Clinical trial NCT01218438: Phase 2/3 Study of IGSC, 20% in PIDD
Official title: A Clinical Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) for the Evaluation of Efficacy, Safety, Tolerability and Pharmacokinetics in Subjects With Primary Immunodeficiency Diseases (PIDD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Primary Immunodeficiency Diseases (PID)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2010-10-11
Enrollment: 86
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to develop a 20% subcutaneous (SC) immunoglobulin preparation for the treatment of patients with primary immunodeficiency diseases (PIDD).


Eligibility criteria: Main Inclusion Criteria:

* Documented diagnosis of a form of primary humoral immunodeficiency involving defective antibody formation and requiring gammaglobulin replacement as defined according to the IUIS Scientific Committee, 2011 and by diagnostic criteria according to Conley et al. (1999). The diagnosis must be confirmed by the Medical Director prior to first treatment with the investigational product (IP) in the study.
* Participant is 2 years or older at the time of screening, and has a minimum body weight of 13 kg.
* Written informed consent has been obtained from either the participant or the participant's legally authorized representative prior to any study-related procedures and study product administration
* Participant has been receiving a stable monthly equivalent dose of IgG at an average minimum dose equivalent to 300 mg/kg bodyweight (BW)/4 weeks and a maximum dose equivalent to 1.0 gram/kg BW/4 weeks, for a minimum of 12 weeks prior to first treatment with the IP in the study.
* Serum trough level of IgG \> 500 mg/dL at screening
* Participant is willing and able to comply with the requirements of the protocol

Main Exclusion Criteria:

* Participant has 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
* Abnormal laboratory values at screening meeting any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent):

  * Persistent alanine aminotransferase (ALT) and aspartate amino transferase(AST) \> 2.5 times the upper limit of normal for the testing laboratory
  * Persistent severe neutropenia (defined as an absolute neutrophil count \[ANC\] ≤500/mm\^3)
* Creatinine clearance (CLcr) value that is \< 60% of normal for age and gender either measured, or calculated according to the Cockcroft-Gault formula
* Malignancy (other than adequately treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix), unless the disease-free period prior to screening exceeds 5 years
* Participant is receiving anti-coagulation therapy (low dose aspirin at ≤325 mg/day is permitted) or has a history of thrombotic episodes (including deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism) or sickle cell disease with crisis within 12 months prior to screening or a history of thrombophilia
* Abnormal protein loss (protein losing enteropathy, nephrotic syndrome)
* Anemia that would preclude phlebotomy for laboratory studies according to standard practice at the site
* Acute serious bacterial infection within 3 months prior to screening
* Ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following intravenous immunoglobulin, subcutaneous immunoglobulin, and/or Immune Serum Globulin (ISG) infusions
* Severe immunoglobulin A (IgA) deficiency (less than 0.07g/L) with known anti-IgA antibodies and a history of hypersensitivity
* Participant is on continuous systemic antibacterial antibiotics at doses sufficient to treat or prevent bacterial infections, and, in the opinion of the investigator, cannot stop these for the duration of the study without putting the patient at risk of increased infections
* Participant has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening
* Bleeding disorder or thrombocytopenia with 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 subcutaneous therapy
* Total protein \> 9 g/dL or myeloma or macroglobulinemia (IgM) or paraproteinemia
* Severe dermatitis that would preclude adequate sites for safe product administration
* Women of childbearing potential meeting any one of the following criteria:

  * Participant presents with a positive pregnancy test
  * Participant is breast feeding
  * Participant intends to begin nursing during the course of the study
  * Participant does not agree to employ adequate birth-control measures (e.g. intrauterine device, diaphragm or condom \[for male partner\] with spermicidal jelly or foam, or birth control pills/patches) throughout the course of the study
* Participation in another clinical study and exposure to an investigational product or device within 30 days prior to study enrollment (exception: treatment in a previous Baxter immunoglobulin study)
* Participant is scheduled to participate in another (non-Baxter) non-observational (interventional) clinical study involving an investigational product or device during the course of the study

Primary outcomes:
measure: Rate of Acute Serious Bacterial Infections Per Year (ASBI)
timeFrame: 1 year
Secondary outcomes:
measure: Annual Rate of All Infections Per Participant
timeFrame: 1 year
measure: Annual Rate of Sinus Infections Per Participant
timeFrame: 1 year
measure: Annual Rate of Fever Episodes Per Participant
timeFrame: 1 year
measure: Annual Rate of Days Off School/Work or Days Unable to Perform Normal Daily Activities Due to Illness or Infection Per Participant
timeFrame: 1 year
measure: Annual Rate of Days on Antibiotics Per Participant
timeFrame: 1 year
measure: Annual Rate of Hospitalizations for Illness or Infection Per Participant
timeFrame: 1 year
measure: Annual Rate of Days of Hospitalizations for Illness or Infection Per Participant
timeFrame: 1 year
measure: Annual Rate of Acute (Urgent or Unscheduled) Physician Visits, or Visits to the Emergency Room for Illness or Infection Per Participant
timeFrame: 1 year
measure: Bioavailability of IGSC, 20% as Measured by the Ratio of the Geometric Means of Immunoglobulin G (IgG) AUCSC (Epoch 4) to IgG AUCIV,0-τ (Standardized to 1 Week) (Epoch 1) Adjusted for Dose and Dosing Frequency (Participants ≥12 Years Old)
timeFrame: Epoch 1: 3 week IV administration interval: Week 10, 11, 12, 13. Epoch 1: 4 week IV interval: Week 9, 10, 11, 12, 13. Epoch 4 Subcutaneous administration weeks 17, 18
measure: Trough Levels of IgG (Total), and IgG Subclasses at the End of the Treatment Intervals
timeFrame: Epoch 1: 3 week IV interval- weeks 0, 1, 4, 7, 10, 13. Epoch 1: 4 week IV interval- weeks 0, 1, 5, 9, 13. Epochs 2 & 3: Subcutaneous (SC) weeks 5, 9. Epoch 4: SC weeks 1, 9, 17, 29, 40 (week dependent on time to establish "Adjusted Dose" in Epoch 2)
measure: Trough Levels of Anti-Tetanus Antibody
timeFrame: Epoch 1: 3 week IV interval- weeks 1, 4, 7, 10, 13. Epoch 1: 4 week IV interval- weeks 1, 5, 9, 13. Epochs 2 & 3: Subcutaneous (SC) weeks 5, 9. Epoch 4: SC weeks 1, 9, 17, 29, 40 (week dependent on time to establish "Adjusted Dose" in Epoch 2)
measure: Trough Levels of Anti-Haemophilus Influenza B Antibody
timeFrame: Epoch 1: 3 week IV interval- weeks 1, 4, 7, 10, 13. Epoch 1: 4 week IV interval- weeks 1, 5, 9, 13. Epochs 2 & 3: Subcutaneous (SC) weeks 5, 9. Epoch 4: SC weeks 1, 9, 17, 29, 40 (week dependent on time to establish "Adjusted Dose" in Epoch 2)
measure: Trough Levels of Anti-Hepatitis B Antibody
timeFrame: Epoch 1: 3 week IV interval- weeks 1, 4, 7, 10, 13. Epoch 1: 4 week IV interval- weeks 1, 5, 9, 13. Epochs 2 & 3: Subcutaneous (SC) weeks 5, 9. Epoch 4: SC weeks 1, 9, 17, 29, 40 (week dependent on time to establish "Adjusted Dose" in Epoch 2)
measure: Pharmacokinetics Parameters for Immunoglobulin G (IgG): Area Under the Curve (AUC)
timeFrame: Epoch 1: 3 week IV interval: Weeks 10-13. Epoch 1: 4 week IV interval: Weeks 9-13. Epoch 2: Subcutaneous (SC) weeks 9-10. Epoch 4: SC weeks 17-18
measure: Pharmacokinetics Parameters for Immunoglobulin G (IgG): Dose Per Weight-adjusted Area Under the Curve (AUC)
timeFrame: Epoch 1: 3 week IV interval: Weeks 10-13. Epoch 1: 4 week IV interval: Weeks 9-13. Epoch 2: Subcutaneous (SC) weeks 9-10. Epoch 4: SC weeks 17-18
measure: Pharmacokinetics Parameters for Immunoglobulin G (IgG): Clearance (CL) for Immune Globulin Administered Intravenously (IGIV) and Apparent Clearance for Immune Globulin Administered Subcutaneously (IGSC)
timeFrame: Epoch 1: 3 week IV interval: Weeks 10-13. Epoch 1: 4 week IV interval: Weeks 9-13. Epoch 2: Subcutaneous (SC) weeks 9-10. Epoch 4: SC weeks 17-18
measure: Pharmacokinetics Parameters for Immunoglobulin G (IgG): Maximum Concentration (Cmax)
timeFrame: Epoch 1: 3 week IV interval: Weeks 10-13. Epoch 1: 4 week IV interval: Weeks 9-13. Epoch 2: Subcutaneous (SC) weeks 9-10. Epoch 4: SC weeks 17-18
measure: Pharmacokinetics Parameters for Immunoglobulin G (IgG): Time to Maximum Concentration (Tmax)
timeFrame: Epoch 1: 3 week IV interval: Weeks 10-13. Epoch 1: 4 week IV interval: Weeks 9-13. Epoch 2: Subcutaneous (SC) weeks 9-10. Epoch 4: SC weeks 17-18
measure: Pharmacokinetics Parameters for Immunoglobulin G (IgG): Minimum Concentration (Cmin)
timeFrame: Epoch 1: 3 week IV interval: Weeks 10-13. Epoch 1: 4 week IV interval: Weeks 9-13. Epoch 2: Subcutaneous (SC) weeks 9-10. Epoch 4: SC weeks 17-18
measure: Pharmacokinetics Parameters for Haemophilus Influenza B Antibody: Area Under the Curve (AUC)
timeFrame: Epoch 1: 3 week IV interval: Weeks 10-13. Epoch 1: 4 week IV interval: Weeks 9-13. Epoch 2: Subcutaneous (SC) weeks 9-10. Epoch 4: SC weeks 17-18
measure: Pharmacokinetics Parameters for Haemophilus Influenza B Antibody: Dose Per Weight-adjusted Area Under the Curve (AUC)
timeFrame: Epoch 1: 3 week IV interval: Weeks 10-13. Epoch 1: 4 week IV interval: Weeks 9-13. Epoch 2: Subcutaneous (SC) weeks 9-10. Epoch 4: SC weeks 17-18
measure: Pharmacokinetics Parameters for Haemophilus Influenza B Antibody: Clearance (CL) for Immune Globulin Administered Intravenously (IGIV) and Apparent Clearance (CL/F) for Immune Globulin Administered Subcutaneously
timeFrame: Epoch 1: 3 week IV interval: Weeks 10-13. Epoch 1: 4 week IV interval: Weeks 9-13. Epoch 2: Subcutaneous (SC) weeks 9-10. Epoch 4: SC weeks 17-18
measure: Pharmacokinetics Parameters for Haemophilus Influenza B Antibody: Maximum Concentration (Cmax)
timeFrame: Epoch 1: 3 week IV interval: Weeks 10-13. Epoch 1: 4 week IV interval: Weeks 9-13. Epoch 2: Subcutaneous (SC) weeks 9-10. Epoch 4: SC weeks 17-18
measure: Pharmacokinetics Parameters for Haemophilus Influenza B Antibody: Time to Maximum Concentration (Tmax)
timeFrame: Epoch 1: 3 week IV interval: Weeks 10-13. Epoch 1: 4 week IV interval: Weeks 9-13. Epoch 2: Subcutaneous (SC) weeks 9-10. Epoch 4: SC weeks 17-18
measure: Pharmacokinetics Parameters for Haemophilus Influenza B Antibody: Minimum Concentration (Cmin)
timeFrame: Epoch 1: 3 week IV interval: Weeks 10-13. Epoch 1: 4 week IV interval: Weeks 9-13. Epoch 2: Subcutaneous (SC) weeks 9-10. Epoch 4: SC weeks 17-18
measure: Correction Factor to Determine the Individually Adapted Dose in Study 170904 Epoch 4 (Dose Adjustment Table)
timeFrame: 29 weeks
measure: Number of Serious (SAEs) and Non-serious Adverse Events (AEs) (Including and Excluding Infections) Deemed Related to the Investigational Product Per Participant
timeFrame: Up to 20 months per subject (throughout entire study)
measure: Number of Serious (SAEs) and Non-serious Adverse Events (AEs) (Including and Excluding Infections) Deemed Related to the Investigational Product Per Infusion
timeFrame: Up to 20 months per subject (throughout entire study)
measure: Number of Serious (SAEs) and Non-serious Adverse Events (AEs) (Including and Excluding Infections) Regardless of Relationship to the Investigational Product Per Participant
timeFrame: Up to 20 months per subject (throughout entire study)
measure: Number of Serious (SAEs) and Non-serious Adverse Events (AEs) (Including and Excluding Infections) Regardless of Relationship to the Investigational Product Per Infusion
timeFrame: Up to 20 months per subject (throughout entire study)
measure: Number of Adverse Events (AEs) (Including and Excluding Infections) That Begin During or Within 72 Hours of Completion of Infusion Per Participant
timeFrame: Within 72 hours of completion of infusion
measure: Number of Adverse Events (AEs) (Including and Excluding Infections) That Begin During or Within 72 Hours of Completion of Infusion Per Infusion
timeFrame: Within 72 hours of completion of infusion
measure: Number of Adverse Events (AEs) (Including and Excluding Infections) That Begin During or Within 24 Hours of Completion of Infusion Per Participant
timeFrame: Within 24 hours of completion of infusion
measure: Number of Adverse Events (AEs) (Including and Excluding Infections) That Begin During or Within 24 Hours of Completion of Infusion Per Infusion
timeFrame: Within 24 hours of completion of infusion
measure: Number of Adverse Events (AEs) (Including and Excluding Infections) That Begin During or Within 1 Hour of Completion of Infusion Per Participant
timeFrame: Within 1 hour of completion of infusion
measure: Number of Adverse Events (AEs) (Including and Excluding Infections) That Begin During or Within 1 Hour of Completion of Infusion
timeFrame: Within 1 hour of completion of infusion
measure: Causally Related and/or Temporally Associated Adverse Events (AEs) Per Infusion
timeFrame: Within 72 hours post infusion for Temporally Associated AEs; End of each Study Epoch (Epoch 1, Epoch 2, Epoch 3, and Epoch 4) for Causally Related AEs
measure: Percentage of Infusions Associated With One or More Local Non-serious Adverse Events (Non-SAEs)
timeFrame: Up to 20 months (throughout entire study)
measure: Percentage of Participants Reporting One or More Local Non-serious Adverse Events (Non-SAEs)
timeFrame: Up to 20 months (throughout entire study)
measure: Number of Infusions for Which the Infusion Rate Was Reduced and/or the Infusion Was Interrupted or Stopped Due to Tolerability Concerns or AEs
timeFrame: Up to 20 months (throughout entire study)
measure: Percentage of Participants for Whom the Infusion Rate Was Reduced and/or the Infusion Was Interrupted or Stopped Due to Tolerability Concerns or AEs
timeFrame: Up to 20 months (throughout entire study)
measure: Percentage of Infusions Tolerated With Intravenous or Subcutaneous Administration
timeFrame: Up to 20 months (throughout entire study)
measure: Short Term Tolerance - Change in Systolic Blood Pressure: Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 1
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Systolic Blood Pressure: Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 2
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Systolic Blood Pressure: Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 3
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Systolic Blood Pressure: Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 1
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Systolic Blood Pressure: Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 2
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Systolic Blood Pressure: Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 3
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Diastolic Blood Pressure: Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 1
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Diastolic Blood Pressure: Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 2
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Diastolic Blood Pressure: Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 3
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Diastolic Blood Pressure: Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 1
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Diastolic Blood Pressure: Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 2
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Diastolic Blood Pressure: Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 3
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Heart Rate (Pulse): Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 1
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Heart Rate (Pulse): Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 2
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Heart Rate (Pulse): Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 3
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Heart Rate (Pulse): Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 1
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Heart Rate (Pulse): Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 2
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Heart Rate (Pulse): Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 3
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Respiratory Rate: Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 1
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Respiratory Rate: Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 2
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Respiratory Rate: Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 3
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Respiratory Rate: Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 1
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Respiratory Rate: Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 2
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Respiratory Rate: Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 3
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Body Temperature: Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 1
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Body Temperature: Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 2
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Body Temperature: Immune Globulin Administered Intravenously (Human), 10% (IV 10%), Infusion 3
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Body Temperature: Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 1
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Body Temperature: Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 2
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Short Term Tolerance - Change in Body Temperature: Immune Globulin Administered Subcutaneously (Human), 20% (SC 20%), Infusion 3
timeFrame: Within 30 minutes pre-infusion, during infusion and within 30 minutes post infusion
measure: Number of Participants With Laboratory Confirmed Hemolysis That Occurred Following Investigational Product Administration
timeFrame: Epoch 1: 3 week IV interval- weeks 0, 10. Epoch 1: 4 week IV interval- weeks 0, 9. Epoch 3: Subcutaneous (SC) week 9. Epoch 4: SC weeks 17, 18, 40
measure: Quality of Life- Pediatric Quality of Life Inventory^TM (PEDS-QL^TM) (Observer: Parent) for the Age Group 2 to 7 Years
timeFrame: Up to 20 months (throughout entire study)
measure: Quality of Life- PEDS-QL^TM (Observer: Participant) for the Age Group 8 to 13 Years of Age
timeFrame: Up to 20 months (throughout entire study)
measure: Quality of Life- Short-Form 36v2 (SF-36v2) for the Age Group 14 Years and Older
timeFrame: Up to 20 months (throughout entire study)
measure: Treatment Satisfaction Questionnaire for Medication (TSQM) - 2 to 12 Years Old
timeFrame: Up to 20 months per subject (throughout entire study)
measure: Treatment Satisfaction Questionnaire for Medication (TSQM) - 13 Years and Older
timeFrame: Up to 20 months per subject (throughout entire study)
measure: Life Quality Index - 2 to 12 Years Old
timeFrame: Up to 20 months (throughout entire study)
measure: Life Quality Index - 13 Years and Older
timeFrame: Up to 20 months per subject (throughout entire study)
Other outcomes:
Locations:
city: Irvine
country: United States
facility: University of California, Irvine
state: California
city: Centennial
country: United States
facility: IMMUNOe International Research Centers
state: Colorado
city: North Palm Beach
country: United States
facility: Allergy Associates of the Palm Beaches
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Louisville
country: United States
facility: Family Allergy and Asthma Research Institute
state: Kentucky
city: New Orleans
country: United States
facility: Children's Hospital New Orleans- LSUHSC School of Medicine
state: Louisiana
city: Plymouth
country: United States
facility: Midwest Immunology Clinical and Infusion Center
state: Minnesota
city: Saint Louis
country: United States
facility: SSM Cardinal Glennon Children's Medical Center
state: Missouri
city: Bronx
country: United States
facility: Montefiore Medical Center Division of Allergy/Immunology
state: New York
city: Oklahoma City
country: United States
facility: Oklahoma Institute of Allergy and Asthma Clinical Research
state: Oklahoma
city: Tulsa
country: United States
facility: Vital Prospects Clinical Research Institute, PC
state: Oklahoma
city: Dallas
country: United States
facility: Dallas Allergy Immunology Research
state: Texas
city: Irving
country: United States
facility: Allergy Asthma and Immunology Clinic PA
state: Texas
city: Layton
country: United States
facility: Rocky Mountain Asthma/Allergy/Immunology
state: Utah
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Montreal
country: Canada
facility: Centre Hospitalier Universitaire (CHU) Sainte-Justine
state: Quebec
city: Montreal
country: Canada
facility: Montreal Children's Hospital- McGill University health Center
state: Quebec
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: Al-Herz W, Bousfiha A, Casanova JL, Chapel H, Conley ME, Cunningham-Rundles C, Etzioni A, Fischer A, Franco JL, Geha RS, Hammarstrom L, Nonoyama S, Notarangelo LD, Ochs HD, Puck JM, Roifman CM, Seger R, Tang ML. Primary immunodeficiency diseases: an update on the classification from the international union of immunological societies expert committee for primary immunodeficiency. Front Immunol. 2011 Nov 8;2:54. doi: 10.3389/fimmu.2011.00054. eCollection 2011.
citation: Conley ME, Notarangelo LD, Etzioni A. Diagnostic criteria for primary immunodeficiencies. Representing PAGID (Pan-American Group for Immunodeficiency) and ESID (European Society for Immunodeficiencies). Clin Immunol. 1999 Dec;93(3):190-7. doi: 10.1006/clim.1999.4799. No abstract available.
citation: Li Z, McCoy B, Engl W, Yel L. Steady-State Serum IgG Trough Levels Are Adequate for Pharmacokinetic Assessment in Patients with Immunodeficiencies Receiving Subcutaneous Immune Globulin. J Clin Immunol. 2021 Aug;41(6):1331-1338. doi: 10.1007/s10875-021-00990-z. Epub 2021 May 26.
citation: Suez D, Krivan G, Jolles S, Stein M, Gupta S, Paris K, van Hagen PM, Brodszki N, Engl W, Leibl H, McCoy B, Yel L. Safety and tolerability of subcutaneous immunoglobulin 20% in primary immunodeficiency diseases from two continents. Immunotherapy. 2019 Aug;11(12):1057-1065. doi: 10.2217/imt-2019-0057. Epub 2019 Jul 3.
citation: Gupta S, Stein M, Hussain I, Paris K, Engl W, McCoy B, Rabbat CJ, Yel L. Tolerability of Ig20Gly during onboarding in patients with primary immunodeficiency diseases. Ann Allergy Asthma Immunol. 2019 Sep;123(3):271-279.e1. doi: 10.1016/j.anai.2019.06.004. Epub 2019 Jun 20.
citation: Paris K, Haddad E, Borte M, Brodszki N, Derfalvi B, Marodi L, Hussain I, Darter A, Engl W, Leibl H, McCoy B, Yel L. Tolerability of subcutaneous immunoglobulin 20%, Ig20Gly, in pediatric patients with primary immunodeficiencies. Immunotherapy. 2019 Apr;11(5):397-406. doi: 10.2217/imt-2018-0088. Epub 2019 Jan 9.
citation: Suez D, Stein M, Gupta S, Hussain I, Melamed I, Paris K, Darter A, Bourgeois C, Fritsch S, Leibl H, McCoy B, Gelmont D, Yel L. Efficacy, Safety, and Pharmacokinetics of a Novel Human Immune Globulin Subcutaneous, 20 % in Patients with Primary Immunodeficiency Diseases in North America. J Clin Immunol. 2016 Oct;36(7):700-12. doi: 10.1007/s10875-016-0327-9. Epub 2016 Aug 31.