Treatment for early myelofibrosis using ropeginterferon alfa-2b
Efficacy and Safety of Ropeginterferon Alfa-2b for Pre-fibrotic Primary Myelofibrosis and DIPSS Low/Intermediate-1 Risk Myelofibrosis
This study is testing if a new treatment called ropeginterferon alfa-2b can help people with early myelofibrosis feel better and stay safe over two years.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | The University of Hong Kong Academic / other |
| Locations | 1 site (Hong Kong) |
| Trial ID | NCT04988815 on ClinicalTrials.gov |
What this trial studies
This multi-centre phase 2 open-label study evaluates the efficacy and safety of ropeginterferon alfa-2b in patients with pre-fibrotic primary myelofibrosis or low/intermediate-1 risk myelofibrosis. Participants will undergo regular assessments over 24 months to monitor hematological parameters, liver and spleen size, and molecular responses. The study aims to determine the treatment's effectiveness and safety, with continued treatment for those showing any molecular response at the 24-month mark. Safety follow-ups will occur 28 days after treatment ends.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older diagnosed with primary myelofibrosis or low/intermediate-1 risk myelofibrosis.
Not a fit: Patients who have previously used interferon alpha preparations for myelofibrosis may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve outcomes for patients with early myelofibrosis by potentially halting disease progression.
How similar studies have performed: Other studies have explored treatments for myelofibrosis, but this specific approach using ropeginterferon alfa-2b is relatively novel.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Hong Kong
- Department of Medicine, the University of Hong Kong, Queen Mary Hospital — Hong Kong, Hong Kong (Recruiting)
Study contacts
- Study coordinator: Harinder Gill
- Email: gillhsh@hku.hk
- Phone: +852 22554542
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.