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Juvenile Autoimmune Necrotizing Myopathies (MYONECPED)

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ClinicalTrials.gov Identifier: NCT04295785
Recruitment Status : Not yet recruiting
First Posted : March 5, 2020
Last Update Posted : March 5, 2020
Sponsor:
Information provided by (Responsible Party):
Central Hospital, Nancy, France

Brief Summary:
Autoimmune necrotizing myopathies (AINM) in adult patients are characterized by severity of muscle damage, presence of necrosis with little inflammation on muscle biopsy and anti-HMGCR or anti-SRP auto-antibodies. Data on AINM in children are currently lacking. The purpose of this study is to specify the characteristics at AINM diagnosis, treatments and evolution of juvenile AINM with anti-HMGCR or anti-SRP antibodies.

Condition or disease
Immune-Mediated Necrotizing Myopathy

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Study Type : Observational
Estimated Enrollment : 10 participants
Observational Model: Case-Only
Time Perspective: Retrospective
Official Title: Clinical, Paraclinical, Functional Characteristics and Evolution of Juvenile Autoimmune Necrotizing Myopathies in a National Retrospective Cohort
Estimated Study Start Date : March 2020
Estimated Primary Completion Date : November 2020
Estimated Study Completion Date : November 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Muscle Disorders

Group/Cohort
autoimmune necrotizing myopathy beginning before 18



Primary Outcome Measures :
  1. Type of muscle involvment [ Time Frame: At diagnosis ]
    Proximal or distal or axial muscle involvement by muscle testing

  2. Type of cutaneous involvment [ Time Frame: At diagnosis ]
    Rash or Gottron lesions

  3. Other clinical manifestations [ Time Frame: At diagnosis ]
    Dysphagia or dyspnea or fever or interstitial lung involvement or articular involvement or gastrointestinal involvment

  4. Blood CK level [ Time Frame: At diagnosis ]
    Blood CK level

  5. Histology [ Time Frame: At diagnosis ]
    Results of muscle biopsy : necrosis

  6. Histology [ Time Frame: At diagnosis ]
    Results of muscle biopsy : inflammation

  7. MMT score [ Time Frame: At diagnosis ]
    MMT score

  8. CMAS score [ Time Frame: At diagnosis ]
    CMAS score


Secondary Outcome Measures :
  1. Type of drugs used [ Time Frame: At last visit ]
    Drugs ever used : name and duration

  2. Iatrogenic complications [ Time Frame: At last visit ]
    Infectious complications

  3. Iatrogenic complications [ Time Frame: At last visit ]
    Bone complications

  4. Status of the disease at last visit [ Time Frame: At last visit ]
    Status of the disease at last visit : active or remission or death

  5. Number of relapses [ Time Frame: At last visit ]
    Number of relapses

  6. Type of muscle involvment [ Time Frame: At last visit ]
    Proximal or distal or axial muscle involvement by muscle testing

  7. Type of cutaneous involvment [ Time Frame: At last visit ]
    Rash or Gottron lesions

  8. Other clinical manifestations [ Time Frame: At last visit ]
    Dysphagia or dyspnea or fever or interstitial lung involvement or articular involvement or gastrointestinal involvment

  9. Blood CK level [ Time Frame: At last visit ]
    Blood CK level

  10. CMAS score [ Time Frame: At last visit ]
    CMAS score

  11. MMT score [ Time Frame: At last visit ]
    MMT score



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
AINM histologically proved (i.e. predominant muscle fibers necrosis lesions associated with minimal or absent inflammatory lesions), either anti-SRP or anti-HMGCR antibodies positivity and a disease onset before age 18
Criteria

Inclusion Criteria:

  • AINM histologically proved by predominant muscle fibers necrosis lesions associated with minimal or absent inflammatory lesions
  • Either anti-SRP or anti-HMGCR antibodies positivity
  • Disease onset before age 18

Exclusion Criteria:

  • Seronegative necrotizing myopathies
  • Forms compatible with another type of myositis (e.g. dermatomyositis)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04295785


Contacts
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Contact: Irène LEMELLE 03 83 15 45 96 i.lemelle@chru-nancy.fr

Locations
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France
Hopital d'enfants CHRU Nancy
Vandœuvre-lès-Nancy, France, 54 500
Sponsors and Collaborators
Central Hospital, Nancy, France
Publications:

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Responsible Party: Central Hospital, Nancy, France
ClinicalTrials.gov Identifier: NCT04295785    
Other Study ID Numbers: 2019PI192
First Posted: March 5, 2020    Key Record Dates
Last Update Posted: March 5, 2020
Last Verified: January 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Central Hospital, Nancy, France:
Autoimmune necrotizing myopathy
anti-HMGCR antibodies
anti-SRP antibodies
Additional relevant MeSH terms:
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Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases