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Trial Assessing Safety and Efficacy of Gammanorm® in Autoimmune Diseases

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ClinicalTrials.gov Identifier: NCT03656640
Recruitment Status : Recruiting
First Posted : September 3, 2018
Last Update Posted : September 9, 2019
Sponsor:
Information provided by (Responsible Party):
Octapharma

Brief Summary:
This observational trial assesses the safety and efficacy of Gammanorm® in autoimmune diseases.

Condition or disease Intervention/treatment
Autoimmune Diseases Drug: Gammanorm

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Study Type : Observational
Estimated Enrollment : 80 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Prospective, Multicenter Observational Trial Assessing the Safety and Efficacy of Gammanorm® in Autoimmune Diseases
Actual Study Start Date : November 23, 2016
Estimated Primary Completion Date : November 23, 2020
Estimated Study Completion Date : November 23, 2020

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
Patients Receiving Gammanorm®
Patients Receiving Gammanorm®
Drug: Gammanorm
Patients already receiving Gammanorm will be observed over the course of 2 years every 3 months. Gammanorm will be given standard of care.




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. SMOG score [ Time Frame: 24 months ]
    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.

  2. Rankin score [ Time Frame: 24 months ]
    For patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) and Multifocal motor neuropathy (MMN), efficacy will be assessed using the Rankin score

  3. MRC [ Time Frame: 24 months ]
    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).

  4. ONLS [ Time Frame: 24 months ]
    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.

  5. Muscular Testings and Myositis Functional Rating Scale [ Time Frame: 24 months ]
    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

  6. Evaluate treatment compliance [ Time Frame: 24 months ]
    Evaluate treatment compliance in terms of number of infusions per week

  7. Evaluate reasons for stopping the treatment [ Time Frame: 24 months ]
    Evaluate reasons for stopping the treatment by categorizing across the study the reasons subjects stopped treatment.

  8. Patient satisfaction regarding Gammanorm [ Time Frame: 24 months ]
    Evaluate patient satisfaction with use of patient diaries where the patient will complete a questionnaire on Life Quality Index (LQI)

  9. Patient Quality of Life regarding Gammanorm [ Time Frame: 24 months ]
    Evaluate patient quality of life with use of patient diaries where the patient will complete a questionnaire on quality of life (SF-12)



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
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
Criteria

Inclusion Criteria:

  1. Adult man or woman older than 18 years,
  2. Patient with autoimmune disease such as CIDP, MMN, PM, DM, MI, ITP, NAM or any other off-label use of Gammanorm®,
  3. Patient who has a prescription of Gammanorm® treatment for immunomodulation,
  4. Patient accepting to participate in the study by oral consent after having received oral and written information on the study.

Exclusion Criteria:

  1. Patient treated with Gammanorm® for immune substitution, or other authorized use
  2. Patient who refuses to participate in the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03656640


Contacts
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Contact: Mikaela Grupp 866-337-1868 ctgov@clinicalresearchmgt.com

Locations
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France
APHP - Pitie Salpetriere Not yet recruiting
Paris, France, 75651
Chu de Rouen - Bois Guillaume Recruiting
Rouen, France, 76031
Martinique
CHU de la Martinique- Hopital Pierre Zobda Quitman Recruiting
Fort-de-France, Martinique, 97261
Sponsors and Collaborators
Octapharma

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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT03656640     History of Changes
Other Study ID Numbers: IMMUNORM
First Posted: September 3, 2018    Key Record Dates
Last Update Posted: September 9, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Autoimmune Diseases
Immune System Diseases