Safety and Efficacy Study of QL0911 to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients

A Phase III, Randomized, Double-Blind, Placebo-Controlled Study of QL0911 in Pediatric Patients With Primary Immune Thrombocytopenia.

The purpose of this study is to evaluate efficacy and of safety QL0911 in the treatment of thrombocytopenia in pediatric patients with previously treated chronic ITP.

Inclusion Criteria:

1. Age ≥ 1 years old and < 18 years old.
2. Diagnosed primary ITP for at least 12 months;
3. Had received at least one first-line ITP treatment with no response or recurrence after treatment;
4. Had a platelet count <30×10^9/L within 48 hours before the first dose;
5. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2;
6. Patients of child-bearing potential must agree to use effective contraception during the study and for 3 months following the last dose of study treatment.

Exclusion Criteria:

1. Had a history of bone marrow stem cell abnormalities or myelodysplastic syndrome other than ITP-specific changes.
2. Underwent splenectomy within 12 weeks before the first dose;
3. Had received ITP treatments (including rescue treatment) within 2 weeks before the first dose;
4. Had received romiplostim (Nplate®) or eltrombopag (Revolade®), rhTPO or other agents that stimulate TPO receptors (also known as c-Mpl)within 4 weeks before the first dose;
5. Had received antibody-based therapies within 14 weeks before the first dose.
6. Patients with concurrent or past malignant disease.
7. Serum creatinine or total bilirubin >1.5*ULN) alanine transaminase (ALT) or aspartate transaminase (AST) >3* ULN.
8. Had received antibody-based therapies within 14 weeks before the first dose.
9. 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.