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Ropeginterferon Alfa 2b for Early MyelofibrosisDIPSS Low/Intermediate-1 Risk Myelofibrosis

Primary Purpose

Pre-fibrotic Myelofibrosis

Status
Recruiting
Phase
Phase 2
Locations
Taiwan
Study Type
Interventional
Intervention
Ropeginterferon Alfa-2B Prefilled Syringe [Besremi]
Sponsored by
National Taiwan University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pre-fibrotic Myelofibrosis focused on measuring Early or Primary Myelofibrosis

Eligibility Criteria

20 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Adults ≥ 18 years (or based on the legal age of the territory) Diagnosed of primary myelofibrosis, post-PV and post-ET myelofibrosis according to the WHO 2016 classification Bone marrow reticulin fibrosis grade of 0-1 or low/intermediate-1 risk according to DIPSS Compensated liver function defined as: bilirubin ≤ 1.5 x upper limit normal (ULN); alanine aminotransferase (ALT) ≤ 2 x ULNor aspartate aminotransferase (AST) ≤ 2 x ULN; prothrombin time versus control <3 seconds at screening Glomerular filtration rate ≥ 50 mL/min (by MDRD equation or Cockcroft-Gault formula) Men and women of childbearing potential must agree to perform contraception until 28 days after the last dose of ropeg. Women must avoid breast-feeding during the study. Able to give a written informed consent and fully comply to the requirements of the study. Exclusion Criteria: Prior or current use of IFNα preparations for PMF or secondary MF. Prior use of IFNα for antecedent PV or ET is allowed provided that the time from the last dose of IFNα to recruitment is > 4 weeks. Patients currently on other investigational therapy (ies) Contraindications or hypersensitivity to IFNα preparations History of organ transplantation Pregnant or lactating women Documented autoimmune disease at screening Infection with human immunodeficiency virus (HIV) Active and uncontrolled infections with hepatitis B virus (HBV) and hepatitis C virus (HCV). Please note that patients on antiviral therapy with undetectable HBV DNA and HCV RNA may be recruited. Evidence of severe retinopathy including but not limited to macular degeneration, diabetic retinopathy and hypertensive retinopathy. History of clinically significant neuropsychiatric conditions including but not limited to depression and epilepsy. Clinically significant neuropsychiatric conditions including but not limited to depression and epilepsy. Presence of other active malignancies within three years prior to the time of recruitment. History of malignant disease, including solid tumours and haematological malignancies (except basal cell and squamous cell carcinomas of the skin and carcinoma in situ of the cervix that have been completely excised and are considered cured) within the last 3 years. Evidence of alcohol or drug abuse within 6 months

Sites / Locations

  • National Taiwan University HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Ropeginterferon alfa-2b

Arm Description

Eligible subjects will receive ropeg subcutaneously (SC) every 2 weeks at the starting dose of 250µg at week 0, 350 µg at week 2, then 500µg at a fixed dose from week 4 onwards until week 104. In patients achieving a clinical or molecular response at 24 months (week 104), treatment with ropeg will be continued until disease progression. Intervention: Drug: Ropeginterferon alfa-2b

Outcomes

Primary Outcome Measures

Clinicohematological responses at 24 weeks
Overall haematological response rate at 6, 12 and 24 months, based on the IWG-MRT and ELN consensus report

Secondary Outcome Measures

Adverse events
Adverse events according to the CTCAE version 5.0.

Full Information

First Posted
February 7, 2023
Last Updated
February 7, 2023
Sponsor
National Taiwan University Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT05731245
Brief Title
Ropeginterferon Alfa 2b for Early MyelofibrosisDIPSS Low/Intermediate-1 Risk Myelofibrosis
Official Title
Efficacy and Safety of Ropeginterferon Alfa-2b for Pre-fibrotic Primary Myelofibrosis and DIPSS Low/Intermediate-1 Risk Myelofibrosis
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Recruiting
Study Start Date
February 8, 2023 (Actual)
Primary Completion Date
October 12, 2024 (Anticipated)
Study Completion Date
October 12, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Taiwan University Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No

5. Study Description

Brief Summary
This is a multi-centre phase 2 open-label prospective study designed to assess the efficacy and safety of ropeg patients with pre-fibrotic primary myelofibrosis or DIPSS low/intermediate-1 risk myelofibrosis after 24 months of treatment.
Detailed Description
This is a multi-centre phase 2 open-label prospective study designed to assess the efficacy and safety of ropeg patients with pre-fibrotic primary myelofibrosis or DIPSS low/intermediate-1 risk myelofibrosis after 24 months of treatment. . In patients achieving any molecular response at 24 months, treatment with ropeg will be continued until disease progression. After obtaining a written informed consent, screening evaluations will be performed. Eligibility will be determined based on the inclusion and exclusion criteria in section 6 of this protocol. Subject visits will be scheduled regularly after recruitment for efficacy evaluations and safety assessments. A safety follow-up will be scheduled 28 days after end-of-treatment visit. Efficacy evaluations, safety assessments and sample collection will be performed according to the schedule laid in section 2 in this protocol. Efficacy will be evaluated using laboratory assessment of haematological parameters, physical examination for liver and spleen size assessment, quantitative assessment of JAK2V617F, CALR, MPL and other driver mutations, bone marrow examination, and symptom burden assessment by MPN-SAF-TSS. Quantitative assessment of JAK2V617F, CALR, MPL and other driver mutations will be performed using real-time quantitative PCR or ddPCR at the laboratory at the Department of Medicine, the University of Hong Kong, National Taiwan University Hospital and Chang Gung Memorial Hospital Chiayi. Safety evaluations will be performed using symptoms, physical examination, laboratory studies, Chest X-rays, ophthalmic assessment, ECOG performance status and CTCAE version 5.0.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pre-fibrotic Myelofibrosis
Keywords
Early or Primary Myelofibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
50 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Ropeginterferon alfa-2b
Arm Type
Experimental
Arm Description
Eligible subjects will receive ropeg subcutaneously (SC) every 2 weeks at the starting dose of 250µg at week 0, 350 µg at week 2, then 500µg at a fixed dose from week 4 onwards until week 104. In patients achieving a clinical or molecular response at 24 months (week 104), treatment with ropeg will be continued until disease progression. Intervention: Drug: Ropeginterferon alfa-2b
Intervention Type
Drug
Intervention Name(s)
Ropeginterferon Alfa-2B Prefilled Syringe [Besremi]
Intervention Description
Eligible subjects will receive Ropeginterferon alfa-2b subcutaneously (SC) every 2 weeks at the starting dose of 250µg at week 0, 350 µg at week 2, then 500µg at a fixed dose from week 4 onwards
Primary Outcome Measure Information:
Title
Clinicohematological responses at 24 weeks
Description
Overall haematological response rate at 6, 12 and 24 months, based on the IWG-MRT and ELN consensus report
Time Frame
24 months
Secondary Outcome Measure Information:
Title
Adverse events
Description
Adverse events according to the CTCAE version 5.0.
Time Frame
24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Adults ≥ 18 years (or based on the legal age of the territory) Diagnosed of primary myelofibrosis, post-PV and post-ET myelofibrosis according to the WHO 2016 classification Bone marrow reticulin fibrosis grade of 0-1 or low/intermediate-1 risk according to DIPSS Compensated liver function defined as: bilirubin ≤ 1.5 x upper limit normal (ULN); alanine aminotransferase (ALT) ≤ 2 x ULNor aspartate aminotransferase (AST) ≤ 2 x ULN; prothrombin time versus control <3 seconds at screening Glomerular filtration rate ≥ 50 mL/min (by MDRD equation or Cockcroft-Gault formula) Men and women of childbearing potential must agree to perform contraception until 28 days after the last dose of ropeg. Women must avoid breast-feeding during the study. Able to give a written informed consent and fully comply to the requirements of the study. Exclusion Criteria: Prior or current use of IFNα preparations for PMF or secondary MF. Prior use of IFNα for antecedent PV or ET is allowed provided that the time from the last dose of IFNα to recruitment is > 4 weeks. Patients currently on other investigational therapy (ies) Contraindications or hypersensitivity to IFNα preparations History of organ transplantation Pregnant or lactating women Documented autoimmune disease at screening Infection with human immunodeficiency virus (HIV) Active and uncontrolled infections with hepatitis B virus (HBV) and hepatitis C virus (HCV). Please note that patients on antiviral therapy with undetectable HBV DNA and HCV RNA may be recruited. Evidence of severe retinopathy including but not limited to macular degeneration, diabetic retinopathy and hypertensive retinopathy. History of clinically significant neuropsychiatric conditions including but not limited to depression and epilepsy. Clinically significant neuropsychiatric conditions including but not limited to depression and epilepsy. Presence of other active malignancies within three years prior to the time of recruitment. History of malignant disease, including solid tumours and haematological malignancies (except basal cell and squamous cell carcinomas of the skin and carcinoma in situ of the cervix that have been completely excised and are considered cured) within the last 3 years. Evidence of alcohol or drug abuse within 6 months
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Gill Harinder
Phone
+852 22554542
Email
gillhsh@hku.hk
Facility Information:
Facility Name
National Taiwan University Hospital
City
Taipei, Taiwan, 100
Country
Taiwan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
hsinan hou
Phone
0972653089
Email
hsinanhou@ntu.edu.tw

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Ropeginterferon Alfa 2b for Early MyelofibrosisDIPSS Low/Intermediate-1 Risk Myelofibrosis

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