Thrombocytopenia, Immune
Conditions
Keywords
Immune Thrombocytopenia
Brief summary
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.
Interventions
DRUGQL0911
The starting dose of QL0911 is 1 µg/kg administered weekly by subcutaneous injection. Participants will return to the clinic weekly to provide platelet counts and undergo dose titrations under the supervision of the treating physician. Weekly dose increases will continue in increments of 1 µg/kg up to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10\^9/L. Dose adjustment will be allowed during the treatment period to maintain a platelet count between ≥ 50 x 10\^9/L and ≤ 200 x 10\^9/L.
DRUGPlacebo
Matching placebo administered by subcutaneous injection.
Sponsors
Qilu Pharmaceutical Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
1 Years to 17 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With a Durable Platelet Response | Week 18 to week 25 | A participant with durable platelet response was defined as achieving at least 6 weekly platelet counts of ≥ 50 x 10\^9/L from week 18 to week 25. If a platelet count from a participant was not available (missing) in a certain week, that week was imputed as non-response for that participant. Platelet counts were not deemed as a positive response for 4 weeks after the administration of rescue medication. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With an Overall Platelet Response | 24 weeks | Overall platelet response is defined as either a durable platelet response or transient platelet response. |
| The proportion of subjects with a weekly platelet count ≥ 30 × 10^9/Land at least twice the baseline platelet count without bleeding during the double-blind period of 24 weeks. | 24 weeks | The proportion of subjects with a weekly platelet count ≥ 30 × 10\^9/Land at least twice the baseline platelet count without bleeding during the double-blind period of 24 weeks. |
| Number of Weeks With Platelet Response | 24 weeks | Number of weeks with platelet counts ≥ 50 x 10\^9/L. |
| Percentage of Participants Who Received Rescue Medication During the Treatment Period. | 24 weeks | Rescue medication is any medication that is intended to increase platelet counts or prevent bleeding. |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Up to Week 38 | — |
| Number of Participants With Antidrug Antibodies (ADAs) , Anti endogenous TPO antibody and Neutralizing Antibodies (Nab)； | Up to Week 38 | — |
Contacts
CONTACTRun hui Wu, Doctorate
Outcome results
None listed