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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02806986
Registration number
NCT02806986
Ethics application status
Date submitted
3/06/2016
Date registered
21/06/2016
Titles & IDs
Public title
Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency
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Scientific title
A Multi-Center, Open-Label, Single-Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency
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Secondary ID [1]
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2015-003290-15
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Secondary ID [2]
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GTI1503
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Primary Immunodeficiency
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Condition category
Condition code
Inflammatory and Immune System
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Autoimmune diseases
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Other - IGSC 20%
Experimental: IGSC 20% - 13 doses of IGSC 20% in Treatment Stage 1 and 39 doses of IGSC 20% in Treatment Stage 2 for a total of 52 doses if IGSC 20%
Treatment: Other: IGSC 20%
Weekly administration of IGSC 20% via intravenous infusion
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Intervention code [1]
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Treatment: Other
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Rate of Serious Bacterial Infection (SBI) Per Participant Per Year
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Assessment method [1]
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The rate of SBI events per participant per year during IGSC 20% treatment was calculated as the total number of SBI events divided by the total duration of exposure in years across all participants. The 2-sided 98% confidence interval (CI) was determined from a generalized linear model for poisson regression for the log-transformed number of events with log-transformed duration of exposure in years as an offset variable.
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Timepoint [1]
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IGSC 20% Treatment Stage 1: Week 1 to 13; IGSC 20% Treatment Stage 2: Week 14 to 53; IGSC 20% Overall: Week 1 to 53
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Secondary outcome [1]
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Mean Trough Total IgG Concentration
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Assessment method [1]
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Mean trough total IgG concentration during the previous regimen phase was calculated as the average of the trough concentrations at the previous IVIG (pIV)#1 and pIV#2 visits for participants entering the study on a previous IVIG regimen, or at the previous subcutaneous immune globulin (SCIG) (pSC)#1 and baseline/SC#1 visits for participants entering the study on a previous SCIG regimen. Mean trough total IgG concentration during the IGSC 20% phase was calculated as the average of all steady state trough concentrations measured during the IGSC 20% treatment stage 2 at the visits corresponding to Weeks 17, 18, 20, 24, 28, 32, 36, 40, 44, 48, 52 and 53.
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Timepoint [1]
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Previous Regimen Phase: 2 timepoints pre-dose of pIV or pSC between Screening and Baseline (up to 8 weeks). IGSC 20% Phase: Pre-dose of IGSC 20% at Baseline (Week 1), Weeks 2, 5, 9, 13, 17, 18, 20, 24, 28, 32, 36, 40, 44, 48, 52, and at Week 53
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Secondary outcome [2]
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Rate of Infection of Any Kind Per Participant Per Year
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Assessment method [2]
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The total number of infections of any kind (serious/non-serious including acute sinusitis, exacerbation of chronic sinusitis, acute otitis media, pneumonia, acute bronchitis, infectious diarrhea, etc.) as determined by the investigator were evaluated. The rate of infection events per participant per year during IGSC 20% treatment was calculated as the total number of infection events divided by the total duration of exposure in years across all participants. The 2-sided 95% CI was determined from a generalized linear model for poisson regression for the log-transformed number of events with log-transformed duration of exposure in years as an offset variable.
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Timepoint [2]
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IGSC 20% Treatment Stage 1: Week 1 to 13; IGSC 20% Treatment Stage 2: Week 14 to 53; IGSC 20% Overall: Week 1 to 53
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Secondary outcome [3]
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Rate of Days on Antibiotics Per Participants Per Year
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Assessment method [3]
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The rate of days on antibiotics per participants per year during IGSC 20% treatment was calculated as the total number of days on antibiotic divided by the total duration of exposure in years across all participants. The 2-sided 95% CI was determined from a generalized linear model for poisson regression for the log transformed number of days with log-transformed duration of exposure in years as an offset variable.
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Timepoint [3]
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IGSC 20% Treatment Stage 1: Week 1 to 13; IGSC 20% Treatment Stage 2: Week 14 to 53; IGSC 20% Overall: Week 1 to 53
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Secondary outcome [4]
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Rate of Hospitalization Due to Infection Per Participants Per Year
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Assessment method [4]
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The rate of hospitalization due to infection events per participant per year during IGSC 20% treatment was calculated as the total number of hospitalization due to infection events divided by the total duration of exposure in years across all participants. The 2-sided 95% CI was determined from a generalized linear model for poisson regression for the log-transformed number of events with log-transformed duration of exposure in years as an offset variable.
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Timepoint [4]
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IGSC 20% Treatment Stage 1: Week 1 to 13; IGSC 20% Treatment Stage 2: Week 14 to 53; IGSC 20% Overall: Week 1 to 53
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Secondary outcome [5]
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Rate of Days of Work/School/Daily Activities Missed Per Participants Per Year Due to Infections and Related Treatment
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Assessment method [5]
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The rate of days of work, school or daily activities missed per participant per year during IGSC 20% treatment was calculated as the total number of days of work/school/daily activities missed divided by the total duration of exposure in years across all participants. The 2-sided 95% CI was determined from a generalized linear model for poisson regression for the log-transformed number of days with log transformed duration of exposure in years as an offset variable.
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Timepoint [5]
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IGSC 20% Treatment Stage 1: Week 1 to 13; IGSC 20% Treatment Stage 2: Week 14 to 53; IGSC 20% Overall: Week 1 to 53
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Eligibility
Key inclusion criteria
* Pre-existing diagnosis of PI with features of hypogammaglobulinemia requiring IgG replacement therapy
* No serious bacterial infection within the 3 months prior to Screening and has no serious bacterial infections (SBIs) up to the time of the Baseline Visit
* Currently on IgG replacement therapy (stable regimen [dose and dosing interval] via IV or SC infusion) for = 3 consecutive months at a dosage of at least 200 mg/kg per infusion
* Documented (within previous 3 months) of an IgG trough level of = 500 mg/dL on current IgG replacement therapy regimen
* Screening/pre-Baseline trough IgG levels must be = 500 mg/dL.
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Minimum age
2
Years
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Maximum age
75
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Known serious adverse reaction to immunoglobulin or any severe anaphylactic reaction to blood or any blood-derived product
* History of blistering skin disease, clinically significant thrombocytopenia, bleeding disorder, diffuse rash, recurrent skin infections, or other disorders where SC therapy would be contraindicated during the study
* Isolated IgG subclass deficiency, isolated specific antibody deficiency disorder, or transient hypogammaglobulinemia of infancy
* Nephrotic syndrome, and/or a history of acute renal failure, and/or severe renal impairment, and/or is on dialysis
* Known previous infection with or clinical signs and symptoms consistent with current hepatitis B virus or hepatitis C virus infection
* History of (year prior to Screening or 2 episodes in lifetime) or current diagnosis of deep venous thrombosis or thromboembolism (e.g., myocardial infarction, cerebrovascular accident, or transient ischemic attack)
* Acquired medical condition known to cause secondary immune deficiency, such as chronic lymphocytic leukemia, lymphoma, multiple myeloma, chronic or recurrent neutropenia (absolute neutrophil count less than 1000/µL [1.0 x 10^9/L]), or human immunodeficiency virus infection/acquired immune deficiency syndrome
* HIV positive by nucleic acid amplification technology based on a Screening blood sample
* Uncontrolled arterial hypertension (adult subjects: systolic blood pressure > 160 mmHg and/or diastolic blood pressure > 100 mmHg)
* Receiving any of the following medications: (a) immunosuppressants including chemotherapeutic agents, (b) immunomodulators, (c) long-term systemic corticosteroids defined as daily dose > 1 mg of prednisone equivalent/kg/day for > 30 days Note: Intermittent courses of corticosteroids of not more than 10 days would not exclude a subject. Inhaled or topical corticosteroids are allowed.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/06/2016
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
15/05/2019
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Sample size
Target
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Accrual to date
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Final
61
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Wesley Medical Research - Auchenflower
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Recruitment hospital [2]
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Royal Melbourne Hospital - Parkville
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Recruitment postcode(s) [1]
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4066 - Auchenflower
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Recruitment postcode(s) [2]
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3050 - Parkville
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Recruitment outside Australia
Country [1]
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Czechia
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State/province [1]
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Hradec Kralove
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Country [2]
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France
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State/province [2]
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Montpellier
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Country [3]
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Germany
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State/province [3]
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Dortmund
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Country [4]
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Germany
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State/province [4]
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Hamburg
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Country [5]
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Germany
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State/province [5]
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Leipzig
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Country [6]
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Germany
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State/province [6]
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Mainz
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Country [7]
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Germany
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State/province [7]
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Sankt Augustin
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Country [8]
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Hungary
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State/province [8]
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Budapest
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Country [9]
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Hungary
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State/province [9]
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Nyiregyháza
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Country [10]
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Poland
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State/province [10]
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Warsaw
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Country [11]
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Spain
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State/province [11]
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Barcelona
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Country [12]
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Spain
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State/province [12]
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Cordoba
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Country [13]
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Spain
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State/province [13]
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Madrid
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Country [14]
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Spain
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State/province [14]
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Sevilla
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Country [15]
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Sweden
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State/province [15]
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Stockholm
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Country [16]
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United Kingdom
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State/province [16]
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Cambridge
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Country [17]
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United Kingdom
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State/province [17]
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London
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Country [18]
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United Kingdom
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State/province [18]
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Plymouth
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Grifols Therapeutics LLC
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Approximately 60 subjects will be enrolled in order to have approximately 20 adult subjects and 20 pediatric subjects treated with subcutaneously administered Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) who complete the entire study. This study will include 3 study stages: Screening/Previous Regimen Phase, IGSC 20% Treatment Stage 1 (13 IGSC 20% weekly doses), and IGSC 20% Treatment Stage 2 (39 IGSC 20% weekly doses). A total of 52 doses of IGSC 20% will be administered with a final follow-up visit 1 week after the last dose at Week 53. Subjects/caregivers will be trained on self-administration of IGSC 20% by the clinical site personnel.
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Trial website
https://clinicaltrials.gov/study/NCT02806986
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Phone
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Fax
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Email
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/86/NCT02806986/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/86/NCT02806986/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02806986