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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
Date last updated
16/06/2020

Titles & IDs
Public title
Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency
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
Secondary ID [1] 0 0
2015-003290-15
Secondary ID [2] 0 0
GTI1503
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Immunodeficiency 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Rate of Serious Bacterial Infection (SBI) Per Participant Per Year
Timepoint [1] 0 0
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
Secondary outcome [1] 0 0
Mean Trough Total IgG Concentration
Timepoint [1] 0 0
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
Secondary outcome [2] 0 0
Rate of Infection of Any Kind Per Participant Per Year
Timepoint [2] 0 0
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
Secondary outcome [3] 0 0
Rate of Days on Antibiotics Per Participants Per Year
Timepoint [3] 0 0
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
Secondary outcome [4] 0 0
Rate of Hospitalization Due to Infection Per Participants Per Year
Timepoint [4] 0 0
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
Secondary outcome [5] 0 0
Rate of Days of Work/School/Daily Activities Missed Per Participants Per Year Due to Infections and Related Treatment
Timepoint [5] 0 0
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

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.
Minimum age
2 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
15/05/2019
Sample size
Target
Accrual to date
Final
61
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Wesley Medical Research - Auchenflower
Recruitment hospital [2] 0 0
Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
4066 - Auchenflower
Recruitment postcode(s) [2] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
Czechia
State/province [1] 0 0
Hradec Kralove
Country [2] 0 0
France
State/province [2] 0 0
Montpellier
Country [3] 0 0
Germany
State/province [3] 0 0
Dortmund
Country [4] 0 0
Germany
State/province [4] 0 0
Hamburg
Country [5] 0 0
Germany
State/province [5] 0 0
Leipzig
Country [6] 0 0
Germany
State/province [6] 0 0
Mainz
Country [7] 0 0
Germany
State/province [7] 0 0
Sankt Augustin
Country [8] 0 0
Hungary
State/province [8] 0 0
Budapest
Country [9] 0 0
Hungary
State/province [9] 0 0
Nyiregyháza
Country [10] 0 0
Poland
State/province [10] 0 0
Warsaw
Country [11] 0 0
Spain
State/province [11] 0 0
Barcelona
Country [12] 0 0
Spain
State/province [12] 0 0
Cordoba
Country [13] 0 0
Spain
State/province [13] 0 0
Madrid
Country [14] 0 0
Spain
State/province [14] 0 0
Sevilla
Country [15] 0 0
Sweden
State/province [15] 0 0
Stockholm
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Cambridge
Country [17] 0 0
United Kingdom
State/province [17] 0 0
London
Country [18] 0 0
United Kingdom
State/province [18] 0 0
Plymouth

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Grifols Therapeutics LLC
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT02806986
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02806986