Primary Myelofibrosis, Prefibrotic StagePhase 2NCT04988815
Ropeginterferon Alfa 2b for Early Myelofibrosis
A Phase 2 study testing Ropeginterferon alfa-2b in people with Primary Myelofibrosis, Prefibrotic Stage and Myelofibrosis, run by The University of Hong Kong at 1 site in Hong Kong.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Myelofibrosis, Prefibrotic Stage, Myelofibrosis
- What is being tested?
- Ropeginterferon alfa-2b
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteHong Kong
- Study dates
- 1 Sep 2021to 31 Dec 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 13Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
Hong Kong1 site, 1 recruiting
- Recruiting
Department of Medicine, the University of Hong Kong, Queen Mary Hospital
Hong Kong
Harinder Singh Harry Gill, +852 22554542, gillhsh@hku.hk
About this study
Official title: Efficacy and Safety of Ropeginterferon Alfa-2b for Pre-fibrotic Primary Myelofibrosis and DIPSS Low/Intermediate-1 Risk Myelofibrosis
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.
Read the 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.
- Being tested
- Ropeginterferon alfa-2b
- Keywords
- Pre-fibrotic PMF, Early myelofibrosis, Ropeginterferon alfa-2b
- Medical subject headings
- Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ropeginterferon alfa-2bDrug
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
| Study group | Role | What they receive |
|---|---|---|
| Ropeginterferon alfa-2b | Experimental | 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. Receives: Drug: Ropeginterferon alfa-2b |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinicohematological responses at 24 weeks
Overall haematological response rate at 6, 12 and 24 months, based on the IWG-MRT and ELN consensus report
Measured: 24 months
Secondary outcomes (1)
Adverse events
Adverse events according to the CTCAE version 5.0.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2021
- Study start1 Sep 2021
- Primary completion31 Jul 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Myelofibrosis, Prefibrotic StageStudies of the same condition, open and under way.
- More trials run by The University of Hong KongThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The University of Hong Kong
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04988815
- Sponsor's protocol ID
- P1101MF
- Status last verified
- Oct 2022
- First submitted
- 25 Jul 2021
- Last updated
- 6 Oct 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Gill H, Au L, Yim R, Lee P, Li V, Chin L, Zhang Q, Chan PY, Lee CYY, Wu TKY, Leung GMK, Kwong YL. Ropeginterferon alfa-2b for prefibrotic myelofibrosis and lower-risk myelofibrosis requiring cytoreduction. Blood Adv. 2026 Jun 9;10(11):3920-3930. doi: 10.1182/bloodadvances.2026019612.
PubMed 41875235
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Ropeginterferon Alfa-2b for Pre-fibrotic Primary Myelofibrosis and DIPSS Low/Intermediate-1 Risk Myelofibrosis
- ClinicalTrials.gov ID
- NCT04988815
- Acronym / sponsor ID
- P1101MF
- Keywords
- Pre-fibrotic PMF, Early myelofibrosis, Ropeginterferon alfa-2b
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- The University of Hong KongUniversity, hospital or other
- Study start
- 1 Sep 2021 (actual)
- Primary completion
- 31 Jul 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 4 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ropeginterferon alfa-2b | Experimental | Drug: Ropeginterferon alfa-2bEligible 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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)
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ropeginterferon alfa-2bEligible 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 | Drug | Ropeginterferon alfa-2b |
Primary outcome
Clinicohematological responses at 24 weeks
Time frame: 24 months
Overall haematological response rate at 6, 12 and 24 months, based on the IWG-MRT and ELN consensus report
1 secondary outcome
Adverse events
Time frame: 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2022.
- Derived from this study: PubMed 41875235Gill H, Au L, Yim R, Lee P, Li V, Chin L, Zhang Q, Chan PY, Lee CYY, Wu TKY, Leung GMK, Kwong YL. Ropeginterferon alfa-2b for prefibrotic myelofibrosis and lower-risk myelofibrosis requiring cytoreduction. Blood Adv. 2026 Jun 9;10(11):3920-3930. doi: 10.1182/bloodadvances.2026019612.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ropeginterferon alfa-2b in people with Primary Myelofibrosis, Prefibrotic Stage and Myelofibrosis, run by The University of Hong Kong at 1 site in Hong Kong.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 6 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Hong Kong. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Harinder Gill (+852 22554542, gillhsh@hku.hk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinicohematological responses at 24 weeks (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04988815, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2022.