Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia

Octapharma40 enrolled

Overview

NewGam is a newly developed human normal immunoglobulin solution for intravenous administration (IGIV). This study will evaluate the safety and efficacy of NewGam 10% in patients with primary immune thrombocytopenia.

The primary objective of the study is to assess the efficacy of NewGam in correcting the platelet count. The secondary objective of the study is to evaluate the safety of NewGam. Safety will be assessed by monitoring vital signs, physical examination, evaluation of adverse events (AE) and laboratory parameters, and viral safety testing.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Primary Thrombocytopenia

Interventions

NewGamDRUG

NewGam is a solution of human normal immunoglobulin 10% treated with solvent/detergent and nanofiltered for intravenous administration.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Age of ≥ 18 and ≤ 65 years. 2. Confirmed diagnosis of chronic primary immune thrombocytopenia (ITP) (diagnosed with a threshold platelet count \< 100x10\^9/L) of at least 12 months duration. 3. Platelet count of no more than 20x10\^9/L with or without bleeding manifestations. 4. Freely given written informed consent from patient. 5. Women of childbearing potential must have a negative result on a pregnancy test (human chorionic gonadotropin \[HCG\]-based assay) and need to practice contraception using a method of proven reliability for the duration of the study. Exclusion Criteria: 1. Thrombocytopenia secondary to other diseases (such as acquired immunodeficiency syndrome \[AIDS\] or systemic lupus erythematosus \[SLE\]), or drug-related thrombocytopenia. 2. Administration of intravenous immunoglobulin (IGIV), anti-D immunoglobulin or thrombopoietin receptor agonists, or other platelet enhancing drugs (including immunosuppressive or other immunomodulatory drugs) within 3 weeks before enrollment, except for: * Long-term corticosteroid therapy when the dose has been stable during the preceding 3 weeks and no dosage change is planned until study Day 22. * Long-term azathioprine, cyclophosphamide, or attenuated androgen therapy when the dose has been stable during the preceding 3 months and no dosage change is planned until study Day 22. 3. Unresponsive to previous treatment with IGIV or anti-D immunoglobulin.

Locations (1)

Abdulgabar Salama

Berlin, Germany

Outcomes

Primary Outcomes

Percentage of Responders

A responder is a study participant with an increase in platelets to ≥ 50x10\^9/L within 7 days after the first infusion, ie, by study Day 8.

Time frame: Day 1 to Day 8

Secondary Outcomes

Percentage of Alternative Responders

An alternative responder is a study participant with an increase in platelets to ≥ 30x10\^9/L and to at least double the baseline platelet count, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.

Time frame: Day 1 to Day 22

Percentage of Complete Responders

A complete responder is a study participant with an increase in platelets to ≥ 100x10\^9/L, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.

Time frame: Day 1 to Day 22

Percentage of Alternative Responders Who Lost the Response

An alternative responder who lost the response is a study participant who met the criterion for an alternative response but who then deteriorated, ie, their platelet count decreased to \< 30x10\^9/L, their platelet count decreased to less than double the baseline count, or bleeding occurred.

Time frame: Day 1 to Day 22

Percentage of Complete Responders Who Lost the Response

A complete responder who lost the response is a study participant who met the criterion for a complete response but who then deteriorated, ie, their platelet count decreased to \< 100x10\^9/L or bleeding occurred.

Time frame: Day 1 to Day 22

Time to a Response

A study participant had a response if their platelets increased to ≥ 50x10\^9/L within 7 days after the first infusion, ie, by study Day 8.

Data from ClinicalTrials.gov

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