Ongoing trials - Institut Myologie

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

ARGX-113-2003 – ADAPT NXT

A phase 3b, randomised, open-label, parallel-group study to evaluate different dosing regimens of intravenous efgartigimod in patients with generalised myasthenia gravis.

Code: ARGX-113-2003 – ADAPT NXT
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

ReSOLVE FSHD International

Clinical trial readiness to solve barriers to drug development in FSHD.

Code: ReSOLVE-FSHD-International
Disease: Facioscapulohumeral muscular dystrophy
Principal investigator: Guillaume Bassez
Sponsor: CHU de Nice
Status: Active
Public: Adults
Trial site: I-Motion Adults

R3918-MG-2018

Efficacy and safety of pozelimab and cemdisiran combination therapy in patients with autoimmune myasthenia gravis.

Code: R3918-MG-2018
Disease: Autoimmune myasthenia gravis
Principal investigator: Sophie Demeret
Sponsor: Regeneron
Status: Ongoing
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

FSHD2-INSIGHT

An 18-month prospective natural history study of FSHD2, to understand the pathophysiology and progression of the disease.

Code: FSHD2-INSIGHT
Disease: Facioscapulohumeral muscular dystrophy
Principal investigator: Marion Masingue
Sponsor: CHU Nice
Status: Active
Public: Adults
Trial site: I-Motion Adults

MOM-M281-011

A phase 3, randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety, pharmacodynamics and pharmacokinetics of nipocalimab.

Code: MOM-M281-011
Disease: Autoimmune myasthenia gravis
Principal investigator: Sophie Demeret
Sponsor: Jannsen-Cilag
Status: Ongoing
Public: Adults
Phase: Phase 3
Trial site: I-Motion Adults

View on ClinicalTrials.gov

ASPIRO

A phase 1/2, randomised, controlled, open-label, ascending-dose clinical study to evaluate the safety and preliminary efficacy of AT132, a gene therapy delivered by an adeno-associated virus serotype 8 (AAV8) vector, in patients with X-linked myotubular myopathy (XLMTM).

Code: ASPIRO
Disease: Myotubular myopathy and other centronuclear myopathies
Principal investigator: Andreea Seferian
Sponsor: Audentes Therapeutics
Status: Active
Public: Paediatric
Phase: Phase 1
Trial site: I-Motion Pediatric

View on ClinicalTrials.gov

MYAPATH

Creation of a biological resource collection for the study of conditions that can lead to autoimmune myasthenia gravis.

Code: MYAPATH
Disease: Autoimmune myasthenia gravis
Principal investigator: Anthony Behin
Sponsor: AIM
Status: Ongoing
Public: Adults

NatHis-CNM

A prospective, longitudinal study assessing the natural history and motor function of patients with myotubular myopathy and other centronuclear myopathies.

Code: NatHis-CNM
Disease: Myotubular myopathy and other centronuclear myopathies
Principal investigator: Andreea Seferian
Sponsor: AIM
Status: Completed
Public: Adults, Paediatric

View on ClinicalTrials.gov

MS700568_0183

A phase 3, randomised, double-blind, placebo-controlled, three-arm, three-period study to evaluate the efficacy and safety of a new oral cladribine formulation.

Code: MS700568_0183
Disease: Autoimmune myasthenia gravis
Principal investigator: Anthony Behin
Sponsor: Merck
Status: In preparation
Public: Adults
Phase: Phase 3
Trial site: I-Motion Adults

View on ClinicalTrials.gov

NH-CNM-001

A prospective, longitudinal study of the natural history and functional status of patients with centronuclear myopathies.

Code: NH-CNM-001
Disease: Myotubular myopathy and other centronuclear myopathies
Principal investigator: Anthony Behin
Status: In preparation
Public: Adults
Trial site: I-Motion Adults

LOU064O12301

A phase 3, randomised, double-blind, placebo-controlled study to evaluate the efficacy and safety of a Bruton tyrosine kinase inhibitor in patients with generalised myasthenia gravis.

Code: LOU064O12301
Disease: Autoimmune myasthenia gravis
Principal investigator: Anthony Behin
Sponsor: Novartis
Status: In preparation
Public: Adults
Phase: Phase 3
Trial site: I-Motion Adults

View on ClinicalTrials.gov

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