Juvenile Autoimmune Necrotizing Myopathies

Clinical, Paraclinical, Functional Characteristics and Evolution of Juvenile Autoimmune Necrotizing Myopathies in a National Retrospective Cohort

Status

UNKNOWN

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04295785

Acronym

MYONECPED

Enrollment

Registered

2020-03-05

Start date

2020-03-31

Completion date

2020-11-30

Last updated

2020-03-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Immune-Mediated Necrotizing Myopathy

Keywords

Autoimmune necrotizing myopathy, anti-HMGCR antibodies, anti-SRP antibodies

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.

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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)

Design outcomes

Primary

Measure	Time frame	Description
Type of muscle involvment	At diagnosis	Proximal or distal or axial muscle involvement by muscle testing
Type of cutaneous involvment	At diagnosis	Rash or Gottron lesions
Other clinical manifestations	At diagnosis	Dysphagia or dyspnea or fever or interstitial lung involvement or articular involvement or gastrointestinal involvment
Blood CK level	At diagnosis	Blood CK level
Histology	At diagnosis	Results of muscle biopsy : necrosis
MMT score	At diagnosis	MMT score
CMAS score	At diagnosis	CMAS score

Secondary

Measure	Time frame	Description
Blood CK level	At last visit	Blood CK level
Type of drugs used	At last visit	Drugs ever used : name and duration
MMT score	At last visit	MMT score
CMAS score	At last visit	CMAS score
Iatrogenic complications	At last visit	Infectious complications
Status of the disease at last visit	At last visit	Status of the disease at last visit : active or remission or death
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
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

Countries

France

Contacts

Primary ContactIrène LEMELLE

i.lemelle@chru-nancy.fr03 83 15 45 96

Outcome results