Ongoing trials - Institut Myologie

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

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

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

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