Trial Assessing Safety and Efficacy of Gammanorm® in Autoimmune Diseases

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.

Study Overview

• Status: Terminated
• Conditions: Autoimmune Diseases
• Intervention / Treatment: Drug: Gammanorm

• Study Type: Observational
• Enrollment (Actual): 60

Contacts and Locations

Study Locations

• France
  • Paris, France, 75651
    • APHP - Pitié Salpêtrière
  • Rouen, France, 76031
    • Chu de Rouen - Bois Guillaume
• Martinique
  • Fort-de-France, Martinique, 97261
    • CHU de la Martinique- Hopital Pierre Zobda Quitman

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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	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).	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.	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	24 months
Evaluate treatment compliance	Evaluate treatment compliance in terms of number of infusions per week	24 months
Evaluate reasons for stopping the treatment	Evaluate reasons for stopping the treatment by categorizing across the study the reasons subjects stopped treatment.	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)	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)	24 months

Collaborators and Investigators

• Sponsor: Octapharma

Study record dates

Study Major Dates

• Study Start (Actual): November 23, 2016
• Primary Completion (Actual): December 9, 2019
• Study Completion (Actual): December 9, 2019

Study Registration Dates

• First Submitted: November 30, 2017
• First Submitted That Met QC Criteria: August 31, 2018
• First Posted (Actual): September 4, 2018

Study Record Updates

• Last Update Posted (Actual): March 10, 2021
• Last Update Submitted That Met QC Criteria: March 9, 2021
• Last Verified: March 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Autoimmune Diseases
• Other Study ID Numbers: IMMUNORM

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No