A Phase 2 interventional study of Ropeginterferon Alfa-2B Prefilled Syringe [Besremi] in Pre-fibrotic Myelofibrosis, sponsored by National Taiwan University Hospital. Recruiting at 1 site in Taiwan. Open to participants aged 20 Years and older. Per ClinicalTrials.gov, last updated 2023-02-16.
Sponsored by National Taiwan University Hospital · Phase 2, Interventional, and Treatment
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.
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.
419 studies on the registry are indexed under Primary Myelofibrosis; 117 are open to participants now.
This study's planned enrollment of 50 is above the median of 44 across 347 interventional studies indexed under Primary Myelofibrosis.
Browse Primary Myelofibrosis studies →National Taiwan University Hospital is the lead sponsor of 2,563 studies on the registry; 569 are open to participants now.
Of its 11 completed or terminated interventional studies of FDA-regulated products, 2 (18%) have results posted.
Counted across the registry records on this site, refreshed daily.
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:
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
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
Drug: Ropeginterferon Alfa-2B Prefilled Syringe [Besremi]
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
Clinicohematological responses at 24 weeks
Overall haematological response rate at 6, 12 and 24 months, based on the IWG-MRT and ELN consensus report
Time frame: 24 months
Adverse events
Adverse events according to the CTCAE version 5.0.
Time frame: 24 months
Plan to share: No
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National Taiwan University Hospital