Study regarding the safety of Gammanorm in autoimmune diseases
| ISRCTN | ISRCTN32265704 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN32265704 |
| Secondary identifying numbers | GAN-08 |
- Submission date
- 15/06/2016
- Registration date
- 05/07/2016
- Last edited
- 01/07/2016
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Skin and Connective Tissue Diseases
Plain English summary of protocol
Background and study aims
Immunotherapy is a drugs therapy that can be used to treat diseases that affect the immune system. Gammanorm® is a immunoglobulin and contains antibodies against bacteria and viruses. It is used as a replacement antibody therapy in both adults and children to treat a number of immune system diseases, for example, severe combined immunodeficiency, common variable immunodeficiency (CVID), IgG subclass deficiencies and myeloma or chronic lymphatic leukaemia with severe secondary hypogammaglobulinaemia and recurrent infections. However, it can also be used “off label” in treating other conditions (that is, conditions it has not been licenced to treat). The main name of this study is to assess the safety of Gammanorm® for “off-label” uses.
Who can participate?
Adults aged at least 18 and treated with Gammanorm® for an autoimmune disease, such as chronic inflammatory demyelinating polyradiculoneuropathy, multifocal motor neuropathy, dermatomyositis , polymyositis, inclusion body myositis, idiopathic thrombocytopenic purpura , and necrotizing myopathy.
What does the study involve?
Participants are treated with Gammanorm® from between a few months to several years, depending on their condition, typically being given the drug twice a week. The follow-up period for each patient varies, depending on how long they receive Gammanorm® , but is for the maximum of two years.
During this period, information on adverse effects of taking the drug, whether the participants stick to the treatment regime and how well the treatment works is collected.
What are the possible benefits and risks of participating?
Not provided at time of registration
Where is the study run from?
1. Hospital Center University Rouen (France)
2. University Hospital Of Martinique (France)
When is the study starting and how long is it expected to run for?
January 2015 to December 2020
Who is funding the study?
Octapharma France
Who is the main contact?
Dr Jean-Christophe Delain
Contact information
Scientific
62 bis avenue André Morizet
Boulogne Billancourt
92100
France
Study information
| Study design | Observational prospective, multicenter study |
|---|---|
| Primary study design | Observational |
| Secondary study design | Longitudinal study |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Scientific title | Prospective, observational, multicenter study regarding the safety of Gammanorm in autoimmune diseases |
| Study acronym | Immunorm Study |
| Study objectives | The marketing authorization of SCIg (human normal immunoglobulin), especially Gammanorm® is currently restricted to immunosubstitution indications. However, pharmacovigilance show SCIg use in immunomodulation indication, in the absence of a therapeutic alternative to replacing IVIg tolerance reasons. Thus the main objective of this study is to regulate this practice in accordance with our obligations evaluating the safety of treatment with Gammanorm® in various off-label uses. |
| Ethics approval(s) | No Ethical approval is needed in such study. Only the "CCTIRS" (evaluation of collected data) and "CNIL" (evaluation and safety of the data/database) is needed in France for an observational project. |
| Health condition(s) or problem(s) studied | Autoimmune diseases which needs an immunoglobulin treatment - including dermatomyositis, polymyositis, inclusion body myositis, MMN, CIDP |
| Intervention | The study will be conducted in adult patients (>18 years old) treated with Gammanorm® for a autoimmune disease, such as chronic inflammatory demyelinating polyradiculoneuropathy, multifocal motor neuropathy, dermatomyositis , polymyositis, inclusion body myositis, idiopathic thrombocytopenic purpura , necrotizing myopathy or any other off-label use. The main objective is safety so the appearance of adverse events will be noted. Gammanorm® is a subcutaneous polyvalent immunoglobulins preparation at 16,5% (165mg/ml. Typical dosage is around 2 g/kg/month. Typical frequency of administration is twice a week. Treatment duration depends on the patient and could be anywhere from a few months to several years. The follow-up period also depends on how long the patient has been treated for but the maximum follow-up period is two years. |
| Intervention type | |
| Primary outcome measure | Occurrence of adverse events, by counting and evaluating these events as soon as they occur. The physician completes the Adverse event declaration page in the eCRF and this adverse event is automatically declared in pharmacovigilance. information coklected includes: 1. Type of event 2. The outcomes 3. Severity 4. Duration 5. The link with Gammanorm 6. Actions (stop of the treatment, corrective treatment,...) |
| Secondary outcome measures | 1. Efficacy of treatment: 1.1. For ITP (Immune Thrombocytopenic Purpura ) : SMOG Score and echography of spleen 1.2. For NMM (Multifocal motor neuropathy) and CIDP (Chronic Inflammatory Demyelinating Polyneuropathy) : Rankin modified score, and MRC (Medical research council of Great Britain) and ONLS (Overall Neuropathy Limitation Scale) 1.3. For dermatomyositis, Polymyositis or Inclusion body myositis or necrotic myositis : a muscular testing, a myostis functional scale 2. Treatment compliance and therapeutic regimen, monitored using the following data: 2.1. Dosage for a month 2.2. Number of perfusions during a week 2.3. Dosage (in g/kg) 2.4. Speed infusion 2.5. Batch numbers 2.6. Number of vials since the last visit 3. Evaluate patient satisfaction (LQI scale) and quality of life (SF12 scale) Measured at baseline and then every 3 months and at study end. |
| Overall study start date | 22/01/2015 |
| Completion date | 31/12/2020 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Sex | Both |
| Target number of participants | 80 |
| Key inclusion criteria | 1. Adult patient 2. Patient with an autoimmune disease like CIDP, MMN, Polymyositis, Dermatomyositis, Inclusion body myositis, necrotizing myopathy or other autoimmune dideases 3. Patient treated by Gammanorm® 4. Patient had given oral consent after patient information note |
| Key exclusion criteria | 1. Patient with Gammanorm® and with primary or secondary immunodeficiency 2. Patient does not wish to participate |
| Date of first enrolment | 01/09/2016 |
| Date of final enrolment | 01/09/2018 |
Locations
Countries of recruitment
- France
Study participating centres
Rouen
76000
France
France
Sponsor information
Industry
62bis avenue André Morizet
Boulogne Billancourt
92100
France
"ROR" |
https://ror.org/02xqq7c82 |
|---|
Funders
Funder type
Industry
No information available
Results and Publications
| Intention to publish date | 31/12/2020 |
|---|---|
| Individual participant data (IPD) Intention to share | Yes |
| IPD sharing plan summary | Available on request |
| Publication and dissemination plan | A publication is expected after the clinical study report of the study : expected end of 2020 abstracts and posters will be made to communicate on progress: depending of inclusions |
| IPD sharing plan |
