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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| QL0911 | Experimental | Participants received once weekly subcutaneous QL0911 at a starting dose of 1 µg/kg; weekly dose increases continued in increments of 1 µg/kg/week to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. |
|
| placebo | Placebo Comparator | Participants received weekly subcutaneous placebo. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| QL0911 | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With a Durable Platelet Response | 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. | Week 18 to week 25 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With an Overall Platelet Response | Overall platelet response is defined as either a durable platelet response or transient platelet response. | 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Run hui Wu, Doctorate | Contact | 13370115037 | wurunhuigcp@163.com |
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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
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| Placebo | Drug | Matching placebo administered by subcutaneous injection. |
|
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 |
| Number of Weeks With Platelet Response | Number of weeks with platelet counts ≥ 50 x 10^9/L. | 24 weeks |
| Percentage of Participants Who Received Rescue Medication During the Treatment Period. | Rescue medication is any medication that is intended to increase platelet counts or prevent bleeding. | 24 weeks |
| 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 |
| D006425 |
| Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |