Efficacy and Safety of QL0911 in Adult Patients With Chronic Primary Immune Thrombocytopenia

Efficacy and Safety of QL0911 in Adult Patients With Chronic Primary Immune Thrombocytopenia: A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase III Trial

QL0911, a recombinant human thrombopoietin mimetic peptide-Fc fusion protein for injection, is a romiplostim (Nplate®) biosimilar for the treatment of primary immune thrombocytopenia (ITP). This phase III study aimed to assess the efficacy and safety of QL0911 in adult patients with primary chronic ITP.

Study Overview

• Status: Completed
• Conditions: Primary Immune Thrombocytopenia
• Intervention / Treatment: Drug: QL0911; Drug: Placebo comparator

Detailed Description

This study consisted of a randomized, double-blind, placebo-controlled, 26-week treatment period, sequentially followed by an open-label, single-arm, 12-week treatment period, and an additional 4-week safety follow-up period.

• Study Type: Interventional
• Enrollment (Actual): 216
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Shandong, China
    • Qilu Hospital Of Shandong University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Aged ≥18 years old;
• Diagnosed primary ITP for at least 12 months;
• Had received at least one first-line ITP treatment with no response or recurrence after treatment;
• Had a platelet count <30×10^9/L within 48 hours before the first dose;
• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2;
• Fully understand and comply with the requirements of this study, and voluntarily sign the informed consent form.

Exclusion Criteria:

• Had a history of bone marrow stem cell abnormalities or myelodysplastic syndrome other than ITP-specific changes.
• Had arterial thrombosis, or venous thromboembolism; severe cardiovascular diseases; malignant tumors; secondary thrombocytopenia caused by autoimmune diseases.
• Underwent splenectomy within 12 weeks before the first dose;
• Had received ITP treatments (including rescue treatment) within 2 weeks before the first dose;
• Had received romiplostim (Nplate®) or eltrombopag (Revolade®), rhTPO or other agents that stimulate TPO receptors (also known as c-Mpl), and hematopoietic growth factors (HGFs) within 4 weeks before the first dose;
• Had received antineoplastic agents within 8 weeks before the first administration, but when treating ITP with hypomethylating agents (HMA) such as decitabine, a 4-week washout period was acceptable, as judged by the investigator;
• Had received antibody-based therapies within 14 weeks before the first dose; 8) had serum creatinine or total bilirubin >1.5 upper limit of normal (ULN), alanine transaminase (ALT) or aspartate transaminase (AST) >3 ULN, hemoglobin < 100g/L, absolute neutrophil count <1.5x10^9/L;
• Had prothrombin time (PT) or prothrombin time-international normalized ratio (PT-INR) or activated partial thromboplastin time (APTT) exceeded 20% of the reference range of normal values.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo comparator; The study in a 2:1 randomization ratio(72 subjects to Placebo ). Qilu investigational product (QL0911 or placebo) will be administered in the clinic by a qualified healthcare provider as a subcutaneous injection.
Experimental: QL0911	Drug: QL0911; The study in a 2:1 randomization ratio(144 subjects to QL0911). Qilu investigational product (QL0911 or placebo) will be administered in the clinic by a qualified healthcare provider as a subcutaneous injection.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
proportion of patients achieving durable platelet response at week 24 during the double-blind treatment period	24weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
proportion of patients with weekly platelet responses within 24 weeks of treatment;	24weeks
Proportion of patients who achieved platelet count ≥ 30 × 10^9/L at least a two-fold increase from baseline platelet count without bleeding during the 24-week double-blind period;	24weeks

Collaborators and Investigators

• Sponsor: Qilu Pharmaceutical Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): October 18, 2019
• Primary Completion (Actual): December 13, 2021
• Study Completion (Actual): December 16, 2021

Study Registration Dates

• First Submitted: November 10, 2022
• First Submitted That Met QC Criteria: November 10, 2022
• First Posted (Actual): November 18, 2022

Study Record Updates

• Last Update Posted (Actual): November 23, 2022
• Last Update Submitted That Met QC Criteria: November 20, 2022
• Last Verified: November 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
• Other Study ID Numbers: QL0911-003

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No