Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)

NCT03866798 | Terminated Phase 4 Results FDA Drug

• 1 other identifier: NGAM-10
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 8

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.

Conditions

Chronic Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• 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).

  8 Days

Secondary Outcomes (3)

• Days to Reach Platelet Count of at Least 50x10^9/L

  Through study completion, up to 37 days

• Duration of Platelet Response

  Through study completion, up to 37 days

• Maximum Platelet Count

  Through study completion, up to 37 days

Study Arms (1)

Panzyga

EXPERIMENTAL

Panzyga

Biological: Panzyga

Interventions

Panzyga BIOLOGICAL

Immune Globulin, intravenous, human-ifas

Panzyga

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Octapharma: lead

Study Sites (8)

Octapharma Research Site

Sacramento, California, 95817, United States

Location

Octapharma Research Site

Minneapolis, Minnesota, 55404, United States

Location

Octapharma Research Site

Rochester, Minnesota, 55905, United States

Location

Octapharma Research Site

Columbus, Ohio, 43205, United States

Location

Octapharma Research Site

Toledo, Ohio, 43606, United States

Location

Octapharma Research Site

Philadelphia, Pennsylvania, 19104, United States

Location

Octapharma Research Site

Providence, Rhode Island, 02903, United States

Location

Octapharma Research Site

Houston, Texas, 77030, United States

Location

MeSH Terms

Interventions

Panzyga

Results Point of Contact

• Title: Patrick Murphy
• Organization: Clinical Research Management Group

p.murphy@crmg-usa.com

4138210022

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 6, 2019
• First Posted: March 7, 2019
• Study Start: January 21, 2020
• Primary Completion: September 19, 2023
• Study Completion: May 17, 2024
• Last Updated: September 24, 2024
• Results First Posted: August 7, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)