Chronic Immune ThrombocytopeniaPhase 4NCT03866798
Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)
A Phase 4 study testing Panzyga in people with Chronic Immune Thrombocytopenia, run by Octapharma at 8 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Chronic Immune Thrombocytopenia
- What is being tested?
- Panzyga
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 8 sitesUnited States
- Study dates
- 21 Jan 2020to 17 May 2024
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
- 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
Who cannot join
- 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
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- Evidence of an active major bleeding episode at Screening
- Splenectomy in the previous 3 months or planned splenectomy throughout the study period
- Evans syndrome (experiencing active disease with 2 out of 3 of the following: autoimmune thrombocytopenia, autoimmune hemolytic anemia, and/or autoimmune neutropenia)
- Known or suspected human immunodeficiency virus (HIV), hepatitis B virus (HBV), and/or hepatitis C virus (HCV) infections
- Emergency surgery in the previous 4 weeks
- 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)
- History of severe hypersensitivity to blood or plasma derived products, or any component of the PANZYGA
- Known immunoglobulin A (IgA) deficiency and antibodies against IgA
- History of, or suspected alcohol or drug abuse in the previous year
- Females who are pregnant or nursing
- Unable or unwilling to comply with the study protocol
- Receipt of any other investigational medicinal product within 3 months before study entry
- Risk factors* for thromboembolic events in whom the risks outweigh the potential benefit of PANZYGA treatment.
- 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.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Octapharma Research Site
Columbus, Ohio, 43205
Octapharma Research Site
Houston, Texas, 77030
Octapharma Research Site
Minneapolis, Minnesota, 55404
Octapharma Research Site
Philadelphia, Pennsylvania, 19104
Octapharma Research Site
Providence, Rhode Island, 02903
Octapharma Research Site
Rochester, Minnesota, 55905
Octapharma Research Site
Sacramento, California, 95817
Octapharma Research Site
Toledo, Ohio, 43606
About this study
Official title: Post-Marketing Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)
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.
- Conditions
- Chronic Immune Thrombocytopenia
- Being tested
- Panzyga
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PanzygaBiological
Immune Globulin, intravenous, human-ifas
| Study group | Role | What they receive |
|---|---|---|
| Panzyga | Experimental | Panzyga Receives: Biological: Panzyga |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 8 Days
Secondary outcomes (3)
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
Measured: Through study completion, up to 37 days
Duration of Platelet Response
Number of days the platelet count remains above at least 50x10^9/L
Measured: Through study completion, up to 37 days
Maximum Platelet Count
Maximum platelet count (10^9/L) for each subjects
Measured: Through study completion, up to 37 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2019
- Study start21 Jan 2020
- Primary completion19 Sep 2023
- Study completion17 May 2024
Why it stopped: FDA released Octapharma from the pediatric post marketing requirement.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Chronic Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by OctapharmaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03866798
- Sponsor's protocol ID
- NGAM-10
- Status last verified
- Sep 2024
- First submitted
- 6 Mar 2019
- Last updated
- 24 Sep 2024
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Post-Marketing Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)
- ClinicalTrials.gov ID
- NCT03866798
- Acronym / sponsor ID
- NGAM-10
- Conditions
- Chronic Immune Thrombocytopenia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: FDA released Octapharma from the pediatric post marketing requirement.
- Enrolment
- 6 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- 21 Jan 2020 (actual)
- Primary completion
- 19 Sep 2023 (actual)
- Study completion
- 17 May 2024 (actual)
- First posted
- 7 Mar 2019 (actual)
- Results
- Posted 7 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Panzyga | Experimental | Biological: PanzygaPanzyga |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (21 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PanzygaImmune Globulin, intravenous, human-ifas | Biological | Panzyga |
Primary outcome
Platelet Count Increase
Time frame: 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).
3 secondary outcomes
Days to Reach Platelet Count of at Least 50x10^9/L
Time frame: Through study completion, up to 37 days
Duration of Platelet Response
Time frame: Through study completion, up to 37 days
Maximum Platelet Count
Time frame: Through study completion, up to 37 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Panzyga in people with Chronic Immune Thrombocytopenia, run by Octapharma at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 24 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Platelet Count Increase (time frame: 8 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2024.