Gammagard Liquid and rHuPH20 in PID

Baxalta

Status and phase: Completed; Phase 3

Conditions: Primary Immunodeficiency Diseases (PID)

Treatments: Biological: Recombinant human hyaluronidase (rHuPH20)+ immune globulin intravenous (IGIV)

Study type: Interventional

Funder types: Industry

Identifiers

NCT00814320

160603

Details and patient eligibility

About

The purpose of the study is to develop a subcutaneous treatment option for participants with Primary Immunodeficiency Diseases (PID) that allows an administration of Immune Globulin Intravenous (Human), 10% at the same frequency as IV administration.

Enrollment: 89 patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participant is 2 years or older at the time of screening
• Written informed consent obtained from either the participant or the participant's legally acceptable representative prior to any study-related procedures and study product administration
• Participant has been diagnosed with a PID disorder requiring antibody replacement as defined by WHO criteria
• Participant has completed or is about to complete Baxter Clinical Study Protocol No. 160601 or has been receiving a regular IGIV-treatment at mean intervals of 21 ± 3 days or 28 ± 3 days, or SC at mean intervals of 5 to 16 days, over a period of at least 3 months prior to enrollment at a minimum dose of 300 mg/kg BW/4 weeks
• Participant has a serum trough level of IgG > 4.5 g/L at the last documented determination
• If female of childbearing potential, participant presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study
• Participant is willing and able to comply with the requirements of the protocol

Exclusion criteria

• Participant has a known history of or is positive at enrollment or screening for one or more of the following: Hepatitis B surface antigen (HbsAg), polymerase chain reaction (PCR) for Hepatitis C Virus (HCV), PCR for Human immunodeficiency virus (HIV) Type 1/2
• Participant has levels of alanine aminotransferase (ALT) or aspartate amino transferase (AST) > 2.5 times the upper limit of normal for the testing laboratory
• Participant has persistent severe neutropenia (defined as an absolute neutrophil count [ANC] <= 500/mm3)
• Participant has creatinine clearance (CLcr) values, calculated according to the formula below, which are < 60% of normal for age and gender for males: CLcr = [(140 - Age(years)) * (body weight (kg))] / [72 * (serum creatinine (mg/dL))] for females: CLcr = [(140 - Age(years)) * (body weight(kg)) * 0.85] / [72 * (serum creatinine (mg/dL))]
• Participant has been diagnosed with, or has a malignancy (other than adequately treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix) within the last 12 months prior to enrollment; participants treated with immunosuppressive chemotherapeutic agents during this period are excluded
• Participant has a history of thrombotic episodes (including deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism) within the last 12 months
• Participant has abnormal protein loss (protein losing enteropathy, nephrotic syndrome)
• Participant has anemia that would preclude phlebotomy for laboratory studies
• Participant has received any blood or blood product other than an IGIV, SC immunoglobulin, immune serum globulin (ISG) preparation, or albumin within the 6 months prior to enrollment
• Participant has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following IGIV, SC immunoglobulin, and/or ISG infusions
• Participant has immunoglobulin A (IgA) deficiency and known anti IgA antibodies
• Participant is on preventative (prophylactic) antibiotics and cannot stop antibiotics at the time of enrollment
• Participant has active infection who started on antibiotic therapy for the treatment of infection within 7 days prior to screening
• Participant has a bleeding disorder or is on anti-coagulation therapy that results in a platelet count less than 20,000/μL or International Normalized Ration (INR) > 2X control, or who, in the opinion of the investigator would be at significant risk of increased bleeding or bruising as a result of SC therapy
• Participant has total protein > 9 g/dL and participants with myeloma, macroglobulinemia (IgM) and paraproteinemia
• Participant has a known allergy to hyaluronidase
• If female, participant is pregnant or lactating at the time of study enrollment
• Participant has participated in another clinical study involving an investigational product (IP) or device within 30 days prior to study enrollment or is scheduled to participate in another clinical study involving an IP or device during the course of this study; exception: Baxter Study No. 160601
• Severe dermatitis that would preclude adequate sites for safe product administration

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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89 participants in 2 patient groups

1

Experimental group

Description:

Efficacy and tolerability of subcutaneous (SC) infusions of immune globulin intravenous (IGIV), 10% with hyaluronidase (rHuPH20) after ramp-up

Treatment:

Biological: Recombinant human hyaluronidase (rHuPH20)+ immune globulin intravenous (IGIV)

Biological: Recombinant human hyaluronidase (rHuPH20)+ immune globulin intravenous (IGIV)

2

Experimental group

Description:

Pharmacokinetics of intravenous (IV) infusions of immune globulin intravenous (IGIV), 10% and efficacy and tolerability of subcutaneous (SC) infusions of immune globulin intravenous (IGIV), 10% with hyaluronidase (rHuPH20) after ramp-up

Treatment:

Biological: Recombinant human hyaluronidase (rHuPH20)+ immune globulin intravenous (IGIV)

Biological: Recombinant human hyaluronidase (rHuPH20)+ immune globulin intravenous (IGIV)