Primary Immunodeficiency Disease (PIDD) MedDRA version: 20.0 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Baxalta US Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject must have a documented diagnosis of a form of primary humoral immunodeficiency involving a defect in antibody formation and requiring gammaglobulin replacement, as defined according to the IUIS (International Union of Immunological Societies) Scientific Committee 2015 (Picard et al., 2015) prior to enrollment. The diagnosis must be confirmed by the sponsor´s Medical Director prior to first treatment with IP in the study. 2. Subject is at least two and below 18 years of age at the time of screening. 3. Subject has been receiving a consistent dose of IgG, administered in compliance with the respective product information for a period of at least three months prior to screening. The average minimum pre-study dose over that interval was equivalent to 300 mg/kg BW/four weeks and a maximum dose equivalent to 1000 mg/kg BW/4 weeks. 4. Subject has a serum trough level of IgG > 5 g/L at screening. 5. If female of childbearing potential, subject presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study. 6. Subject/legally authorized representative is willing and able to comply with the requirements of the protocol. Are the trial subjects under 18? yes Number of subjects for this age range: 40 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subject has a known history of or is positive at 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. 2. Abnormal laboratory values at screening meeting any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent): a. Persistent alanine aminotransferase (ALT) and aspartate amino transferase (AST) >2.5 times the upper limit of normal (ULN) for the testing laboratory b. Persistent severe neutropenia (defined as an absolute neutrophil count [ANC] = 500/mm3) 3. Subject has anemia that would preclude phlebotomy for laboratory studies, according to standard practice at the site. 4. Subject has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following IV immunoglobulin, SC immunoglobulin, and/or Immune Serum Globulin (ISG) infusions. 5. Subject has severe immunoglobulin A (IgA) deficiency (less than 7.0 mg/dL) with known anti-IgA antibodies and a history of hypersensitivity. . 6. Subject has a known allergy to hyaluronidase. 7. Subject has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening. 8. Subject has a bleeding disorder or a platelet count less than 20,000/µL, or who, in the opinion of the investigator, would be at significant risk of increased bleeding or bruising as a result of SC therapy. 9. Subject has severe dermatitis that would preclude adequate sites for safe product administration. 10. Subject has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study. 11. Subject is a family member or employee of the investigator. 12. If female, subject is pregnant or lactating at the time of enrollment.
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy: Trough levels of IgG (for Study Epochs 1 and 2) Safety/Tolerability: Proportion of subjects who achieve a treatment interval of three or four weeks in Epoch 2 Proportion of subjects who maintain a treatment interval of three or four weeks in Epoch 2 for 12 months Number and rate per infusion (excluding infections) of local AEs and ARs Number and rate per infusion (excluding infections) of systemic AEs and ARs Number and rate per infusion (excluding infections) of all AEs and all ARs Number and rate per infusion (excluding infections) of all temporally associated AEs Number and rate per infusion (excluding infections) of all causally related and/or temporally associated AEs Number and rate per infusion (excluding infections) of all SAEs Number/proportion of subjects who develop positive titer (=160) of binding or neutralizing antibodies to rHuPH20 Additional safety outcome measures include. - Clinical laboratory outcomes: raw (actual) values and change from baseline - Vital signs: raw (actual) values and change from baseline;Timepoint(s) of evaluation of this end point: Efficacy: Up to three years. Safety: Up to one year. | — |
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Safety of HyQvia treatment in pediatric subjects with PIDD who have received prior immunoglobulin therapy before enrollment into the study.;Secondary Objective: Further safety assessments (e.g. immunogenicity), tolerability, characteristic of product administration and efficacy (immunoglobulin G [IgG] trough levels).;Primary end point(s): Number and rate per infusion (excluding infections) of all severe related AEs Number and rate per infusion (excluding infections) of related SAEs;Timepoint(s) of evaluation of this end point: Yearly | — |
Countries
Czech Republic, Denmark, France, Greece, Hungary, Slovakia, Sweden, United Kingdom
Contacts
Public ContactLee Hughes
Baxalta US Inc.
Outcome results
None listed