Clinical trial NCT04226950: Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Childhood and Adolescent Essential Thrombocythemia
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Essential Thrombocytopenia
Keywords: Essential Thrombocythemia
Childhood
Interferon Alfa
Pegylated Interferon Alfa-2b
Adolescent
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2020-01-13
Enrollment: 40
Sex: ALL
Maximum age (years): 19
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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

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.

Eligibility criteria: Inclusion Criteria:

* \<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

Exclusion Criteria:

* 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
* 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

Primary outcomes:
measure: Change in platelet count
timeFrame: From the start of study treatment (Day 1) up to the end of month 12
Secondary outcomes:
measure: The complete hematologic response rates
timeFrame: From the start of study treatment (Day 1) up to the end of month 12
measure: Time to response in platelet count
timeFrame: From the start of study treatment (Day 1) up to the end of month 12
measure: Impact of therapy on key biomarkers
timeFrame: From the start of study treatment (Day 1) up to the end of month 12
measure: Incidence of major cardiovascular and thrombotic events
timeFrame: From the start of study treatment (Day 1) up to the end of month 12
measure: Incidence of development of myelodysplastic disorders, myelofibrosis, or leukemic transformation.
timeFrame: From the start of study treatment (Day 1) up to the end of month 12
measure: Change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score
timeFrame: 12 months
measure: Specific pre-defined toxicity
timeFrame: From the start of study treatment (Day 1) up to the end of month 12
measure: Impact of therapy on bone marrow histopathology (selectable)
timeFrame: From the start of study treatment (Day 1) up to the end of month 12
measure: Impact of therapy on cytogenetic abnormalities (selectable)
timeFrame: From the start of study treatment (Day 1) up to the end of month 12
measure: Death while on active treatment or observation following end of treatment
timeFrame: From the start of study treatment (Day 1) up to the end of month 12
measure: Change in platelet count
timeFrame: From the start of study treatment (Day 1) up to the end of month 12
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Institute of Hematology & Blood Diseases Hospital
state: Tianjin
Study officials:
affiliation: Institute of Hematology & Blood Diseases Hospital, China
name: Lei Zhang, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Institute of Hematology & Blood Diseases Hospital, China
name: Rongfeng Fu
role: PRINCIPAL_INVESTIGATOR
References: