NCT05731245

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

Efficacy and Safety of Ropeginterferon Alfa-2b for Pre-fibrotic Primary Myelofibrosis and DIPSS Low/Intermediate-1 Risk Myelofibrosis

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: National Taiwan University Hospital · Academic / Other
• Sex: All
• Age: 20 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Pre-fibrotic Myelofibrosis

Interventions

Type	Name	Description
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

Timeline

• Start date: 2023-02-08
• Primary completion: 2024-10-12
• Completion: 2026-10-12
• First posted: 2023-02-16
• Last updated: 2023-02-16

Locations

1 site across 1 country: Taiwan