 p1101

Every month we try and update this database with for p1101 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 p1101 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.