Trial Assessing Safety and Efficacy of Gammanorm® in Autoimmune Diseases

• Sponsor: Octapharma (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT03656640
• Collaborator: (none)
• Enrollment: 60
• Locations: 3
• Actual Duration (Months): 36.5
• Patients Per Site: 20
• Patients Per Site Per Month: 0.5

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Autoimmune Diseases	Drug: Gammanorm

Study Design

• Study Type: Observational
• Actual Enrollment: 60 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: Nov 23, 2016
• Actual Primary Completion Date: Dec 9, 2019
• Actual Study Completion Date: Dec 9, 2019

Arms and Interventions

Arm	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.

Outcome Measures

Primary Outcome Measures

1. 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

1. 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.

2. 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

3. 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).

4. 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.

5. 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

6. Evaluate treatment compliance [24 months]

   Evaluate treatment compliance in terms of number of infusions per week

7. Evaluate reasons for stopping the treatment [24 months]

   Evaluate reasons for stopping the treatment by categorizing across the study the reasons subjects stopped treatment.

8. 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)

9. 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)

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	APHP - Pitie Salpetriere	Paris		France	75651
2	Chu de Rouen - Bois Guillaume	Rouen		France	76031
3	CHU de la Martinique- Hopital Pierre Zobda Quitman	Fort-de-France		Martinique	97261

Sponsors and Collaborators

• Octapharma

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Octapharma
• ClinicalTrials.gov Identifier: NCT03656640
• Other Study ID Numbers: IMMUNORM
• First Posted: Sep 4, 2018
• Last Update Posted: Mar 10, 2021
• Last Verified: Mar 1, 2021

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Autoimmune Diseases

Study Results

No Results Posted as of Mar 10, 2021