Trial Assessing Safety and Efficacy of Gammanorm® in Autoimmune Diseases

Terminated

• Conditions: Autoimmune Diseases
• Interventions: Drug: Gammanorm

• Registration Number: NCT03656640
• Lead Sponsor: Octapharma

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2016-11-23
• Study First Submitted: 2017-11-30
• Study First Submitted QC: 2018-08-31
• Study First Posted: 2018-09-04
• Primary Completion: 2019-12-09
• Study Completion: 2019-12-09
• Status Verified: 2021-03
• Last Update Submitted: 2021-03-09
• Last Update Posted: 2021-03-10

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 60

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.

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Exclusion Criteria

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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients Receiving Gammanorm®	Gammanorm	Patients Receiving Gammanorm®

Primary Outcome Measures

Name	Time	Method
Evaluate the safety of Gammanorm® in terms of adverse event rate in patients receiving the off-label treatment in immunomodulation.	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

Name	Time	Method
Evaluate treatment compliance	24 months	Evaluate treatment compliance in terms of number of infusions per week
Evaluate reasons for stopping the treatment	24 months	Evaluate reasons for stopping the treatment by categorizing across the study the reasons subjects stopped treatment.
Rankin score	24 months	For patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) and Multifocal motor neuropathy (MMN), efficacy will be assessed using the Rankin score
MRC	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).
SMOG score	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.
Muscular Testings and Myositis Functional Rating Scale	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
ONLS	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.
Patient Quality of Life regarding Gammanorm	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)
Patient satisfaction regarding Gammanorm	24 months	Evaluate patient satisfaction with use of patient diaries where the patient will complete a questionnaire on Life Quality Index (LQI)

Trial Locations

Locations (3)

CHU de la Martinique- Hopital Pierre Zobda Quitman; 🇲🇶 Fort-de-France, Martinique

Chu de Rouen - Bois Guillaume; 🇫🇷 Rouen, France

APHP - Pitie Salpetriere; 🇫🇷 Paris, France