Ongoing trials - Institut Myologie

ARGX-113-2007 ALKIVIA

A phase 2/3, randomised, double-blind, placebo-controlled study evaluating the efficacy and safety of efgartigimod PH20 SC in patients with active idiopathic inflammatory myopathy.

Code: ARGX-113-2007-ALKIVIA
Disease: Myositis
Principal investigator: O. Benveniste
Sponsor: Argenx
Status: Active
Public: Adults
Phase: Phase 2
Trial site: I-Motion Adults

View on ClinicalTrials.gov

ARGX-113-2011 – ALKIVIA+

A phase 3, multicentre, open-label extension study of the long-term safety, tolerability and efficacy of efgartigimod PH20 SC.

Code: ARGX-113-2011
Disease: Myositis
Principal investigator: O. Benveniste
Sponsor: Argenx
Status: Active
Public: Adults
Phase: Phase 3
Trial site: I-Motion Adults

View on ClinicalTrials.gov

GLPG3667-CL-214 – GALARISSO

A multicentre, randomised, double-blind, placebo-controlled study evaluating the efficacy and safety of oral GLPG3667 over 24 weeks in adults with dermatomyositis.

Code: GLPG3667-CL-214
Disease: Myositis
Principal investigator: O. Benveniste
Sponsor: Galapagos
Status: Active
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

ABC008-IBM-201 – ABCURO

A phase 2/3, randomised, double-blind, placebo-controlled, multicentre study evaluating the efficacy and safety of ABC008 in patients with inclusion body myositis.

Code: ABC008-IBM-201
Disease: Myositis
Principal investigator: O. Benveniste
Sponsor: Abcuro
Status: Active
Public: Adults
Phase: Phase 2
Trial site: I-Motion Adults

View on ClinicalTrials.gov

LUPIN-PASS – Namuscla observational study

An observational study to describe the long-term safety and efficacy of Namuscla in the symptomatic management of myotonia in adult patients with non-dystrophic myotonic disorders.

Code: LUPIN-PASS
Disease: Non-dystrophic myotonias
Principal investigator: S. Vicart
Sponsor: Lupin
Status: Active
Public: Adults
Trial site: I-Motion Adults

ARGX-117-2003 (ARDA Ext)

A long-term extension of trial ARGX-117-2002: assessment of the long-term safety, tolerability, efficacy, pharmacodynamics, pharmacokinetics and immunogenicity of ARGX-117.

Code: ARGX-117-2003
Disease: Multifocal motor neuropathy
Principal investigator: K. Viala
Sponsor: Argenx
Status: Active
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

ARGX-113-1802 – ADHERE

A phase 2 trial to investigate the efficacy, safety and tolerability of efgartigimod PH20 SC in adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP).

Code: ARGX-113-1802
Disease: Chronic inflammatory demyelinating polyneuropathy
Principal investigator: R. Debs
Sponsor: Argenx
Status: Active
Public: Adults
Phase: Phase 2
Trial site: I-Motion Adults

View on ClinicalTrials.gov

ARGX-113-1902 – ADHERE +

An open-label extension of study ARGX-113-1802 to investigate the long-term safety, tolerability and efficacy of efgartigimod PH20 SC in patients with chronic inflammatory demyelinating polyneuropathy (CIDP).

Code: ARGX-113-1902
Disease: Chronic inflammatory demyelinating polyneuropathy
Principal investigator: R. Debs
Sponsor: Argenx
Status: Active
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

EFC17236-Sanofi Mobilize

A phase 3, double-blind, placebo-controlled study evaluating the efficacy and safety of riliprubart in participants with refractory chronic inflammatory demyelinating polyneuropathy.

Code: EFC17236-Sanofi-Mobilize
Disease: Chronic inflammatory demyelinating polyneuropathy
Principal investigator: K. Viala
Sponsor: SANOFI
Status: Active
Public: Adults
Phase: Phase 3
Trial site: I-Motion Adults

View on ClinicalTrials.gov

pHeNIx

Hizentra in inflammatory neuropathies.

Code: pHeNIx
Disease: Chronic inflammatory demyelinating polyneuropathy
Principal investigator: C. Benoist
Sponsor: CSL Behring
Status: Active
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

ARISE-JANSSEN

A phase 2/3, multicentre, multi-stage, randomised, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of nipocalimab in adults with chronic inflammatory demyelinating polyneuropathy (CIDP).

Code: ARISE-JANSSEN
Disease: Chronic inflammatory demyelinating polyneuropathy
Principal investigator: K. Viala
Sponsor: JANSSEN
Status: In preparation
Public: Adults
Phase: Phase 2
Trial site: I-Motion Adults

View on ClinicalTrials.gov

STRONG

Measurement of GDF5 protein expression in muscle biopsies and serum from healthy participants.

Code: STRONG
Disease: Sarcopenia
Principal investigator: France Pietri-Rouxel
Sponsor: AIM
Status: Active
Public: Adults
Trial site: I-Motion Adults

ARGX-119-2302

A phase 1b, double-blind, randomised, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics, immunogenicity and efficacy of ARGX-119 in adult participants with congenital myasthenic syndromes related to DOK7 gene mutations.

Code: ARGX-119-2302
Disease: Congenital myasthenic syndromes
Principal investigator: Villars
Sponsor: Argenx
Status: Active
Public: Adults
Phase: Phase 1
Trial site: I-Motion Adults

View on ClinicalTrials.gov

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