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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
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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT02806986