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Essential ThrombocythaemiaPhase 3NCT06514807

A Study to Evaluate the Efficacy and Safety of Ropeginterferon Alfa-2b in Essential Thrombocythaemia Patients

A Phase 3 study testing Ropeginterferon alfa-2b (BESREMi®) in people with Essential Thrombocythaemia, run by AOP Orphan Pharmaceuticals AG at 36 sites in 10 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The primary objective of this study is to assess the efficacy of ropeginterferon alfa-2b in patients with ET who need cytoreductive treatment but are intolerant or refractory to, and/or ineligible for cytoreductive treatments approved and available for the treatment of ET (i.e., HU, ANA, BUS, and PB, when they are… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Essential Thrombocythaemia
What is being tested?
Ropeginterferon alfa-2b (BESREMi®)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
117 participants
Where
36 sitesin 10 countries
Study dates
4 Dec 2023to 15 Mar 2028
Phase
Phase 3
Are there results?
Not yet: the study is under way

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

  • Written informed consent obtained from the patient and ability for the patient to comply with the requirements of the study.
  • Male or female patients ≥ 18 years old
  • Patients diagnosed with ET according to the World Health Organization (WHO) 2016 criteria (with a bone marrow biopsy test result not more than 5 years old) who need cytoreductive treatment but are intolerant or refractory to, and/or ineligible for all cytoreductive treatments approved for the treatment of ET (i.e., HU, ANA, BUS, and PB1).
  • Patients resistant/intolerant to HU must have documented resistance/intolerance as defined by modified ELN criteria (Barosi, et al. 2007), whereby at least one of the following criteria is met:
  • Platelet count >600 x 109/L at ≥2 g/day (or ≥2.5 g/day if patient body weight >80 kg) or maximally tolerated dose if <2 g/day or at maximum dose per local practice after at least 3 months of HU
  • Platelet count >400 x 109/L and WBC count <2.5 x 109/L at any dose and any duration of HU
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  • Platelet count >400 x 109/L and haemoglobin (Hb) <10 g/dL at any dose and any duration of HU
  • Presence of HU-related toxicities at any dose and any duration of therapy (e.g., leg ulcers, mucocutaneous manifestations, pneumonitis, or HU-related fever)
  • Patients resistant/intolerant to ANA, BUS, or PB must meet at least one of the following criteria:
  • Patient designated as non-responder according to the primary efficacy endpoint of this protocol (modified ELN criteria) after at least 3 months of treatment with the recommended dosing defined in SmPC or local practice
  • Presence of treatment-related toxicities at any dose and any duration of therapy Patients ineligible for HU: with contraindication as defined by locally available HU SmPC or designated as such by investigator due to benefit-risk concerns (e.g., patients with toxic ranges of myelosuppression, teratogenic/leukaemogenic/carcinogenic concerns, male patients of reproductive, age not willing or unable to use an effective method of contraception). Patients ineligible for ANA: with contraindication as defined by locally available ANA SmPC or designated as such by investigator due to benefit-risk concerns (e.g., cardiovascular risk factors, including heart failure, QT prolongation, the risk for progression to myelofibrosis). Patient ineligible for BUS and PB (in countries where BUS or PB is available and approved for treatment of ET): with contraindication as defined by locally available BUS/PB SmPC or designated as such by investigator due to benefit-risk concerns (e.g., teratogenic/leukaemogenic/carcinogenic concerns, male patients of reproductive age not willing or unable to use an effective method of contraception).
  • If a patient received prior cytoreductive treatment for ET, the washout period between the last dose of treatment and the first dose of the study drug must be at least 14 days, or longer. (If the washout period not completed at time of first patients screening, washout may be done after obtaining ICF during the 28-day screening phase).
  • Interferon treatment-naïve
  • Adequate hepatic function defined as bilirubin ≤1.5 x upper limit normal (ULN), international normalised ratio ≤1.5 x ULN, albumin >3.5 g/dL, alanine aminotransferase ≤2.0 x ULN, aspartate aminotransferase ≤2.0 x ULN at screening.
  • Hospital Anxiety and Depression Scale (HADS) score 0-7 on both subscales.
  • Patient with HADS score of 8-10 inclusive on either, or both, of the subscales may be eligible following psychiatric assessment that excludes clinical significance of the observed symptoms in the context of potential treatment with an interferon alfa.

Who cannot join

  • Any patient requiring a legally authorised representative
  • Any hypersensitivity to IFN-α or to any of the drug excipients
  • Pre-existing thyroid disease, if not in remission or not controlled with conventional treatment
  • Existence of, or history of severe psychiatric disorders, particularly severe depression, suicidal ideation or suicide attempt
  • Severe cardiovascular disease (i.e., uncontrolled hypertension, congestive heart failure (≥ NYHA class 2), serious cardiac arrhythmia, significant coronary artery stenosis, unstable angina) or recent stroke or myocardial infarction or pulmonary hypertension
  • Patients with diabetes mellitus that cannot be effectively controlled by medicinal products
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  • History or presence of autoimmune disease (excluding well-controlled Hashimoto's disease)
  • Immunosuppressed transplant recipients
  • Concomitant treatment with telbivudine
  • Decompensated cirrhosis of the liver (Child-Pugh B or C)
  • End stage renal disease (GFR <15 mL/min)
  • Symptomatic splenomegaly (per the investigator's judgement)
  • Patients with any other significant medical conditions that, in the opinion of the Investigator, would compromise the results of the study or may impair compliance with the requirements of the protocol, including but not limited to:
  • History of any malignancy within 5 years (except Stage 0 chronic lymphocytic leukaemia, basal cell, squamous cell, and superficial melanoma)
  • Infections with systemic manifestations (e.g., bacterial, fungal, or human immunodeficiency virus [HIV], except hepatitis B [HBV] and/or hepatitis C [HCV], at screening)
  • Evidence of severe retinopathy (e.g., cytomegalovirus retinitis, macular degeneration) or clinically relevant ophthalmological disorder (due to diabetes mellitus or hypertension)
  • History of alcohol or drug abuse within the last year
  • Use of any investigational drug <4 weeks prior to the first dose of study drug, or ongoing effects/symptoms due to prior administration of any investigational agent
  • HADS score of 11 or higher on either, or both, of the subscales, and /or development or worsening of the clinically significant depression or suicidal thoughts
  • Pregnant patients or breastfeeding patients or females of childbearing potential not willing to comply with contraceptive requirements as described in Section 16.1.4

Trial locations and contacts

36 sites in 10 countries.

36 sites in 10 countries

Germany6 sites

  • University Hospital Aachen, Clinic of Oncology, Hematology and Stem Cell Transplantation (Medical Clinic IV)

    Aachen, 52074

  • University Hospital Halle (Saale), Department of Internal Medicine IV - Hematology and Oncology

    Halle, 06120

  • Hannover Medical School, Clinic for haematology, haemostaseology, oncology and stem cell transplantation

    Hanover, 30625

  • University Hospital Mannheim, Medical Clinic III, Hematology and Internistic Oncology

    Mannheim, 68167

  • Johannes Wesling Hospital Minden, Department of Oncology and Hematology

    Minden, 32429

  • University Hospital Ulm, Center for Internal Medicine, Clinic of Internal Medicine III, Department of Hematology, Oncology, Rheumatology, Infectious Diseases

    Ulm, 89081

Other trials in Germany

Italy5 sites

  • Polyclinic S. Orsola-Malpighi

    Bologna, 40138

  • Careggi University Hospital, Department of Hematology

    Florence, 50134

  • Umberto I Polyclinic of Rome

    Rome, 00161

  • University Polyclinic Foundation "Agostino Gemelli" - IRCCS, Service of Hematology

    Rome, 00168

  • University Hospital City of Health and Science of Turin - Hospital Molinette, Complex Structure of Hematology - U

    Turin, 10126

Other trials in Italy

Austria4 sites

  • University Hospital Graz, Department of Internal Medicine, Clinical Department of Hematology

    Graz, Styria, 8036

  • Medical University Innsbruck, Department of Internal Medicine V (Hematology and Oncology)

    Innsbruck, Tyrol, 6020

  • Ordensklinikum Linz GmbH Elisabethinen Hospital, Department of Internal Medicine I - Hemato-Oncology

    Linz, Upper Austria, 4020

  • Medical University Vienna, Department of Internal Medicine I, Clinical Department of Hematology and Hemostaseology

    Vienna

Other trials in Austria

Poland4 sites

  • University Teaching Centre, Hematology and Transplantology Clinic

    Gdansk, 80-952

  • Pratia Oncology Katowice

    Katowice, 40-519

  • Independent Public Healthcare Facility University Hospital in Krakow, Teaching Unit of the Hematology Department

    Krakow, 31-501

  • Nicolaus Copernicus Provincial Multispecialty Oncology and Traumatology Center in Lodz, Department of Hematooncology with Subdivision of Daytime Chemotherapy

    Lodz, 93-513

Other trials in Poland

Romania4 sites

  • Onco Card Srl

    Brasov, 500052

  • "Prof. Dr. Ion Chiricuta" Institute of Oncology, Hematology Department

    Cluj-Napoca, 400015

  • Fundeni Clinical Institute, Center for Hematology and Bone Marrow Transplantation

    Crişan, 022328

  • Iasi Regional Institute of Oncology, Department of Hematology

    Iași, 700483

Other trials in Romania

Spain4 sites

  • University Hospital Germans Trias i Pujol

    Badalona, 08916

  • Hospital Clinic of Barcelona, Department of Hematology

    Barcelona, 08036

  • University Hospital Ramon y Cajal, Hematology Service

    Madrid, 28034

  • Morales Meseguer University General Hospital

    Murcia, 30008

Other trials in Spain

France3 sites

  • Saint-Louis Hospital, Department of Adult Hematology

    Paris, 75010

  • Bordeaux University Hospital, Haut-Leveque Hospital

    Pessac, 33600

  • Centre Hospitalier Universitaire De Poitiers

    Poitiers, Poitiers, 86000

Other trials in France

Czechia2 sites

  • University Hospital Brno, Clinic of Internal Medicine - Hematology and Oncology

    Brno, 625 00

  • University Hospital Kralovske Vinohrady, Clinic of Internal Hematology

    Prague, 10000

Other trials in Czechia

Greece2 sites

  • General Hospital of Athens Alexandra, Therapeutic Clinic, Department of Therapeutics

    Athens, 11528

  • University General Hospital "Attikon"

    Athens, PC 12462

Other trials in Greece

Hungary2 sites

  • Semmelweis University, Department of Internal Medicine and Haematology, Division of Hematology

    Budapest, H-1088

  • University of Debrecen Clinical Center, Clinic of Internal Medicine, Department of Hematology

    Debrecen, 4032

Other trials in Hungary

About this study

Official title: A Phase III, Single Arm, Multicentre Study to Evaluate the Efficacy and Safety of Ropeginterferon Alfa-2b in Essential Thrombocythaemia Patients Who Are Intolerant or Refractory to or Not Eligible for Other Cytoreductive Treatments

The primary objective of this study is to assess the efficacy of ropeginterferon alfa-2b in patients with ET who need cytoreductive treatment but are intolerant or refractory to, and/or ineligible for cytoreductive treatments approved and available for the treatment of ET (i.e., HU, ANA, BUS, and PB, when they are available and approved for ET treatment).

Ropeginterferon alfa-2b is currently the only interferon authorised as a cytoreductive treatment of a myeloproliferative neoplasm (MPN), and the long-term treatment data from its comprehensive clinical development program show its efficacy in the induction of haematologic remission, resolution of disease-associated symptoms, disease-modifying effect, as well as its favourable safety profile (Gisslinger et al., 2020; Kiladjian et al. 2022).

Available clinical data and experience show that ropeginterferon alfa-2b normalises various haematological parameters, including platelets. In addition, suppression of the malignant clone causing ET may be achieved, at least after long-term treatment, which is expected to possibly defer the onset of, or avoid long-term sequelae of ET.

Being tested
Ropeginterferon alfa-2b (BESREMi®)
Keywords
Ropeginterferon alfa-2b, essential thrombocythaemia, intolerant to cytoreductive treatment, refractory to cytoreductive treatment, not eligible for other cytoreductive treatments, JAK2, CALR, MPL mutation status, BESREMi, Thrombocythaemia
Medical subject headings
Thrombocythemia, Essential, Thrombocytosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Ropeginterferon alfa-2b (BESREMi®)Drug

    Ropeginterferon alfa-2b 250 micrograms/0.5 mL or 500 micrograms/0.5 ml solution for injection in pre-filled pen.

Study groups in NCT06514807
Study groupRoleWhat they receive
Ropeginterferon alfa-2b (tradename BESREMi®)ExperimentalRopeginterferon alfa-2b (tradename BESREMi®) 250 micrograms/0.5 mL or 500 micrograms/0.5 ml solution for injection in pre-filled pen. Ropeginterferon alfa-2b will be administered subcutaneously every 2 weeks at a dose of 125 µg / 250 µg / 500 µg per injection (depends on the optimal disease response) for up to 36 months of treatment.
Receives: Drug: Ropeginterferon alfa-2b (BESREMi®)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Durable (for at least 3 months) peripheral blood count remission

    PLTs ≤400 x 109/L AND WBC <10 x 109/L

    Measured: At month 12

  2. Absence of haemorrhagic or thrombotic events and absence of disease progression*

    *Progression is defined as conversion from asymptomatic to symptomatic splenomegaly or clinically relevant progression of spleen size at the Investigator's discretion, respectively.

    Measured: At month 12

  3. Absence or Non-progression* in disease-related signs

    *Progression is defined as conversion from asymptomatic to symptomatic splenomegaly or clinically relevant progression of spleen size at the Investigator's discretion, respectively.

    Measured: At month 12

  4. Durable (for at least 3 months) large symptoms improvement or maintenance of non-progression based on the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)#

    # Large symptoms improvement for ET patients is defined as follows:

    • Baseline TSS ≥20: 10-points reduction in TSS
    • Baseline TSS 15-19, inclusive: 5-points reduction in TSS
    • Baseline TSS 10-14, inclusive: TSS decreases to ≤10
    • Baseline TSS <10: TSS stays <10

    Measured: At month 12

Secondary outcomes (13)

  1. Response at month 9, 18, 24, 30 and 36

    Measured: Month 9, 18, 24, 30 and 36

  2. Longitudinal changes in the ELN response rates over 12 months

    Measured: up to 36 months

  3. Time to first response (as defined by ELN criteria)

    Measured: up to 36 months

  4. Duration of first response (as defined by ELN criteria)

    Measured: up to 36 months

  5. Duration of first durable response (as defined by ELN criteria)

    Measured: up to 36 months

  6. Time to first peripheral blood count remission response

    Measured: up to 36 months

  7. Duration of first durable peripheral blood count remission response

    Measured: up to 36 months

  8. Occurrence of thromboembolic and bleeding events response

    Measured: up to 36 months

  9. Occurrence of disease progression response

    Measured: up to 36 months

  10. Symptomatic improvement assessed by EQ-5D-5L questionnaire response

    Measured: up to 36 months

  11. Symptomatic improvement assessed by the 10-item MPN-SAF TSS

    Measured: up to 36 months

  12. Change in inflammation markers over time

    Measured: up to 36 months

  13. Change in CALR, MPL, or JAK2 allelic burden over time

    Measured: up to 36 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Jul 2024
  2. Study start4 Dec 2023
  3. Primary completion15 Mar 2028 (estimated)
  4. Study completion15 Mar 2028 (estimated)

Resources

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 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
Open-label, single-arm, multicentre, descriptive study of ropeginterferon alfa-2b in essential thrombocythaemia patients
Masking
None (open label)
Enrolment
117 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06514807
Sponsor's protocol ID
ROP-ET
Status last verified
Dec 2024
First submitted
11 Jul 2024
Last updated
19 Dec 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Ropeginterferon alfa-2b (BESREMi®) in people with Essential Thrombocythaemia, run by AOP Orphan Pharmaceuticals AG at 36 sites in 10 countries.

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 16 inclusion and 20 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: under way, not looking for participants, as of the sponsor's last update on 19 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 36 sites in Germany, Italy, Austria, Poland, Romania and 5 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Durable (for at least 3 months) peripheral blood count remission (time frame: At month 12). The study lists 4 primary outcomes in all. 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 NCT06514807.

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 19 Dec 2024.

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