Plain English Summary
Background and study aims
Primary immunodeficiency disorders (PID) are caused by an inherited defect in the immune system, which makes a person more susceptible to infection. There are more than 200 of these disorders, the symptoms of which can vary greatly, ranging from very mild or non-existent (asymptomatic) to severe and debilitating. Gamma globulins are proteins found in the blood plasma. There are different types of gamma globulins, but the most important are immunoglobulins, also known as antibodies. The immune system uses antibodies to recognize and fight infections. In many of the PID’s, people do not have enough of these gamma globulins in their blood, and so are given injections of immunoglobulins to help strengthen their immune systems. When a patient receives this treatment, the injections are often done by the patient themselves at home. The injections can be given using a syringe or an automatic pump (which delivers the dose automatically over a period of time). This study aims to find out whether patients are happier using a syringe or an automatic pump method for receiving their injections.
Who can participate?
Adults with primary immunodeficiency who have received injections of immunoglobulin at use using an automatic pump or syringe for at least one month.
What does the study involve?
Participants are randomly assigned into two groups, each of which receives the treatments in a different order. Each patient is treated for three months with the first treatment option, and then treated for 3 months with the second treatment option (i.e. syringe and then pump, or pump and then syringe). Patients complete a questionnaire about how satisfied they are with each of the treatment options at the end of each three month treatment period.
What are the possible benefits and risks of participating?
Participants will benefit from learning a new technique for administering their immunoglobulin treatment at home, which could be preferable than their current technique. Risks of participating involve the minor risks associated with repeated blood tests, such as pain, bruising or infection.
Where is the study run from?
1. University Medical Center Freiburg (Germany)
2. Klinikum St. Georg (Germany)
3. University Hospital of Wales (UK)
4. Derriford Hospital (UK)
5. The Royal London Hospital (UK)
6. University Hospitals Birmingham (UK)
7. Padova Hospital (Italy)
8. John Radcliffe Hospital, Oxford (UK)
9. Royal Free London Hospital (UK)
10. Campbell Town Hospital (Australia)
11. Canberra Hospital (Australia)
12. Università di Roma "Sapienza", Policlinic Umberto I, Rome (Italy)
When is the study starting and how long is it expected to run for?
November 2014 to December 2017
Who is funding the study?
Octapharma (Austria)
Who is the main contact?
Tatiana Lavrova
tatiana.lavrova@octapharma.com
Trial website
Additional identifiers
EudraCT number
2014-003746-27
ClinicalTrials.gov number
NCT02503293
Protocol/serial number
GAN-06
Study information
Scientific title
A randomised, cross-over study to compare quality of life and satisfaction in primary immunodeficient patients treated with subcutaneous injections of Gammanorm® 165 mg/mL administered with two different delivery devices: injections using pump or rapid push
Acronym
GAN-06
Study hypothesis
The administration of Gammanorm 165 mg/ml using a syringe is not inferior to the administration of Gammanorm 165 mg/ml using a pump regarding patient satisfaction.
Ethics approval
1. Central Ethics Committee, Albert Ludwig University(Germany), 09/02/2015, ref: 561/14(FF-MC)
2. Research Ethics Service, Wales (UK), 12/06/2015, ref: 15/WA/0047
3. Comitato Etico per la Sperimentazione Clinica della Provincia di Padova, 21/04/2016, ref: NRC AOP0707
4. ACT Health, Research Ethics and Governance Office, 21/06/2016, ref: ETH.6.16.113E
5. South Western Sydney Local Health District, 30/05/2016; ref: HREC/16/LPOOL/44
6. Ethics Committee University "Sapienza", 03/03/2017, ref: 4359
Study design
A non-inferiority comparative interventional multi-centre prospective longitudinal randomised open-label cross-over study
Primary study design
Interventional
Secondary study design
Randomised cross over trial
Trial setting
Hospitals
Trial type
Quality of life
Patient information sheet
Not available in web format, please use the contact details below to request a patient information sheet
Condition
Primary immunodeficiency
Intervention
Participants are randomly allocated to one of two groups, who receive the two different treatments in a different order (pump then syringe or syringe then pump). Treatment will be administered subcutaneously at home by the patient.
Pump treatment: The usual dose is 0.6 mL (100 mg) of Gammanorm® 165 mg/mL per kg of body weight once a week, which can be administered at several infusion sites.
Syringe treatment: The usual dose is 0.6 mL (100 mg) of Gammanorm® 165 mg/mL per kg of body weight per week. The weekly dose could be divided into three injections administered every other day at a single infusion site.
Participants use the pump treatment and the syringe treatment for a total of three months, with no wash-out period in between.
Intervention type
Drug
Phase
Phase III/IV
Drug names
Gammanorm
Primary outcome measure
Patient satisfaction is measured using the life quality index (LQI) questionnaire at the end of each 3 month treatment period.
Secondary outcome measures
1. Therapy-related problems and therapy setting is measured using LQI sub-scores at baseline, 3 months and 6 months
2. Patient satisfaction is measured using the Treatment Satisfaction Questionnaire for Medication (TSQM-11) at baseline, 3 months and 6 months
3. Patient preference is measured by patient answer at 6 months
4. Efficacy (clinical efficacy and residual levels of IgG) of Gammanorm® 165 mg/mL is measured by evaluation of IgE level at baseline, 3 months and 6 months
5. Systemic and local tolerability of Gammanorm® 165 mg/mL is measured by evaluation of the diary and AE Reporting during ongoing study
6. Burden of illness is measured using the PRISM test at at baseline, 3 months and 6 months
7. Burden of subcutaneous immunoglobulin treatment delivery device is measured using the PRISM test at at baseline, 3 months and 6 months
8. Costs measured by analyses of the patient diary at 6 months
Overall trial start date
26/11/2014
Overall trial end date
31/12/2017
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
1. Adult patients (≥ 18 years).
2. Presenting with primary immunodeficiency.
3. Having received subcutaneous injections of immunoglobulin at home using an automatic pump or syringe for at least 1 month at the time of inclusion.
4. For whom the investigator decides to maintain immunoglobulin replacement therapy with subcutaneous injections of Gammanorm® 165 mg/mL at home.
5. Women of childbearing potential must have a negative result on a pregnancy test (human chorionic gonadotropine [HCG]-based assay) and need to practice contraception using a method of proven reliability for the duration of the study
Participant type
Patient
Age group
Adult
Gender
Both
Target number of participants
A minimum of 30 patients, but not more than 40 patients will be enrolled in the study.
Participant exclusion criteria
Participating in another interventional clinical study and receiving investigational medicinal product within three months before study entry.
Recruitment start date
25/06/2015
Recruitment end date
14/07/2017
Locations
Countries of recruitment
Australia, Germany, Italy, United Kingdom
Trial participating centre
University Medical Center Freiburg
Centre of Chronic Immunodeficiency
Breisacher Straße 117
Freiburg
79106
Germany
Trial participating centre
Klinikum St. Georg
Delitzscher Street 141
Leipzig
04129
Germany
Trial participating centre
University Hospital of Wales
Dept. of Biochemistry & Immunology
Heath Park
Cardiff
CF14 4XW
United Kingdom
Trial participating centre
Derriford Hospital
Department of Immunology and Allergy
Eden Unit, level 7
Derriford Road
Plymouth
PL6 8DH
United Kingdom
Trial participating centre
The Royal London Hospital
Barts Health NHS Trust
4th Floor Pathology & Pharmacy Building
80 Newark Street
London
E1 2ES
United Kingdom
Trial participating centre
University Hospitals Birmingham
Department of Immunology
Mindelsohn Way
Edgbaston
Birmingham
B15 2GW
United Kingdom
Trial participating centre
Padova Hospital (Azienda Ospedaliera di Padova)
Dipartimento di Medicina (DIMED)
Via N. Giustiniani, 2
Padova
35128
Italy
Trial participating centre
John Radcliff Hospital
Department of Clinical Immunology
Oxford
OX3 9DU
United Kingdom
Trial participating centre
Royal Free London Hospital
Department of Immunology
London
NW3 2QG
United Kingdom
Trial participating centre
Campell Town Hospital
Unit Immunology and Allergy
Therry Road
Campell Town
NSW 2560
Australia
Trial participating centre
Canberra Hospital
Yamba Dr.
Garran
ACT 2605
Australia
Trial participating centre
Università di Roma "Sapienza"
Policlinic Umberto I
Viale del Policlinico, 155
Rome
00161
Italy
Sponsor information
Organisation
Octapharma Pharmazeutika Prod.Ges.m.b.H.
Sponsor details
Oberlaaer Strasse 235
Vienna
1100
Austria
+43 1 61032-1796
tatiana.lavrova@Octapharma.com
Sponsor type
Industry
Website
Funders
Funder type
Industry
Funder name
Octapharma
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
A clinical study report complying with relevant guidelines will be written following statistical analyses. This document will be reviewed and approved by the Sponsor and the principal investigator.
Intention to publish date
31/12/2018
Participant level data
Not expected to be available
Basic results (scientific)
Publication list