Peginterferon α-2b Injection for Hydroxyurea Resistant or Intolerant ET
NCT Number: NCT06552429
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE2
Conditions: Essential Thrombocythemia
Interventions: DRUG: Peginterferon α-2b injection|DRUG: Peginterferon α-2b injection
Sponsor: Xiamen Amoytop Biotech Co., Ltd.
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 27
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-08
Completion Date: 2027-09
Locations: Peking Union Hospital, Chinese Academy of Medical Sciences, Beijing, China|Peking University People's Hospital, Beijing, China|Union Hospital affiliated to Fujian Medical University, Fujian, China|Nanfang Hospital, Southern Medical University, Guangzhou, China|Harbin First Hospital, Harbin, China|Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China|The First Affiliated Hospital of Zhejiang University School of Medicine, Zhejiang, China

Summary:
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.

Primary Outcome Measures: Maximum concentration (Cmax), week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.|Time to maximum concentration (Tmax), week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.|Area under the plasma concentration-time curve, week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.|Apparent volume of distribution after oral administration (Vz/f), week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.|Apparent plasma clearance (CL/F), week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.|Plasma elimination half-life (t1/2), 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., up to 96 weeks.
Secondary Outcome Measures: Rate of complete hematological remission., Week 24, 36, 48, 60, 72, 84, 96.|Platelet counts change from baseline., Week 12, 24, 36, 48, 60, 72, 84, 96.|White blood cell counts change from baseline., Week 12, 24, 36, 48, 60, 72, 84, 96.|Complete remission rate., Week 24, 36, 48, 60, 72, 84, 96.|Time to complete remission from baseline., Week 48, 96.|Duration of complete remission., Week 48, 96.|Remission rate of bone marrow., Week 48, 96.|Incidence of disease progression., Week 48, 96.|Change of JAK2V617F mutations load from baseline., JAK2V617F mutations were quantitatively detected using next-generation sequencing, Week 48, 96.|Change of CALR mutations load from baseline., CALR mutations were quantitatively detected using next-generation sequencing, Week 48, 96.|Change of MPL mutations load from baseline., MPL mutations were quantitatively detected using next-generation sequencing, Week 48, 96.|Change of MPN-SAF TSS scores from baseline, 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., Week 12, 24, 36, 48, 60, 72, 84, 96.|Rate of complete hematological remission maintenance in patients received dose-reduction extension therapy, Week 96.|Duration of complete hematological remission in patients received dose-reduction extension therapy, Week 96.|Change of 3-level Version of EuroQol Five Dimensions(EQ-5D-3L) scores from baseline., Week 12, 24, 36, 48, 60, 72, 84, 96.|Incidence of thrombotic and bleeding events., through study completion, an average of 2 year