PI Pivotal Trial Results—Efficacy
The efficacy and safety of Gammaplex 5% in PI was assessed in a 12-month study
- Open-label, non-comparative study conducted at seven US centers evaluating Gammaplex 5% (immune globulin intravenous [human], 5% liquid) in the treatment of 50 adult patients with primary immunodeficiency (PID) and significant hypogammaglobulinemia1
- Prior to starting Gammaplex 5%, patients were stable on a different IVIG for at least 3 months1
- 43/50 patients (86%) were taking a 10% IVIG product2
- Primary endpoint was the incidence of serious acute bacterial infections (SABIs). SABIs were defined as1:
- Bacterial pneumonia
- Bacteraemia or sepsis
- Osteomyelitis/septic arthritis
- Visceral abscess
- Bacterial meningitis
- Mean IgG half-life was 41.1 days in a predefined subset of 24 patients
- IgG trough levels were similar on Gammaplex 5% and on previous therapy
- Median IgG trough concentrations after 15 weeks on Gammaplex 5% were within the range for subjects without PID, which previously has been shown to confer effective protection against infection in patients with PID
Gammaplex 5% is made from human plasma and may contain infectious agents, e.g., viruses and, theoretically, the Creutzfeldt-Jakob disease agent.
Over 12 months of treatment with Gammaplex 5%, no SABIs were reported1
- The primary endpoint surpassed the FDA minimum requirement of <1.0 SABI per patient per year
- 6/50 patients (12%) reported a SABI in the previous 6 months before switching to Gammaplex 5%
- The most common adverse reactions reported in >5% of PI clinical trial subjects were headache, pyrexia, fatigue, nausea, hypertension, chills, myalgia, pain, and vomiting
*This patient underwent lumbar disc surgery, experienced recurrent wheezing and had a coincidental squamous cell carcinoma (240 days off school/work).
References: 1. Moy JN, Scharenberg AM, Stein MR, et al. Clin Exp Immunol. 2010;162:510-515. 2. Data on File, BPL-GMX01-1008.