Juvenile Autoimmune Necrotizing Myopathies
- Conditions
- Immune-Mediated Necrotizing Myopathy
- Registration Number
- NCT04295785
- Lead Sponsor
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 10
- 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
- Seronegative necrotizing myopathies
- Forms compatible with another type of myositis (e.g. dermatomyositis)
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Type of cutaneous involvment At diagnosis Rash or Gottron lesions
Type of muscle involvment At diagnosis Proximal or distal or axial muscle involvement by muscle testing
Histology At diagnosis Results of muscle biopsy : inflammation
MMT score At diagnosis MMT score
Blood CK level At diagnosis Blood CK level
Other clinical manifestations At diagnosis Dysphagia or dyspnea or fever or interstitial lung involvement or articular involvement or gastrointestinal involvment
CMAS score At diagnosis CMAS score
- Secondary Outcome Measures
Name Time Method CMAS score At last visit CMAS score
Iatrogenic complications At last visit Bone complications
Number of relapses At last visit Number of relapses
Type of muscle involvment At last visit Proximal or distal or axial muscle involvement by muscle testing
MMT score At last visit MMT score
Status of the disease at last visit At last visit Status of the disease at last visit : active or remission or death
Type of cutaneous involvment At last visit Rash or Gottron lesions
Other clinical manifestations At last visit Dysphagia or dyspnea or fever or interstitial lung involvement or articular involvement or gastrointestinal involvment
Type of drugs used At last visit Drugs ever used : name and duration
Blood CK level At last visit Blood CK level
Trial Locations
- Locations (1)
Hopital d'enfants CHRU Nancy
🇫🇷Vandœuvre-lès-Nancy, France