Juvenile Autoimmune Necrotizing Myopathies

Immune-Mediated Necrotizing Myopathy Clinical Trial

— MYONECPED  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04295785
• Other study ID #: 2019PI192
• Status: Not yet recruiting
• Start date: March 2020
• Est. completion date: November 2020

Study information

• Verified date: January 2020
• Source: Central Hospital, Nancy, France
• Contact: Irène LEMELLE
• Phone: 03 83 15 45 96
• Email: i.lemelle[@]chru-nancy.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 10
• Est. completion date: November 2020
• Est. primary completion date: November 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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)

Related Conditions & MeSH terms

• Immune-Mediated Necrotizing Myopathy
• Muscular Diseases

Locations

Country	Name	City	State
France	Hopital d'enfants CHRU Nancy	Vandœuvre-lès-Nancy

Sponsors (1)

Lead Sponsor	Collaborator
Central Hospital, Nancy, France

Country where clinical trial is conducted

France, 

References & Publications (8)

Allenbach Y, Drouot L, Rigolet A, Charuel JL, Jouen F, Romero NB, Maisonobe T, Dubourg O, Behin A, Laforet P, Stojkovic T, Eymard B, Costedoat-Chalumeau N, Campana-Salort E, Tournadre A, Musset L, Bader-Meunier B, Kone-Paut I, Sibilia J, Servais L, Fain O, Larroche C, Diot E, Terrier B, De Paz R, Dossier A, Menard D, Morati C, Roux M, Ferrer X, Martinet J, Besnard S, Bellance R, Cacoub P, Arnaud L, Grosbois B, Herson S, Boyer O, Benveniste O; French Myositis Network. Anti-HMGCR autoantibodies in European patients with autoimmune necrotizing myopathies: inconstant exposure to statin. Medicine (Baltimore). 2014 May;93(3):150-7. doi: 10.1097/MD.0000000000000028. — View Citation

Anti-HMGCR Autoantibodies in Juvenile Idiopathic Inflammatory Myopathies Identify a Rare but Clinically Important Subset of Patients. J Rheumatol. 2017 Sep;44(9):1417. doi: 10.3899/jrheum.160871.C1. — View Citation

Benveniste O, Stenzel W, Allenbach Y. Advances in serological diagnostics of inflammatory myopathies. Curr Opin Neurol. 2016 Oct;29(5):662-73. doi: 10.1097/WCO.0000000000000376. Review. — View Citation

Binns EL, Moraitis E, Maillard S, Tansley S, McHugh N, Jacques TS, Wedderburn LR, Pilkington C, Yasin SA, Nistala K; UK Juvenile Dermatomyositis Research Group (UK and Ireland). Effective induction therapy for anti-SRP associated myositis in childhood: A small case series and review of the literature. Pediatr Rheumatol Online J. 2017 Oct 31;15(1):77. doi: 10.1186/s12969-017-0205-x. Review. — View Citation

Liang WC, Uruha A, Suzuki S, Murakami N, Takeshita E, Chen WZ, Jong YJ, Endo Y, Komaki H, Fujii T, Kawano Y, Mori-Yoshimura M, Oya Y, Xi J, Zhu W, Zhao C, Watanabe Y, Ikemoto K, Nishikawa A, Hamanaka K, Mitsuhashi S, Suzuki N, Nishino I. Pediatric necrotizing myopathy associated with anti-3-hydroxy-3-methylglutaryl-coenzyme A reductase antibodies. Rheumatology (Oxford). 2017 Feb;56(2):287-293. doi: 10.1093/rheumatology/kew386. Epub 2016 Nov 6. — View Citation

Mariampillai K, Granger B, Amelin D, Guiguet M, Hachulla E, Maurier F, Meyer A, Tohmé A, Charuel JL, Musset L, Allenbach Y, Benveniste O. Development of a New Classification System for Idiopathic Inflammatory Myopathies Based on Clinical Manifestations and Myositis-Specific Autoantibodies. JAMA Neurol. 2018 Dec 1;75(12):1528-1537. doi: 10.1001/jamaneurol.2018.2598. — View Citation

Tiniakou E, Pinal-Fernandez I, Lloyd TE, Albayda J, Paik J, Werner JL, Parks CA, Casciola-Rosen L, Christopher-Stine L, Mammen AL. More severe disease and slower recovery in younger patients with anti-3-hydroxy-3-methylglutaryl-coenzyme A reductase-associated autoimmune myopathy. Rheumatology (Oxford). 2017 May 1;56(5):787-794. doi: 10.1093/rheumatology/kew470. — View Citation

Watanabe Y, Uruha A, Suzuki S, Nakahara J, Hamanaka K, Takayama K, Suzuki N, Nishino I. Clinical features and prognosis in anti-SRP and anti-HMGCR necrotising myopathy. J Neurol Neurosurg Psychiatry. 2016 Oct;87(10):1038-44. doi: 10.1136/jnnp-2016-313166. Epub 2016 May 4. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Type of muscle involvment	Proximal or distal or axial muscle involvement by muscle testing	At diagnosis
Primary	Type of cutaneous involvment	Rash or Gottron lesions	At diagnosis
Primary	Other clinical manifestations	Dysphagia or dyspnea or fever or interstitial lung involvement or articular involvement or gastrointestinal involvment	At diagnosis
Primary	Blood CK level	Blood CK level	At diagnosis
Primary	Histology	Results of muscle biopsy : necrosis	At diagnosis
Primary	Histology	Results of muscle biopsy : inflammation	At diagnosis
Primary	MMT score	MMT score	At diagnosis
Primary	CMAS score	CMAS score	At diagnosis
Secondary	Type of drugs used	Drugs ever used : name and duration	At last visit
Secondary	Iatrogenic complications	Infectious complications	At last visit
Secondary	Iatrogenic complications	Bone complications	At last visit
Secondary	Status of the disease at last visit	Status of the disease at last visit : active or remission or death	At last visit
Secondary	Number of relapses	Number of relapses	At last visit
Secondary	Type of muscle involvment	Proximal or distal or axial muscle involvement by muscle testing	At last visit
Secondary	Type of cutaneous involvment	Rash or Gottron lesions	At last visit
Secondary	Other clinical manifestations	Dysphagia or dyspnea or fever or interstitial lung involvement or articular involvement or gastrointestinal involvment	At last visit
Secondary	Blood CK level	Blood CK level	At last visit
Secondary	CMAS score	CMAS score	At last visit
Secondary	MMT score	MMT score	At last visit