NCT04988815

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
50

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Sep 2021

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 25, 2021

Completed
10 days until next milestone

First Posted

Study publicly available on registry

August 4, 2021

Completed
28 days until next milestone

Study Start

First participant enrolled

September 1, 2021

Completed
3.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 31, 2025

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2025

Completed
Last Updated

October 6, 2022

Status Verified

October 1, 2022

Enrollment Period

3.9 years

First QC Date

July 25, 2021

Last Update Submit

October 3, 2022

Conditions

Primary Myelofibrosis, Prefibrotic StageMyelofibrosis

Keywords

Pre-fibrotic PMFEarly myelofibrosisRopeginterferon alfa-2b

Outcome Measures

Primary Outcomes (1)

  • Clinicohematological responses at 24 weeks

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

    24 months

Secondary Outcomes (1)

  • Adverse events

    24 months

Study Arms (1)

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.

Drug: Ropeginterferon alfa-2b

Interventions

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

Ropeginterferon alfa-2b

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Department of Medicine, the University of Hong Kong, Queen Mary Hospital

Hong Kong, Hong Kong

RECRUITING

MeSH Terms

Conditions

Primary Myelofibrosis

Condition Hierarchy (Ancestors)

Myeloproliferative DisordersBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Central Study Contacts

Harinder Gill

CONTACT

+852 22554542gillhsh@hku.hk

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 25, 2021

First Posted

August 4, 2021

Study Start

September 1, 2021

Primary Completion

July 31, 2025

Study Completion

December 31, 2025

Last Updated

October 6, 2022

Record last verified: 2022-10

Data Sharing

IPD Sharing
Will not share

Locations

HK(1)