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

Trial statusNot yet recruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age1-17
SponsorQilu Pharmaceutical Co., Ltd.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age ≥ 1 years old and < 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;

Disqualifiers

Had a history of bone marrow stem cell abnormalities or myelodysplastic syndrome other than ITP-specific changes.

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)within 4 weeks before the first dose;

Trial design

Design model

Parallel

Treatments tested in this trial

  • 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.

  • Placebo

    Drug

    Matching placebo administered by subcutaneous injection.

Treatment groups

60 Participants
are divided into 2 treatment groups
Group A: QL0911Experimental treatment 1 intervention
Group B: placeboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

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.

Time frame
Week 18 to week 25

Secondary outcomes

1

Percentage of Participants With an Overall Platelet Response

Overall platelet response is defined as either a durable platelet response or transient platelet response.

Time frame
24 weeks
2

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.

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.

Time frame
24 weeks
3

Number of Weeks With Platelet Response

Number of weeks with platelet counts ≥ 50 x 10\^9/L.

Time frame
24 weeks
4

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.

Time frame
24 weeks

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

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