Essential ThrombocytopeniaPhase 2NCT04226950
Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Childhood and Adolescent Essential Thrombocythemia
A Phase 2 study testing Recombinant Interferon Alpha and Pegylated interferon alfa-2b in people with Essential Thrombocytopenia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
Objectives: To compare the efficacy and safety in childhood and adolescent patients (<20 years) diagnosed as essential thrombocythemia treated with the Pegylated Interferon Alfa-2b vs. Interferon Alfa. Study Design: A prospective, open-label, nonrandomized, single-center clinical trial Read more about this study
- Who is it for?
- Ages Up to 19 years
- Conditions the study is for
- Essential Thrombocytopenia
- What is being tested?
- Recombinant Interferon Alpha and Pegylated interferon alfa-2b
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 10 Jan 2020to 20 Nov 2024
- 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 Up to 19 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- <20 years old
- Male or Female
- Diagnosis of essential thrombocythemia according to the 2016 WHO criteria.
- Platelet count ≥ 450 × 109 / L for more than 6 months（If the patient has JAK2 V617F, CALR or MPL gene mutation, the history may be less than 6 months）
- Platelet count ≥ 1000 × 109 / L or other therapeutic indications at screening.
- The guardians has provided written informed consent prior to enrollment
Who cannot join
- Known to meet the criteria for primary myelofibrosis or polycythemia vera by 2016 WHO criteria
- Presence of any life-threatening co-morbidity
- Secondary thrombocytosis
- Familial thrombocytosis
- Resistance, or intolerance, or any contraindications to interferon
- Interferon is used in the past 1 month before enrollment
Show all 20Show fewer
- Patients with previous or present thrombosis or active bleeding
- WBC<4× 109 / L
- HGB<110g/L
- Poor control of thyroid dysfunction
- Patients with a prior malignancy within the last 3 years
- Patients with severe cardiac or pulmonary dysfunction
- Severe renal damage (creatinine clearance < 30 ml / min)
- Severe liver dysfunction (ALT or AST > 2.5×ULN)
- Patients diagnosed as diabetes with poor control
- Patients with hepatitis B virus, hepatitis C virus replication or HIV infection
- Patients with a history of drug / alcohol abuse (within 2 years before the study)
- Patients that have participated in other experimental researches within one month before enrollment
- History of psychiatric disorder
- Any other circumstances that the investigator considers that the patient is not suitable to participate in the trial
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
Lei Zhang, MD, +862223909240, zhanglei1@ihcams.ac.cn
About this study
Official title: A Prospective, Single-center Clinical Trial of Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in the Treatment of Childhood and Adolescent Essential Thrombocythemia
Objectives: To compare the efficacy and safety in childhood and adolescent patients (<20 years) diagnosed as essential thrombocythemia treated with the Pegylated Interferon Alfa-2b vs. Interferon Alfa.
Study Design: A prospective, open-label, nonrandomized, single-center clinical trial
Read the detailed description
This is a prospective, open-label, nonrandomized, single-center clinical trial between Interferon Alfa and Pegylated Interferon Alfa-2b in childhood and adolescent essential thrombocythemia (<20 years).
Patients will be divided into the following two treatment groups: 1. Recombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers; 2. Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2).
The current drug therapies and possible risks of Pegylated Interferon Alfa-2b and Interferon Alfa in the treatment of childhood and adolescent essential thrombocythemia will be fully introduced to the guardians (childhood patients) or patients (adolescent patients) by the researchers. Then the patients will be divided into one of the two groups according to the guardians' (childhood patients) or patients' (adolescent patients) will.
The dosage will be adjusted according to the results of laboratory examinations and patient tolerance. The patient will be transferred to the other group if intolerance or resistance occurs.
- Conditions
- Essential Thrombocytopenia
- Being tested
- Recombinant Interferon Alpha, Pegylated interferon alfa-2b
- Keywords
- Essential Thrombocythemia, Childhood, Interferon Alfa, Pegylated Interferon Alfa-2b, Adolescent
- Medical subject headings
- Thrombocythemia, Essential
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Interferon AlphaDrug
Recombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers;
Pegylated interferon alfa-2bDrug
Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2).
| Study group | Role | What they receive |
|---|---|---|
| Recombinant Interferon Alpha | Active comparator | Recombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers. Receives: Drug: Recombinant Interferon Alpha |
| Pegylated Interferon Alfa-2b | Experimental | Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2). Receives: Drug: Pegylated interferon alfa-2b |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in platelet count
Proportion of subjects with a continuous platelet count ≤600×109/L or decrease ≥50% (<1000×109/L ) (at least 12 weeks) from baseline during treatment will be evaluated.
Measured: From the start of study treatment (Day 1) up to the end of month 12
Secondary outcomes (11)
The complete hematologic response rates
To compare the complete hematologic response rates between different treatment groups
Measured: From the start of study treatment (Day 1) up to the end of month 12
Time to response in platelet count
Time to response in platelet count (<600×109/L) between different treatment groups
Measured: From the start of study treatment (Day 1) up to the end of month 12
Impact of therapy on key biomarkers
To compare the proportion of subjects that display change on key biomarkers of the disease- JAK2V617F, CALR, MPL mutations.
Measured: From the start of study treatment (Day 1) up to the end of month 12
Incidence of major cardiovascular and thrombotic events
To estimate incidence of major cardiovascular and thrombotic events (defined as cardiovascular death, myocardial infarction, stroke, transient ischemic attack, pulmonary embolism, Budd Chiari syndrome, deep vein thrombosis, and any other clinically relevant thrombotic event) while on active treatment or observation following end of treatment between different treatment groups
Measured: From the start of study treatment (Day 1) up to the end of month 12
Incidence of development of myelodysplastic disorders, myelofibrosis, or leukemic transformation.
To estimate incidence of development of myelodysplastic disorders, myelofibrosis, or leukemic transformation between different treatment groups
Measured: From the start of study treatment (Day 1) up to the end of month 12
Change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score
To compare the proportion of subjects that display change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score (0-100 scores, higher scores mean a worse outcome) between different treatment groups.
Measured: 12 months
Specific pre-defined toxicity
To compare incidence of specific pre-defined toxicity including fatigue, flu-like symptoms, dizziness, injection site necrosis, dyspnea, pain, depression, blurred Vision, insomnia, anorexia, weight Loss, weakness, pruritis, sweating, fever, decreased Libido, hot Flashes, flushing.
Measured: From the start of study treatment (Day 1) up to the end of month 12
Impact of therapy on bone marrow histopathology (selectable)
To compare the proportion of subjects that display change on bone marrow histopathology
Measured: From the start of study treatment (Day 1) up to the end of month 12
Impact of therapy on cytogenetic abnormalities (selectable)
To compare the proportion of subjects that display change on cytogenetic abnormalities.
Measured: From the start of study treatment (Day 1) up to the end of month 12
Death while on active treatment or observation following end of treatment
To compare the incidence of death while on active treatment or observation following end of treatment
Measured: From the start of study treatment (Day 1) up to the end of month 12
Change in platelet count
Proportion of subjects with a continuous platelet count <1000×109/L in those with platelet count ≥1000×109/L before treatment (at least 12 weeks) will be evaluated.
Measured: From the start of study treatment (Day 1) up to the end of month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2020
- Study start10 Jan 2020
- Primary completion20 Oct 2024 (estimated)
- Study completion20 Nov 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Essential ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, MD, Principal investigator, Institute of Hematology & Blood Diseases Hospital, ChinaRongfeng Fu, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04226950
- Sponsor's protocol ID
- IIT2019001
- Status last verified
- Apr 2024
- First submitted
- 10 Dec 2019
- Last updated
- 3 Jul 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Single-center Clinical Trial of Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in the Treatment of Childhood and Adolescent Essential Thrombocythemia
- ClinicalTrials.gov ID
- NCT04226950
- Acronym / sponsor ID
- IIT2019001
- Conditions
- Essential Thrombocytopenia
- Keywords
- Essential Thrombocythemia, Childhood, Interferon Alfa, Pegylated Interferon Alfa-2b, Adolescent
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 10 Jan 2020 (actual)
- Primary completion
- 20 Oct 2024 (estimated)
- Study completion
- 20 Nov 2024 (estimated)
- First posted
- 13 Jan 2020 (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.
Objectives: To compare the efficacy and safety in childhood and adolescent patients (<20 years) diagnosed as essential thrombocythemia treated with the Pegylated Interferon Alfa-2b vs. Interferon Alfa.
Study Design: A prospective, open-label, nonrandomized, single-center clinical trial
Detailed description
This is a prospective, open-label, nonrandomized, single-center clinical trial between Interferon Alfa and Pegylated Interferon Alfa-2b in childhood and adolescent essential thrombocythemia (<20 years).
Patients will be divided into the following two treatment groups: 1. Recombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers; 2. Pegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2).
The current drug therapies and possible risks of Pegylated Interferon Alfa-2b and Interferon Alfa in the treatment of childhood and adolescent essential thrombocythemia will be fully introduced to the guardians (childhood patients) or patients (adolescent patients) by the researchers. Then the patients will be divided into one of the two groups according to the guardians' (childhood patients) or patients' (adolescent patients) will.
The dosage will be adjusted according to the results of laboratory examinations and patient tolerance. The patient will be transferred to the other group if intolerance or resistance occurs.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Recombinant Interferon Alpha | Active comparator | Drug: Recombinant Interferon AlphaRecombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers. |
| Pegylated Interferon Alfa-2b | Experimental | Drug: Pegylated interferon alfa-2bPegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2). |
Who may qualify
- Ages
- Up to 19 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- <20 years old
- Male or Female
- Diagnosis of essential thrombocythemia according to the 2016 WHO criteria.
- Platelet count ≥ 450 × 109 / L for more than 6 months（If the patient has JAK2 V617F, CALR or MPL gene mutation, the history may be less than 6 months）
- Platelet count ≥ 1000 × 109 / L or other therapeutic indications at screening.
Key exclusion criteria (20 listed)
- Known to meet the criteria for primary myelofibrosis or polycythemia vera by 2016 WHO criteria
- Presence of any life-threatening co-morbidity
- Secondary thrombocytosis
- Familial thrombocytosis
- Resistance, or intolerance, or any contraindications to interferon
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 |
|---|---|---|
| Recombinant Interferon AlphaRecombinant Interferon Alpha, with an initial dose of 300 wu twice a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available, and the specific dose will be determined by the researchers; | Drug | Recombinant Interferon Alpha |
| Pegylated interferon alfa-2bPegylated Interferon Alfa-2b, with an initial dose of 135 ug once a week (body surface area < 1.73 m2) or 180 ug once a week ( body surface area≥1.73 m2). | Drug | Pegylated Interferon Alfa-2b |
Primary outcome
Change in platelet count
Time frame: From the start of study treatment (Day 1) up to the end of month 12
Proportion of subjects with a continuous platelet count ≤600×109/L or decrease ≥50% (<1000×109/L ) (at least 12 weeks) from baseline during treatment will be evaluated.
11 secondary outcomes
The complete hematologic response rates
Time frame: From the start of study treatment (Day 1) up to the end of month 12
Time to response in platelet count
Time frame: From the start of study treatment (Day 1) up to the end of month 12
Impact of therapy on key biomarkers
Time frame: From the start of study treatment (Day 1) up to the end of month 12
Incidence of major cardiovascular and thrombotic events
Time frame: From the start of study treatment (Day 1) up to the end of month 12
Incidence of development of myelodysplastic disorders, myelofibrosis, or leukemic transformation.
Time frame: From the start of study treatment (Day 1) up to the end of month 12
Change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score
Time frame: 12 months
Specific pre-defined toxicity
Time frame: From the start of study treatment (Day 1) up to the end of month 12
Impact of therapy on bone marrow histopathology (selectable)
Time frame: From the start of study treatment (Day 1) up to the end of month 12
Impact of therapy on cytogenetic abnormalities (selectable)
Time frame: From the start of study treatment (Day 1) up to the end of month 12
Death while on active treatment or observation following end of treatment
Time frame: From the start of study treatment (Day 1) up to the end of month 12
Change in platelet count
Time frame: From the start of study treatment (Day 1) up to the end of month 12
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jul 2024.
Related trials for Essential Thrombocytopenia
RecruitingPhase 2
Selinexor and Pacritinib in JAK Inhibitor-naïve MF Patients With Cytopenias
Myelofibrosis, Post-polycythemia Vera Myelofibrosis
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.
Lei Zhang, MD principal investigator
Institute of Hematology & Blood Diseases Hospital, China
Rongfeng Fu principal investigator
Institute of Hematology & Blood Diseases Hospital, China
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 Recombinant Interferon Alpha and Pegylated interferon alfa-2b in people with Essential Thrombocytopenia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
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 6 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: status not confirmed, as of the sponsor's last update on 3 Jul 2024. 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 China. 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 Rongfeng Fu, MD (+862223909009, furongfeng@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in platelet count (time frame: From the start of study treatment (Day 1) up to the end of month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Jul 2024.