Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Th… (NCT03866798) | Clinical Trial Compass
TerminatedPhase 4
Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)
Stopped: FDA released Octapharma from the pediatric post marketing requirement.
United States6 participantsStarted 2020-01-21
Plain-language summary
This is a prospective, open-label, single-arm, multicenter, Phase 4 study evaluating the efficacy and safety of PANZYGA in pediatric patients with chronic ITP.
Who can participate
Age range
1 Year – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Females and males aged from ≥1 year to \<18 years old
. Confirmed diagnosis of Chronic Immune Thrombocytopenia (ITP) according to American Society of Hematology (ASH) 2019 guidelines
. Platelets count \<30x10\^9/L at the Baseline Visit
. Voluntarily given written informed consent (provided by patient's parent or legal guardian) and assent (provided by patient \[if age-appropriate per IRB (Institutional Review Board) requirements\])
. Sexually active females who have been using at least 1 acceptable form of birth control for a minimum of 30 days (or a minimum of 3 months for hormonal contraceptives) prior to the Screening visit and must agree to use at least 1 acceptable method of contraception throughout the study and for 30 days after the last dose of PANZYGA. Acceptable methods of birth control for this study include: intrauterine device (IUD), hormonal contraception, male or female condom, spermicide gel, diaphragm, sponge, or cervical cap. For non-sexually active females who have begun menstruating, abstinence is considered an acceptable method of birth control.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. Parent or legal guardian must agree and be willing to assist the participant attend study visits, and to follow all protocol requirements and instructions of the study doctor
Exclusion criteria
. Thrombocytopenia secondary to other diseases (such as Acquired Immunodeficiency Syndrome \[AIDS\] or systemic lupus erythematosus \[SLE\]), drug-related thrombocytopenia, or congenital thrombocytopenia
. Administration of intravenous immunoglobulin (IGIV) or anti-D immunoglobulin within 3 weeks (+/- 3 days) before enrollment
. Administration of thrombopoietin receptor agonists when the dose has NOT been stable within 3 weeks before enrollment and a dosage change is planned before Day 32
. Administration of oral immunosuppressants when the dose has NOT been stable during the preceding 2 months (2 weeks for long-term corticosteroid therapy) and a dosage change is planned before Day 32 (Note: topical agents and inhaled corticosteroid therapy use is permitted)
. Administration of long-term anti-prolific agents or attenuated androgen therapy when the dose has NOT been stable during the preceding 2 months and a dosage change is planned before Day 32
. Nonresponsive to previous treatment with IGIV or anti-D immunoglobulin
. Evidence of an active major bleeding episode at Screening
. Splenectomy in the previous 3 months or planned splenectomy throughout the study period