Ongoing trials - Institut Myologie

ARMGO – CL-EPI-001

An observational study in participants with ryanodine receptor 1-related myopathies (RYR1-RM), to determine optimal outcome measures.

Code: ARMGO-CL-EPI-001
Disease: Congenital myopathies
Principal investigator: Ana Ferreiro
Sponsor: ARMGO
Status: In preparation
Public: Adults
Trial site: I-Motion Adults

IMCOMG

Immediate corticosteroid therapy and rituximab to prevent generalisation in ocular myasthenia gravis: a multicentre, open-label, randomised controlled PROBE trial.

Code: IMCOMG
Disease: Autoimmune myasthenia gravis
Principal investigator: Sophie Demeret
Sponsor: Fondation Rothschild
Status: In preparation
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

Jain COS – International dysferlinopathy study

An international study of clinical outcome measures in dysferlinopathies, a form of limb-girdle muscular dystrophy.

Code: Jain-COS
Disease: Limb-girdle muscular dystrophies
Principal investigator: Tanya Stojkovic
Sponsor: AIM
Status: On hold
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

Becker-Coeur

Cardiac involvement in Becker muscular dystrophy: assessment of prognostic markers.

Code: Becker-Coeur
Disease: Becker muscular dystrophy
Principal investigator: K. Wahbi
Sponsor: AIM
Status: Ongoing
Public: Adults

View on ClinicalTrials.gov

ATA-001-FKRP

A multicentre, two-stage, phase 1-2 study to evaluate the safety and efficacy of intravenous GNT0006, an adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle muscular dystrophy (LGMD R9, formerly LGMD2I).

Code: ATA-001-FKRP
Disease: Limb-girdle muscular dystrophies
Principal investigator: Tanya Stojkovic
Sponsor: ATAMYO Therapeutics
Status: Active
Public: Adults
Phase: Phase 1
Trial site: I-Motion Adults

View on ClinicalTrials.gov

EDG-5506 BMD

A phase 2, randomised, double-blind, placebo-controlled study to evaluate the safety, biomarkers and pharmacokinetics of EDG-5506, and its effects on functional measures, in adults and adolescents with Becker muscular dystrophy.

Code: EDG-5506 BMD
Disease: Becker muscular dystrophy
Principal investigator: T. Stojkovic
Sponsor: Edgewise Th
Status: Active
Public: Adults
Phase: Phase 2
Trial site: I-Motion Adults

View on ClinicalTrials.gov

ATA-003-GSAR (Atamyo)

A phase 1-2, open-label, dose-escalation study to evaluate the safety of three doses of intravenous ATA-200, an adeno-associated viral vector carrying the human gamma-sarcoglycan gene, in patients with gamma-sarcoglycanopathy (LGMD R5, formerly LGMD 2C).

Code: ATA-003-GSAR
Disease: Limb-girdle muscular dystrophies
Principal investigator: Marina Colella
Sponsor: ATAMYO Therapeutics
Status: In preparation
Public: Paediatric
Phase: Phase 1
Trial site: I-Motion Pediatric

View on ClinicalTrials.gov

GNT-016-MDYF

A clinical trial of gene therapy with a microdystrophin (GNT0004) in DMD: a gene therapy study using a microdystrophin for the treatment of Duchenne muscular dystrophy.

Code: GNT-016-MDYF
Disease: Duchenne muscular dystrophy
Principal investigator: Silvana De Lucia
Sponsor: Genethon
Status: Pending
Public: Paediatric
Trial site: I-Motion Pediatric

COL6 registry

Phenotypic and molecular characterisation of a cohort of patients with a COL6 gene-related myopathy (retrospective study).

Code: Registre-COL6
Disease: Collagen VI-related myopathies
Principal investigator: Tanya Stojkovic
Sponsor: AP-HP
Status: Ongoing
Public: Adults

MIS51ON

A randomised, double-blind, dose-finding and dose-comparison study of eteplirsen in DMD, evaluating the safety and efficacy of high-dose eteplirsen in patients with DMD with deletion mutations amenable to exon 51 skipping.

Code: MIS51ON
Disease: Duchenne muscular dystrophy
Principal investigator: Andreea Seferian
Sponsor: SAREPTA
Status: Active
Public: Paediatric
Trial site: I-Motion Pediatric

View on ClinicalTrials.gov

Col6-RD

A study of modifiers of collagen VI-related dystrophies (COL6-RD). Identification of the genetic modifiers that determine the severity of COL6 myopathy (ICF).

Code: Col6-RD
Disease: Collagen VI-related myopathies
Principal investigator: Tanya Stojkovic
Sponsor: AIM
Status: In preparation
Public: Adults

Italfarmaco 51

An open-label study of the long-term safety, tolerability and efficacy of givinostat in DMD: a study evaluating the long-term safety, tolerability and efficacy of givinostat in all previously treated patients with Duchenne muscular dystrophy.

Code: Italfarmaco-51
Disease: Duchenne muscular dystrophy
Principal investigator: Odile Boespflug-Tanguy
Sponsor: Italfarmaco
Status: Active
Public: Paediatric
Trial site: I-Motion Pediatric

View on ClinicalTrials.gov

ADSVF-in-IBM – Cell therapy in inclusion body myositis

Intramuscular injection of autologous cells from the stromal vascular fraction of adipose tissue: a phase 1 trial.

Code: ADSVF-in-IBM
Disease: Myositis
Principal investigator: O. Benveniste
Sponsor: APHP (PHRC)
Status: Active
Public: Adults
Phase: Phase 1
Trial site: I-Motion Adults

View on ClinicalTrials.gov

GNT-014-MDYF – Natural history of DMD

A prospective, interventional, reference study examining the natural history of DMD in young male patients aged 4 to 6 years.

Code: GNT-014-MDYF
Disease: Duchenne muscular dystrophy
Principal investigator: Silvana De Lucia
Sponsor: Genethon
Status: Active
Public: Paediatric
Trial site: I-Motion Pediatric

View on ClinicalTrials.gov

IgPro20_3007 – RECLAIIM study

Assessment of the efficacy, safety and pharmacokinetics of IgPro20 in adults with dermatomyositis.

Code: IgPro20_3007
Disease: Myositis
Principal investigator: O. Benveniste
Sponsor: CSL Behring
Status: Active
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

UMD-DMD – Dystrophinopathy database

Dystrophinopathy database bringing together the French molecular diagnostic laboratories and all the neuromuscular reference and competence centres.

Code: UMD-DMD
Disease: Duchenne muscular dystrophy
Principal investigator: Rabah Ben Yaou / France Leturcq / Sylvie Tuffery-Giraud
Sponsor: Laboratoires francais de diagnostic moleculaire des dystrophinopathies + tous les centres de reference et competence MNM
Status: Ongoing
Public: Adults, Paediatric

View on ClinicalTrials.gov

DT4RD

Feasibility, validation and application of digital tools for the follow-up of patients with neuromuscular diseases in daily life.

Code: DT4RD
Disease: Cross-disease studies
Principal investigator: Marion Masingue
Sponsor: AIM
Status: Active
Public: Adults
Trial site: I-Motion Adults

Resolve France Expansion – CTRN FSHD France

An 18-month prospective MRI study in facioscapulohumeral muscular dystrophy.

Code: Resolve-France-Expansion
Disease: Facioscapulohumeral muscular dystrophy
Principal investigator: Teresinha Evangelista
Sponsor: CHU de Nice
Status: Active
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

ARGX113-2308 ADAPT (Seroneg)

A phase 3, randomised, double-blind, placebo-controlled study to evaluate the efficacy and safety of intravenous efgartigimod in adult patients with seronegative generalised myasthenia gravis.

Code: ARGX113-2308
Disease: Autoimmune myasthenia gravis
Principal investigator: Sophie Demeret
Sponsor: Argenx
Status: Active
Public: Adults
Phase: Phase 3
Trial site: I-Motion Adults

View on ClinicalTrials.gov

PROGRESS FSHD

Remote assessment and artificial intelligence to validate new measures, biomarkers and new therapeutic targets in facioscapulohumeral muscular dystrophy.

Code: PROGRESS-FSHD
Disease: Facioscapulohumeral muscular dystrophy
Principal investigator: Teresinha Evangelista
Sponsor: CHU de Nice
Status: Active
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov