This is a multicenter, randomized, open-label Phase 2 clinical study. It is aimed to enroll 27 essential thrombocytopenia (ET) patients who are resistant to or intolerant of hydroxyurea(HU). Eligible patients will be randomized to receive either Peginterferon α-2b 135 mcg or Peginterferon α-2b 180 mcg at a ratio of 1:2, and all subjects will go through a target treatment period (Weeks 1 \~ Week 48), an extension treatment period (Weeks 49 \~ Week 96) and a follow-up period (Weeks 97 \~ Week 100). Pharmacokinetics, safety, efficacy will be evaluated.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
27
Peginterferon α-2b injection, 135 mcg, s.c., once a week, during the targeted treatment period (the first 48 week), peginterferon α-2b dose is depended on the patient's response and tolerability during the extension treatment (week 49 to week 96).
Peginterferon α-2b injection, 180 mcg, s.c., once a week, during the targeted treatment period (the first 48 week), peginterferon α-2b dose is depended on the patient's response and tolerability during the extension treatment (week 49 to week 96).
Peking Union Hospital, Chinese Academy of Medical Sciences
Beijing, China
RECRUITINGPeking University People's Hospital
Beijing, China
RECRUITINGUnion Hospital affiliated to Fujian Medical University
Fujian, China
RECRUITINGNanfang Hospital, Southern Medical University
Guangzhou, China
RECRUITINGHarbin First Hospital
Harbin, China
RECRUITINGHenan Cancer Hospital
Henan, China
RECRUITINGRuijin Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, China
RECRUITINGThe First Affiliated Hospital of Zhejiang University School of Medicine
Zhejiang, China
RECRUITINGMaximum concentration (Cmax)
Time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Time to maximum concentration (Tmax)
Time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Area under the plasma concentration-time curve
Time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Apparent volume of distribution after oral administration (Vz/f)
Time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Apparent plasma clearance (CL/F)
Time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Plasma elimination half-life (t1/2)
Time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Relationship between exposure and the effect (desired-effectiveness or undesirable-toxicity) in a pharmacokinetic model and pharmacodynamic model.
Time frame: up to 96 weeks.
Rate of complete hematological remission.
Time frame: Week 24, 36, 48, 60, 72, 84, 96.
Platelet counts change from baseline.
Time frame: Week 12, 24, 36, 48, 60, 72, 84, 96.
White blood cell counts change from baseline.
Time frame: Week 12, 24, 36, 48, 60, 72, 84, 96.
Complete remission rate.
Time frame: Week 24, 36, 48, 60, 72, 84, 96.
Time to complete remission from baseline.
Time frame: Week 48, 96.
Duration of complete remission.
Time frame: Week 48, 96.
Remission rate of bone marrow.
Time frame: Week 48, 96.
Incidence of disease progression.
Time frame: Week 48, 96.
Change of JAK2V617F mutations load from baseline.
JAK2V617F mutations were quantitatively detected using next-generation sequencing
Time frame: Week 48, 96.
Change of CALR mutations load from baseline.
CALR mutations were quantitatively detected using next-generation sequencing
Time frame: Week 48, 96.
Change of MPL mutations load from baseline.
MPL mutations were quantitatively detected using next-generation sequencing
Time frame: Week 48, 96.
Change of MPN-SAF TSS scores from baseline
Time frame: Week 12, 24, 36, 48, 60, 72, 84,96.
Change of spleen size from baseline
The maximum length of the spleen was measured by ultrasound.
Time frame: Week 12, 24, 36, 48, 60, 72, 84, 96.
Rate of complete hematological remission maintenance in patients received dose-reduction extension therapy
Time frame: Week 96.
Duration of complete hematological remission in patients received dose-reduction extension therapy
Time frame: Week 96.
Change of 3-level Version of EuroQol Five Dimensions(EQ-5D-3L) scores from baseline.
Time frame: Week 12, 24, 36, 48, 60, 72, 84, 96.
Incidence of thrombotic and bleeding events.
Time frame: through study completion, an average of 2 year
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