Primary Immunodeficiency Clinical Trial
Official title:
A Phase III, Multicenter, Open-Label Study To Evaluate The Efficacy, Safety, and Pharmacokinetics of Gammaplex® in Primary Immunodeficiency Diseases
The main objective of this study is to see if GAMMAPLEX is efficacious with respect to Food and Drug Administration (FDA) minimal requirements (no more than 1 serious, acute, bacterial infection per subject per year) in subjects with Primary Immunodeficiency Diseases (PID). The secondary objectives are to assess the safety and tolerability of GAMMAPLEX and to determine if GAMMAPLEX has a pharmacokinetic (PK) profile comparable with that of intact Immunoglobulin G (IgG) in subjects with PID.
Primary Efficacy Variable The primary variable is the number of serious, acute, bacterial
infections/subject/year, and it will be based on the total of all of the following events as
defined by the FDA: bacterial Pneumonia, bacteremia/sepsis, osteomyelitis/septic arthritis,
visceral abscess, and bacterial meningitis.
Secondary Efficacy Variables Secondary efficacy will be determined by using the following
variables: number of days of work/school missed because of infection per subject year;
number and days of hospitalizations because of infection per subject year; number of visits
to physicians for acute problems and/or number of visits to hospital emergency rooms per
subject year; other infections documented by fever or a positive result on a radiograph
and/or culture; number of infectious episodes per subject per year; number of days on
therapeutic antibiotics.These data will be entered into the subject diary, confirmed by the
physician, and entered on the electronic-CRF (e-CRF).
Safety Variables. The variables used to assess safety will be the following: adverse events
(AEs); vital signs; clinical laboratory tests and Direct Coombs' Test; transmission of
viruses; physical examination.
Test product, dose/mode of administration, batch number(s): The GAMMAPLEX dose is 300-800
mg/kg/infusion (milligram per killgram per infusion) every 21 or 28 days, intravenously. At
least 2 batches will be used in this study and no more than 1 batch in any given infusion.
Duration of treatment:
The total duration of treatment is 12 months.
;
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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