Autoimmune DiseasesNCT03656640
Trial Assessing Safety and Efficacy of Gammanorm® in Autoimmune Diseases
An observational study of Autoimmune Diseases, run by Octapharma at 3 sites in 2 countries.
Trial summary
This observational trial assesses the safety and efficacy of Gammanorm® in autoimmune diseases. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Diseases
- What is being tested?
- Gammanorm
- How many people
- 60 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 23 Nov 2016to 9 Dec 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult men and women over 18 prescribed Gammanorm with autoimmune disease or any other off-label use of Gammanorm who are willing to participate in the study
Who may be able to join
- Adult man or woman older than 18 years,
- Patient with autoimmune disease such as CIDP, MMN, PM, DM, MI, ITP, NAM or any other off-label use of Gammanorm®,
- Patient who has a prescription of Gammanorm® treatment for immunomodulation,
- Patient accepting to participate in the study by oral consent after having received oral and written information on the study.
Who cannot join
- Patient treated with Gammanorm® for immune substitution, or other authorized use
- Patient who refuses to participate in the study.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
France2 sites
APHP - Pitie Salpetriere
Paris, 75651
Chu de Rouen - Bois Guillaume
Rouen, 76031
Martinique1 site
CHU de la Martinique- Hopital Pierre Zobda Quitman
Fort-de-France, 97261
About this study
Official title: A Prospective, Multicenter Observational Trial Assessing the Safety and Efficacy of Gammanorm® in Autoimmune Diseases
This observational trial assesses the safety and efficacy of Gammanorm® in autoimmune diseases.
- Conditions
- Autoimmune Diseases
- Being tested
- Gammanorm
- Medical subject headings
- Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GammanormDrug
Patients already receiving Gammanorm will be observed over the course of 2 years every 3 months. Gammanorm will be given standard of care.
| Study group | Role | What they receive |
|---|---|---|
| Patients Receiving Gammanorm® | Patients Receiving Gammanorm® Receives: Drug: Gammanorm |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the safety of Gammanorm® in terms of adverse event rate in patients receiving the off-label treatment in immunomodulation.
Evaluate the safety of Gammanorm® in terms of adverse event rate in patients receiving the off-label treatment in immunomodulation.
Measured: 24 months
Secondary outcomes (9)
SMOG score
For patients with idiopathic thrombocytopenic purpura, efficacy will be assessed using the SMOG score. SMOG score : Bleeding manifestations are grouped into three major domains: skin (S), visible mucosae (M), and organs (O), with gradation of severity (SMOG). Each bleeding manifestation is assessed at the time of examination. Severity is graded from 0 to 3 or 4, with grade 5 for any fatal bleeding.
Measured: 24 months
Rankin score
For patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) and Multifocal motor neuropathy (MMN), efficacy will be assessed using the Rankin score
Measured: 24 months
MRC
For patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) and Multifocal motor neuropathy (MMN), efficacy will be assessed using the MRC. MRC (Medical research council) scale for testing muscle : The muscle scale grades muscle power on a scale of 0 to 5 in relation to the maximum expected for that muscle (5= Normal power , 0= No contraction visible or palpable).
Measured: 24 months
ONLS
For patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) and Multifocal motor neuropathy (MMN), efficacy will be assessed using the ONLS (overall neuropathy limitations score). Score ONLS (Overall Neuropathy Limitation Scale) : The OLNS focuses on upper and lower limb functions, and consists of a checklist for interviewing patients. It is scored from 0 to 5 on the upper limb section and from 0 to 7 on the lower limb section. A score of 0 indicates no limitations (the ceiling of the scale) and a score of 5 or 7 indicates no purposeful movement.
Measured: 24 months
Muscular Testings and Myositis Functional Rating Scale
For patients with inflammatory myopathies including dermatomyositis (DM), polymyositis (PM), inclusion body myositis (IBM), and autoimmune necrotizing myopathy (AINM), efficacy will be assessed using the muscular testings and myositis functional rating scale
Measured: 24 months
Evaluate treatment compliance
Evaluate treatment compliance in terms of number of infusions per week
Measured: 24 months
Evaluate reasons for stopping the treatment
Evaluate reasons for stopping the treatment by categorizing across the study the reasons subjects stopped treatment.
Measured: 24 months
Patient satisfaction regarding Gammanorm
Evaluate patient satisfaction with use of patient diaries where the patient will complete a questionnaire on Life Quality Index (LQI)
Measured: 24 months
Patient Quality of Life regarding Gammanorm
Evaluate patient quality of life with use of patient diaries where the patient will complete a questionnaire on quality of life (SF-12)
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Sep 2018
- Study start23 Nov 2016
- Primary completion9 Dec 2019
- Study completion9 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune DiseasesStudies of the same condition, open and under way.
- More trials run by OctapharmaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03656640
- Sponsor's protocol ID
- IMMUNORM
- Status last verified
- Mar 2021
- First submitted
- 30 Nov 2017
- Last updated
- 10 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter Observational Trial Assessing the Safety and Efficacy of Gammanorm® in Autoimmune Diseases
- ClinicalTrials.gov ID
- NCT03656640
- Acronym / sponsor ID
- IMMUNORM
- Conditions
- Autoimmune Diseases
- Study type
- Observational
- Recruitment status
- Terminated
- Enrolment
- 60 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- 23 Nov 2016 (actual)
- Primary completion
- 9 Dec 2019 (actual)
- Study completion
- 9 Dec 2019 (actual)
- First posted
- 4 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This observational trial assesses the safety and efficacy of Gammanorm® in autoimmune diseases.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients Receiving Gammanorm® | – | Drug: GammanormPatients Receiving Gammanorm® |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult men and women over 18 prescribed Gammanorm with autoimmune disease or any other off-label use of Gammanorm who are willing to participate in the study
Key inclusion criteria (4 listed)
- Adult man or woman older than 18 years,
- Patient with autoimmune disease such as CIDP, MMN, PM, DM, MI, ITP, NAM or any other off-label use of Gammanorm®,
- Patient who has a prescription of Gammanorm® treatment for immunomodulation,
- Patient accepting to participate in the study by oral consent after having received oral and written information on the study.
Key exclusion criteria (2 listed)
- Patient treated with Gammanorm® for immune substitution, or other authorized use
- Patient who refuses to participate in the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GammanormPatients already receiving Gammanorm will be observed over the course of 2 years every 3 months. Gammanorm will be given standard of care. | Drug | Patients Receiving Gammanorm® |
Primary outcome
Evaluate the safety of Gammanorm® in terms of adverse event rate in patients receiving the off-label treatment in immunomodulation.
Time frame: 24 months
Evaluate the safety of Gammanorm® in terms of adverse event rate in patients receiving the off-label treatment in immunomodulation.
9 secondary outcomes
SMOG score
Time frame: 24 months
Rankin score
Time frame: 24 months
MRC
Time frame: 24 months
ONLS
Time frame: 24 months
Muscular Testings and Myositis Functional Rating Scale
Time frame: 24 months
Evaluate treatment compliance
Time frame: 24 months
Evaluate reasons for stopping the treatment
Time frame: 24 months
Patient satisfaction regarding Gammanorm
Time frame: 24 months
Patient Quality of Life regarding Gammanorm
Time frame: 24 months
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2021.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Autoimmune Diseases, run by Octapharma at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 10 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in France, Martinique. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early. Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the safety of Gammanorm® in terms of adverse event rate in patients receiving the off-label treatment in immunomodulation. (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2021.