Clinical trial NCT05395507: Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Adult Essential Thrombocythemia
Official title: A Prospective, Multicenter, Randomized Controlled Clinical Trial of Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in the Treatment of Adult Essential Thrombocythemia
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Essential Thrombocytopenia
Keywords: Essential Thrombocytopenia
Interferon Alfa
Pegylated Interferon Alfa-2b
Efficacy
Safety
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2022-05-27
Enrollment: 194
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Objectives: To compare the efficacy and safety in Adult patients (≥18 years) diagnosed as essential thrombocythemia treated with the Pegylated Interferon Alfa-2b vs. Interferon Alfa. Study Design: A prospective, open-label, multicenter, randomized controlled clinical trial.

Detailed description: This is a prospective, open-label, multicenter, randomized controlled clinical trial between Interferon Alfa and Pegylated Interferon Alfa-2b in adult essential thrombocythemia (≥18 years).

Patients will be randomly divided into the following two treatment groups: 1. Recombinant Interferon Alpha, with an initial dose of 300 wu three times a week. Other interferons that have been listed can be used if Recombinant Interferon Alpha (300 wu) is not available. 2. Pegylated Interferon Alfa-2b, with an initial dose of 135 ug at week 0 , and then 180 ug once a week from week 1 to week 52.

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:

* ≥18 years old.
* Male or Female.
* Diagnosis of essential thrombocythemia according to the 2016 World Health Organization criteria.
* Those who have not use interferon within 4 weeks before the first medication.
* Patients with indications for cytoreductive therapy.
* Men and women with reproductive potential, as well as all women with menopause less than 2 years, must agree to use acceptable contraceptive methods until 28 days after the last dose of study drug, and women must agree not to breastfeed during the study period.
* Voluntary written informed consent.

Exclusion Criteria:

* Resistance, or intolerance, or any contraindications to interferon.
* Patients with active thrombosis or active bleeding.
* Neutrophil count \< 1.0x10\^9/L.
* Hemoglobin \< 11g/dL for male, or \< 10g/dL for female.
* 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 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: Complete hematological remission (CHR) rates
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52
Secondary outcomes:
measure: CHR rates at week 24 and 36
timeFrame: From the start of study treatment (Week 0) up to the end of Week 24 and Week 36, respectively
measure: Time to CHR
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: The proportion of patients crossed to the contralateral group
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: The CHR rates after crossover
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: Impact of therapy on driver mutations
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: Impact of therapy on non-driver mutations
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: Change of splenomegaly
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: Change of bone marrow pathology
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: The incidence of major thrombotic events
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: The incidence of major bleeding events
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: The incidence of progressing to bone marrow fibrosis
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: The incidence of progressing to acute leukemia
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: Change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: Change of quality of life
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: Change of microcirculation disturbance
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
measure: Specific pre-defined toxicity
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
Other outcomes:
measure: Changes of immune cell subgroups
timeFrame: From the start of study treatment (Week 0) up to the end of Week 52.
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
affiliation: Tianjin Medical University General Hospital
name: Rong Fu
role: PRINCIPAL_INVESTIGATOR
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