Trial Assessing Safety and Efficacy of Gammanorm® in Autoimmune Diseases

Autoimmune Diseases Clinical Trial

Official title:

A Prospective, Multicenter Observational Trial Assessing the Safety and Efficacy of Gammanorm® in Autoimmune Diseases

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03656640
• Other study ID #: IMMUNORM
• Status: Terminated
• Start date: November 23, 2016
• Est. completion date: December 9, 2019

Study information

• Verified date: March 2021
• Source: Octapharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 60
• Est. completion date: December 9, 2019
• Est. primary completion date: December 9, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Autoimmune Diseases

Intervention

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.

Locations

Country	Name	City	State
France	APHP - Pitie Salpetriere	Paris
France	Chu de Rouen - Bois Guillaume	Rouen
Martinique	CHU de la Martinique- Hopital Pierre Zobda Quitman	Fort-de-France

Sponsors (1)

Lead Sponsor	Collaborator
Octapharma

Countries where clinical trial is conducted

France,  Martinique, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Evaluate treatment compliance	Evaluate treatment compliance in terms of number of infusions per week	24 months
Secondary	Evaluate reasons for stopping the treatment	Evaluate reasons for stopping the treatment by categorizing across the study the reasons subjects stopped treatment.	24 months
Secondary	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
Secondary	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