Chronic Immune Thrombocytopenia
Conditions
Brief 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.
Interventions
BIOLOGICALPanzyga
Immune Globulin, intravenous, human-ifas
Sponsors
Octapharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
1. Females and males aged from ≥1 year to \<18 years old 2. Confirmed diagnosis of Chronic Immune Thrombocytopenia (ITP) according to American Society of Hematology (ASH) 2019 guidelines 3. Platelets count \<30x10\^9/L at the Baseline Visit 4. Voluntarily given written informed consent (provided by patient's parent or legal guardian) and assent (provided by patient \[if age-appropriate per IRB (Institutional Review Board) requirements\]) 5. 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. 6. 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
1. Thrombocytopenia secondary to other diseases (such as Acquired Immunodeficiency Syndrome \[AIDS\] or systemic lupus erythematosus \[SLE\]), drug-related thrombocytopenia, or congenital thrombocytopenia 2. Administration of intravenous immunoglobulin (IGIV) or anti-D immunoglobulin within 3 weeks (+/- 3 days) before enrollment 3. 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 4. 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) 5. 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 6. Nonresponsive to previous treatment with IGIV or anti-D immunoglobulin 7. Evidence of an active major bleeding episode at Screening 8. Splenectomy in the previous 3 months or planned splenectomy throughout the study period 9. Evans syndrome (experiencing active disease with 2 out of 3 of the following: autoimmune thrombocytopenia, autoimmune hemolytic anemia, and/or autoimmune neutropenia) 10. Known or suspected human immunodeficiency virus (HIV), hepatitis B virus (HBV), and/or hepatitis C virus (HCV) infections 11. Emergency surgery in the previous 4 weeks 12. Severe liver and/or kidney disease (alanine aminotransferase \[ALT\] \>3x upper limit of normal (ULN), aspartate aminotransferase \[AST\] \>3x upper limit of normal (ULN), and/or creatinine \>120 µmol/L) 13. History of severe hypersensitivity to blood or plasma derived products, or any component of the PANZYGA 14. Known immunoglobulin A (IgA) deficiency and antibodies against IgA 15. History of, or suspected alcohol or drug abuse in the previous year 16. Females who are pregnant or nursing 17. Unable or unwilling to comply with the study protocol 18. Receipt of any other investigational medicinal product within 3 months before study entry 19. Risk factors\* for thromboembolic events in whom the risks outweigh the potential benefit of PANZYGA treatment. 20. Any other condition(s), that in the Investigator's opinion, make it undesirable for the patient to participate in the study or may interfere with protocol compliance. * Risk factors include, but are not limited to: obesity, advanced age, hypertension, diabetes, a history of atherosclerosis/vascular disease or thrombotic events, hyperlipidemia, multiple cardiovascular risk factors, acquired or inherited thrombophilic disorders, prolonged periods of immobilization, severe hypovolemia, central venous catheterization, active malignancy and/or known or suspected hyperviscosity.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Platelet Count Increase | 8 Days | Number of subjects with an increase in platelet count at least once to ≥50 × 10\^9/L within 7 days after the first infusion, i.e., by Day 8 (increase must have occurred at least once on any day up to and including Day 8). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Days to Reach Platelet Count of at Least 50x10^9/L | Through study completion, up to 37 days | Number of days for subjects to reach Platelet Count of at least 50x10\^9/L after infusion |
| Duration of Platelet Response | Through study completion, up to 37 days | Number of days the platelet count remains above at least 50x10\^9/L |
| Maximum Platelet Count | Through study completion, up to 37 days | Maximum platelet count (10\^9/L) for each subjects |
Countries
United States
Participant flow
Recruitment details
Six subjects aged ≥1 year to \<18 years were recruited between 21-Jan-2020 and 19-Sep-2023. The subjects were enrolled at five research sites across the United States, with a total of eight research sites activated in the study.
Participants by arm
| Arm | Count |
|---|---|
| Panzyga Panzyga
Panzyga: Immune Globulin, intravenous, human-ifas | 6 |
| Total | 6 |
Baseline characteristics
| Characteristic | Panzyga |
|---|---|
| Age, Categorical <=18 years | 6 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 3 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 3 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Region of Enrollment United States | 6 participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 6 |
| other Total, other adverse events | 4 / 6 |
| serious Total, serious adverse events | 0 / 6 |
Outcome results
Primary
Platelet Count Increase
Number of subjects with an increase in platelet count at least once to ≥50 × 10\^9/L within 7 days after the first infusion, i.e., by Day 8 (increase must have occurred at least once on any day up to and including Day 8).
Time frame: 8 Days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Panzyga | Platelet Count Increase | 4 Participants |
Secondary
Days to Reach Platelet Count of at Least 50x10^9/L
Number of days for subjects to reach Platelet Count of at least 50x10\^9/L after infusion
Time frame: Through study completion, up to 37 days
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Panzyga | Days to Reach Platelet Count of at Least 50x10^9/L | 2 days | 3 Participants |
| Panzyga | Days to Reach Platelet Count of at Least 50x10^9/L | 3 days | 1 Participants |
| Panzyga | Days to Reach Platelet Count of at Least 50x10^9/L | Did not reach Platelet Count of at least 50x10^9/L | 2 Participants |
Secondary
Duration of Platelet Response
Number of days the platelet count remains above at least 50x10\^9/L
Time frame: Through study completion, up to 37 days
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Panzyga | Duration of Platelet Response | 6 days | 1 Participants |
| Panzyga | Duration of Platelet Response | 7 days | 1 Participants |
| Panzyga | Duration of Platelet Response | 33 days | 1 Participants |
| Panzyga | Duration of Platelet Response | 34 days | 1 Participants |
| Panzyga | Duration of Platelet Response | Did not reach Platelet Count of at least 50x10^9/L | 2 Participants |
Secondary
Maximum Platelet Count
Maximum platelet count (10\^9/L) for each subjects
Time frame: Through study completion, up to 37 days
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Panzyga | Maximum Platelet Count | 99 (10^9/L) | 1 Participants |
| Panzyga | Maximum Platelet Count | 11 (10^9/L) | 1 Participants |
| Panzyga | Maximum Platelet Count | 516 (10^9/L) | 1 Participants |
| Panzyga | Maximum Platelet Count | 386 (10^9/L) | 1 Participants |
| Panzyga | Maximum Platelet Count | 204 (10^9/L) | 1 Participants |
| Panzyga | Maximum Platelet Count | 30 (10^9/L) | 1 Participants |