A clinical trial protocol for the study of intravenous immunoglobulin in patients with primary antibody immunodeficiency disorders

Abstract

This protocol has been excerpted from protocols recently used to study the safety, efficacy, and pharmacokinetics of 2 new intravenous immunoglobulins (IVIG). It incorporates the safety and efficacy parameters provided by the United States Food and Drug Administration in in a Guidance for Industry that contains recommendations for Investigational New Drug Application sponsors and Biologic License applicants for testing IVIG products as replacement therapy in primary humoral immunodeficiency. The format and contents of the protocol are generally the same as described in the ICH Guideline for Good Clinical Practice. We describe here the topics found in the main body of the protocol. We have omitted items that will be included in a formal protocol such as the title page, identifying number, name and contact information of the sponsor, names and contact information of the persons authorized to sign the protocol and amendments, etc. The success of a study of IVIG in patients with primary humoral immunodeficiency is very much dependent on the care in which patients are selected. It is extremely important to enroll patients who have been verified to be antibody deficient. Therefore, patient selection criteria should involve a thorough review of each patient’s infection history, serum IgG level prior to immunoglobulin therapy, and antibody responses to polysaccharide and protein vaccines. Statement of novelty: This protocol is designed to select study subjects with well-documented antibody deficiencies and therefore will benefit from immunoglobulin therapy.