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Immune-Mediated Necrotizing MyopathyMYONECPEDNCT04295785

Juvenile Autoimmune Necrotizing Myopathies

An observational study of Immune-Mediated Necrotizing Myopathy, run by Central Hospital, Nancy, France at 1 site in France.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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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. Read more about this study

Who is it for?
Any age
Conditions the study is for
Immune-Mediated Necrotizing Myopathy
How many people
10 participants
Where
1 siteFrance
Study dates
Mar 2020to Nov 2020
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

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

Who may be able to join

  • 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

Who cannot join

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

Trial locations and contacts

1 site in 1 country.

Irène LEMELLE contact

03 83 15 45 96

i.lemelle@chru-nancy.fr

France1 site

  • Hopital d'enfants CHRU Nancy

    Vandœuvre-lès-Nancy, 54 500

Other trials in France

About this study

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

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.

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

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT04295785
Study groupRoleWhat they receive
autoimmune necrotizing myopathy beginning before 18Not described

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Type of muscle involvment

    Proximal or distal or axial muscle involvement by muscle testing

    Measured: At diagnosis

  2. Type of cutaneous involvment

    Rash or Gottron lesions

    Measured: At diagnosis

  3. Other clinical manifestations

    Dysphagia or dyspnea or fever or interstitial lung involvement or articular involvement or gastrointestinal involvment

    Measured: At diagnosis

  4. Blood CK level

    Blood CK level

    Measured: At diagnosis

  5. Histology

    Results of muscle biopsy : necrosis

    Measured: At diagnosis

  6. Histology

    Results of muscle biopsy : inflammation

    Measured: At diagnosis

  7. MMT score

    MMT score

    Measured: At diagnosis

  8. CMAS score

    CMAS score

    Measured: At diagnosis

Secondary outcomes (11)

  1. Type of drugs used

    Drugs ever used : name and duration

    Measured: At last visit

  2. Iatrogenic complications

    Infectious complications

    Measured: At last visit

  3. Iatrogenic complications

    Bone complications

    Measured: At last visit

  4. Status of the disease at last visit

    Status of the disease at last visit : active or remission or death

    Measured: At last visit

  5. Number of relapses

    Number of relapses

    Measured: At last visit

  6. Type of muscle involvment

    Proximal or distal or axial muscle involvement by muscle testing

    Measured: At last visit

  7. Type of cutaneous involvment

    Rash or Gottron lesions

    Measured: At last visit

  8. Other clinical manifestations

    Dysphagia or dyspnea or fever or interstitial lung involvement or articular involvement or gastrointestinal involvment

    Measured: At last visit

  9. Blood CK level

    Blood CK level

    Measured: At last visit

  10. CMAS score

    CMAS score

    Measured: At last visit

  11. MMT score

    MMT score

    Measured: At last visit

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Mar 2020
  2. Study startMar 2020 (estimated)
  3. Primary completionNov 2020 (estimated)
  4. Study completionNov 2020 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Case only
Time perspective
Retrospective
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04295785
Sponsor's protocol ID
2019PI192
Acronym
MYONECPED
Status last verified
Jan 2020
First submitted
9 Jan 2020
Last updated
5 Mar 2020
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • 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.

    PubMed 27538058

  • Mariampillai K, Granger B, Amelin D, Guiguet M, Hachulla E, Maurier F, Meyer A, Tohme 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.

    PubMed 30208379

  • 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.

    PubMed 27147697

  • 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):150-157. doi: 10.1097/MD.0000000000000028.

    PubMed 24797170

  • 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. No abstract available.

    PubMed 28864670

  • 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.

    PubMed 27818386

  • 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.

    PubMed 28096458

  • 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.

    PubMed 29089059

Questions about this trial

What is this clinical trial studying?

An observational study of Immune-Mediated Necrotizing Myopathy, run by Central Hospital, Nancy, France at 1 site in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 5 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Irène LEMELLE (03 83 15 45 96, i.lemelle@chru-nancy.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Type of muscle involvment (time frame: At diagnosis). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04295785, which also lists 8 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Mar 2020.

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