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出境医 / 临床实验 / Immunoglobulin M (IgM)-Anti-myelin-associated-glycoprotein(MAG) Peripheral Neuropathy Study (IMAGiNe-Lyon)

Immunoglobulin M (IgM)-Anti-myelin-associated-glycoprotein(MAG) Peripheral Neuropathy Study (IMAGiNe-Lyon)

Study Description
Brief Summary:

There is no international consensus regarding how to assess and treat patients with immunoglobulin M (IgM)-anti-myelin-associated-glycoprotein (MAG) monoclonal gammopathy associated peripheral neuropathy. The purpose of the IMAGiNe study-Lyon is to prospectively collect standardized clinical data of 25 patients with IgM-anti-MAG monoclonal gammopathy associated peripheral neuropathy in order to participate in the international IMAGiNe Study.

The main objective is to describe in detail the various clinical subtypes, clinical course, treatment responses, antibody titers of IgM-anti-MAG monoclonal gammopathy associated peripheral neuropathy using a variety of outcome measures. The obtained observational data will allow the construction of an IgM monoclonal gammopathy associated peripheral neuropathy-specific Rasch-built overall Disability Scale (RODS) that should fulfill all modern clinimetric requirements, including cross-cultural validity. The coordinating centers for the IgM-RODS construction will be the Maastricht University Medical Center and University Medical Center Utrecht, both in the Netherlands.


Condition or disease Intervention/treatment
Immunoglobin M Anti-myelin-associated-glycoprotein Peripheral Neuropathy Other: Clinical examination and patients' questionnaires regarding activity and participation (preliminary Immunogloblin M (IgM)-Rasch-built overall Disability Scale (RODS), and finalized IgM-RODS)

Study Design
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Study Type : Observational
Estimated Enrollment : 25 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Immunoglobulin M (IgM)-Anti-myelin-associated-glycoprotein(MAG) Peripheral Neuropathy Study: From Proper Assessment to Trial Needs - IMAGiNe Study-Lyon
Estimated Study Start Date : September 1, 2020
Estimated Primary Completion Date : September 1, 2026
Estimated Study Completion Date : September 1, 2026
Arms and Interventions
Group/Cohort Intervention/treatment
Immunogloblin M-anti myelin-associated-glycoprotein neuropathy
Patient group (25 subjects) presenting with an Immunogloblin M-anti myelin-associated-glycoprotein peripheral neuropathy
 Other: Clinical examination and patients' questionnaires regarding activity and participation (preliminary Immunogloblin M (IgM)-Rasch-built overall Disability Scale (RODS), and finalized IgM-RODS)
Rasch-built overall Disability Scale (RODS) is a disease-specific, patient-based, linearly weighted scale that captures activity and social participation limitations to detect activity limitations in patients with Immunogloblin M (IgM) anti-myelin-associated-glycoprotein peripheral neuropathy. For this study, the preliminary RODS is a 146 item patient-reported outcome instrument that assesses activity and social participation limitation. Rasch Measurement Methods examine the extent observed data (patients' actual responses to scale items) accord with predictions of those responses from a mathematical model.
Outcome Measures
Primary Outcome Measures :
  1. Change in patient activity and participation according to patient questionnaires [ Time Frame: 0, 6, 12, 24 and 36 months ]
    Preliminary Immunogloblin M (IgM)-Rasch-built Overall Disability Scale (RODS), and after its construction the finalized IgM-RODS. Rasch-built overall Disability Scale (RODS) is a disease-specific, patient-based, linearly weighted scale that captures activity and social participation limitations to detect activity limitations in patients with Immunogloblin M (IgM) anti-myelin-associated-glycoprotein peripheral neuropathy. For this study, the preliminary RODS is a 146 item patient-reported outcome instrument that assesses activity and social participation limitation. Rasch Measurement Methods examine the extent observed data (patients' actual responses to scale items) accord with predictions of those responses from a mathematical model.


Eligibility Criteria
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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
Patients at least 18 years old fulfilling the international criteria for Immunogloblin M (IgM)-anti-myelin-associated-glycoprotein (MAG) monoclonal gammopathy and peripheral neuropathy
Criteria

Inclusion Criteria:

  • fulfilling the international criteria for the diagnosis Immunogloblin M (IgM) monoclonal gammopathy, with anti-myelin-associated-glycoprotein (MAG) antibodies, and peripheral neuropathy.
  • age of 18 years or older followed in neuromuscular department of Lyon;
  • informed consent given by the participant

Exclusion Criteria:

  • concomitant diseases possibly interfering with peripheral nerve and function, as well as physical functioning, such as diabetes, renal insufficiency, (prior) treatment with chemotherapy for diseases other than their Immunogloblin M (IgM) monoclonal gammopathy associated peripheral neuropathy, history of hereditary neuropathy, vitamin deficiency, connective tissue disorder, hepatitis B and C and human immunodeficiency virus (HIV) infection, alcohol abuse (more than 5 International Units(IU)/day);
  • an active malignancy with poor prognosis, undergoing treatment aside from monoclonal gammopathy associated peripheral neuropathy;
  • pregnant;
  • the use of any medication that may cause peripheral nerve function
Contacts and Locations

Contacts
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Contact: Juliette SVAHN, MD 4 72 11 66 08 ext 33 juliette.svahn@chu-lyon.fr

Locations
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France
Hospices Civils de Lyon Service Neurologie C, Pathologies Neuromusculaires, Unité 302
Bron, France, 69500
Contact: Juliette SVAHN, MD         
Principal Investigator: Juliette SVAHN, MD         
Sponsors and Collaborators
Hospices Civils de Lyon
Investigators
Layout table for investigator information
Principal Investigator: Juliette SVAHN, MD Hospices Civils de Lyon
Tracking Information
First Submitted Date April 15, 2019
First Posted Date April 17, 2019
Last Update Posted Date July 29, 2020
Estimated Study Start Date September 1, 2020
Estimated Primary Completion Date September 1, 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 15, 2019) 		Change in patient activity and participation according to patient questionnaires [ Time Frame: 0, 6, 12, 24 and 36 months ]
Preliminary Immunogloblin M (IgM)-Rasch-built Overall Disability Scale (RODS), and after its construction the finalized IgM-RODS. Rasch-built overall Disability Scale (RODS) is a disease-specific, patient-based, linearly weighted scale that captures activity and social participation limitations to detect activity limitations in patients with Immunogloblin M (IgM) anti-myelin-associated-glycoprotein peripheral neuropathy. For this study, the preliminary RODS is a 146 item patient-reported outcome instrument that assesses activity and social participation limitation. Rasch Measurement Methods examine the extent observed data (patients' actual responses to scale items) accord with predictions of those responses from a mathematical model.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Immunoglobulin M (IgM)-Anti-myelin-associated-glycoprotein(MAG) Peripheral Neuropathy Study
Official Title Immunoglobulin M (IgM)-Anti-myelin-associated-glycoprotein(MAG) Peripheral Neuropathy Study: From Proper Assessment to Trial Needs - IMAGiNe Study-Lyon
Brief Summary

There is no international consensus regarding how to assess and treat patients with immunoglobulin M (IgM)-anti-myelin-associated-glycoprotein (MAG) monoclonal gammopathy associated peripheral neuropathy. The purpose of the IMAGiNe study-Lyon is to prospectively collect standardized clinical data of 25 patients with IgM-anti-MAG monoclonal gammopathy associated peripheral neuropathy in order to participate in the international IMAGiNe Study.

The main objective is to describe in detail the various clinical subtypes, clinical course, treatment responses, antibody titers of IgM-anti-MAG monoclonal gammopathy associated peripheral neuropathy using a variety of outcome measures. The obtained observational data will allow the construction of an IgM monoclonal gammopathy associated peripheral neuropathy-specific Rasch-built overall Disability Scale (RODS) that should fulfill all modern clinimetric requirements, including cross-cultural validity. The coordinating centers for the IgM-RODS construction will be the Maastricht University Medical Center and University Medical Center Utrecht, both in the Netherlands.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Patients at least 18 years old fulfilling the international criteria for Immunogloblin M (IgM)-anti-myelin-associated-glycoprotein (MAG) monoclonal gammopathy and peripheral neuropathy
Condition Immunoglobin M Anti-myelin-associated-glycoprotein Peripheral Neuropathy
Intervention Other: Clinical examination and patients' questionnaires regarding activity and participation (preliminary Immunogloblin M (IgM)-Rasch-built overall Disability Scale (RODS), and finalized IgM-RODS)
Rasch-built overall Disability Scale (RODS) is a disease-specific, patient-based, linearly weighted scale that captures activity and social participation limitations to detect activity limitations in patients with Immunogloblin M (IgM) anti-myelin-associated-glycoprotein peripheral neuropathy. For this study, the preliminary RODS is a 146 item patient-reported outcome instrument that assesses activity and social participation limitation. Rasch Measurement Methods examine the extent observed data (patients' actual responses to scale items) accord with predictions of those responses from a mathematical model.
Study Groups/Cohorts Immunogloblin M-anti myelin-associated-glycoprotein neuropathy
Patient group (25 subjects) presenting with an Immunogloblin M-anti myelin-associated-glycoprotein peripheral neuropathy
Intervention: Other: Clinical examination and patients' questionnaires regarding activity and participation (preliminary Immunogloblin M (IgM)-Rasch-built overall Disability Scale (RODS), and finalized IgM-RODS)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Not yet recruiting
Estimated Enrollment
 (submitted: April 15, 2019) 		25
Original Estimated Enrollment Same as current
Estimated Study Completion Date September 1, 2026
Estimated Primary Completion Date September 1, 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • fulfilling the international criteria for the diagnosis Immunogloblin M (IgM) monoclonal gammopathy, with anti-myelin-associated-glycoprotein (MAG) antibodies, and peripheral neuropathy.
  • age of 18 years or older followed in neuromuscular department of Lyon;
  • informed consent given by the participant

Exclusion Criteria:

  • concomitant diseases possibly interfering with peripheral nerve and function, as well as physical functioning, such as diabetes, renal insufficiency, (prior) treatment with chemotherapy for diseases other than their Immunogloblin M (IgM) monoclonal gammopathy associated peripheral neuropathy, history of hereditary neuropathy, vitamin deficiency, connective tissue disorder, hepatitis B and C and human immunodeficiency virus (HIV) infection, alcohol abuse (more than 5 International Units(IU)/day);
  • an active malignancy with poor prognosis, undergoing treatment aside from monoclonal gammopathy associated peripheral neuropathy;
  • pregnant;
  • the use of any medication that may cause peripheral nerve function
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Juliette SVAHN, MD 4 72 11 66 08 ext 33 juliette.svahn@chu-lyon.fr
Listed Location Countries France
Removed Location Countries  
 
Administrative Information
NCT Number NCT03918421
Other Study ID Numbers 69HCL19_0217
2019-A00884-53 ( Other Identifier: ID-RCB )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Hospices Civils de Lyon
Study Sponsor Hospices Civils de Lyon
Collaborators Not Provided
Investigators
Principal Investigator: Juliette SVAHN, MD Hospices Civils de Lyon
PRS Account Hospices Civils de Lyon
Verification Date July 2020

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