Comparison of quality of life and satisfaction in primary immunodeficient patients treated with subcutaneous injections of Gammanorm administered using a pump or a syringe for rapid manual administration
| ISRCTN | ISRCTN55938644 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN55938644 |
| EudraCT/CTIS number | 2014-003746-27 |
| ClinicalTrials.gov number | NCT02503293 |
| Secondary identifying numbers | GAN-06 |
- Submission date
- 02/09/2015
- Registration date
- 15/12/2015
- Last edited
- 28/06/2019
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Haematological Disorders
Plain English summary of protocol
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
Contact information
Scientific
Octapharma Pharmazeutika Prod.Ges.m.b.H.
Oberlaaer Strasse 235
A-1100 Vienna
1100
Austria
Study information
| 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 |
| Study setting(s) | Hospital |
| Study type | Quality of life |
| Participant information sheet | Not available in web format, please use the contact details below to request a patient information sheet |
| 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 |
| Study acronym | GAN-06 |
| Study objectives | 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(s) | 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 |
| Health condition(s) or problem(s) studied | 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 |
| Pharmaceutical study type(s) | |
| Phase | Phase III/IV |
| Drug / device / biological / vaccine name(s) | 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 study start date | 26/11/2014 |
| Completion date | 11/12/2017 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Both |
| Target number of participants | A minimum of 30 patients, but not more than 40 patients will be enrolled in the study. |
| Total final enrolment | 30 |
| Key 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 |
| Key exclusion criteria | Participating in another interventional clinical study and receiving investigational medicinal product within three months before study entry. |
| Date of first enrolment | 25/06/2015 |
| Date of final enrolment | 14/07/2017 |
Locations
Countries of recruitment
- Australia
- England
- Germany
- Italy
- United Kingdom
- Wales
Study participating centres
Breisacher Straße 117
Freiburg
79106
Germany
Leipzig
04129
Germany
Heath Park
Cardiff
CF14 4XW
United Kingdom
Eden Unit, level 7
Derriford Road
Plymouth
PL6 8DH
United Kingdom
4th Floor Pathology & Pharmacy Building
80 Newark Street
London
E1 2ES
United Kingdom
Mindelsohn Way
Edgbaston
Birmingham
B15 2GW
United Kingdom
Via N. Giustiniani, 2
Padova
35128
Italy
Oxford
OX3 9DU
United Kingdom
London
NW3 2QG
United Kingdom
Therry Road
Campell Town
NSW 2560
Australia
Garran
ACT 2605
Australia
Viale del Policlinico, 155
Rome
00161
Italy
Sponsor information
Industry
Oberlaaer Strasse 235
Vienna
1100
Austria
| Phone | +43 1 61032-1796 |
|---|---|
| tatiana.lavrova@Octapharma.com | |
| Website | www.octapharma.com |
"ROR" |
https://ror.org/022k50n33 |
Funders
Funder type
Industry
No information available
Results and Publications
| Intention to publish date | 31/12/2018 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not expected to be made available |
| 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. |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Basic results | 08/02/2019 | 08/02/2019 | No | No | |
| HRA research summary | 28/06/2023 | No | No |
Additional files
- ISRCTN55938644_BasicResults_08Feb2019.pdf
- Uploaded 08/02/2019
Editorial Notes
28/06/2019: Added EudraCT link to basic results (scientific).
26/06/2019: The total final enrolment was added.
08/02/2017: The following changes were made to the trial record:
1. The overall trial end date was changed from 31/12/2017 to 11/12/2017.
2. The basic results of this trial have been uploaded as an additional file.
11/09/2018: The contact details, sponsor contact details and contact in plain English summary were changed from Birgit Taumberger to Tatiana Lavrova
14/08/2018: The following changes have been made to the trial record:
1. Ethics approval from Ethics Committee University "Sapienza" was added.
2. The total target enrolment was changed from 40 to 30
3. The recruitment end date was changed from 31/05/2017 to 14/07/2017
4. Università di Roma "Sapienza" was added as a trial participating centre
5. The plain English summary was updated with Università di Roma "Sapienza" as a study site
6. The intention to publish date has been changed from 31/12/2017 to 31/12/2018
01/02/2017: The target number of participants has been updated from 30 to 40. The overall trial end date has been updated from 31/12/2016 to 31/12/2017 and the recruitment dates have been updated from 07/07/2015 - 30/09/2016 to 25/06/2015 - 31/05/2017. In addition, the ClinicalTrials.gov number for this study has been added.
05/07/2016: the following changes were made to the trial record:
1. The recruitment start date was changed from 25/06/2015 to 07/07/2015.
2. The recruitment end date was changed from 30/06/2016 to 30/09/2016.
3. Australia was added to the countries of recruitment.
4. John Radcliffe Hospital, Royal Free London Hospital, Campbell Town Hospital and Canberra Hospital were added to the trial participating centres.
