Ongoing trials - Institut Myologie

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

PTC-AADC-MA-406

A two-part, international, real-world observational registry of participants diagnosed with aromatic L-amino acid decarboxylase (AADC) deficiency, with or without treatment with eladocagene exuparvovec.

Code: PTC-AADC-MA-406
Disease: AADC deficiency
Principal investigator: Claudia Ravelli
Sponsor: PTC Therapeutics
Status: Active
Public: Paediatric

UMD FHL1 – FHL1 database

Non-interventional data collection on myopathies related to FHL1 gene abnormalities, gathering medical information and following the progression of patients.

Code: UMD-FHL1
Disease: Laminopathies and emerinopathies
Principal investigator: Gisele Bonne, Rabah Ben Yaou
Sponsor: Centre de recherche en myologie Institut de Myologie + UF de Cardiogenetique et Myogenetique Moleculaire et Cellulaire
Status: Ongoing
Public: Adults

UMD-DNM2 – Dynaminopathy databases

Non-interventional data collection on dynaminopathies, neuromuscular diseases caused by mutations in the DNM2 gene, which encodes dynamin 2, a protein involved in endocytosis and intracellular membrane trafficking as well as in the regulation of the actin and microtubule cytoskeletons.

Code: UMD-DNM2
Disease: Dynaminopathies
Principal investigator: Marc Bitoun / Valerie Biancalana
Sponsor: Inserm U974, Institut de Myologie / Laboratoire de diagnostic genetique, Hopital Universitaire de Strasbourg
Status: Ongoing
Public: Adults

OPALE National Registry – Laminopathy and emerinopathy observatory

A national registry collecting data on patients with laminopathies and emerinopathies, in order to follow their progression and compare the different forms of the disease.

Code: OPALE-National
Disease: Laminopathies and emerinopathies
Principal investigator: Karim Wahbi (PI), Gisele Bonne, Rabah Ben Yaou (coordonnateurs operationnels)
Sponsor: Institut de Myologie
Status: Ongoing
Public: Adults, Paediatric

DYNE101-DM1-201

A randomised, placebo-controlled, multiple-ascending-dose study evaluating the safety, tolerability, pharmacodynamics, efficacy and pharmacokinetics of DYNE-101 in participants with myotonic dystrophy type 1.

Code: DYNE101-DM1-201
Disease: Myotonic dystrophies
Principal investigator: Guillaume Bassez
Sponsor: DYNE Therapeutics
Status: Active
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

NMD670

A phase 2a, randomised, double-blind, placebo-controlled study to evaluate the efficacy, safety and tolerability of NMD670 over 21 days in ambulatory adult patients with Charcot-Marie-Tooth disease type 1 and type 2.

Code: NMD670
Disease: Charcot-Marie-Tooth disease
Principal investigator: Marion Masingue
Sponsor: NMD Pharma
Status: Active
Public: Adults
Phase: Phase 2
Trial site: I-Motion Adults

View on ClinicalTrials.gov

PIP4 (MEX-NM-301)

An open-label, non-comparative study to evaluate the steady-state pharmacokinetics, safety and efficacy of mexiletine in adolescents and children with myotonic disorders.

Code: PIP4 (MEX-NM-301)
Disease: Myotonic dystrophies
Principal investigator: Arnaud Isapof
Sponsor: Lupin
Status: Ongoing
Public: Paediatric
Trial site: I-Motion Pediatric

View on ClinicalTrials.gov

Mitochondrial disease registry

Prospective follow-up of a cohort of adult patients with mitochondrial diseases (observational study).

Code: Registre-Mitochondriopathies
Disease: Mitochondrial diseases
Principal investigator: Pascal Laforet
Sponsor: Institut de Myologie
Status: Active
Public: Adults

PIP7 (MEX-NM-303)

An open-label extension study to evaluate the long-term safety and efficacy of mexiletine in paediatric patients with myotonic disorders who completed study MEX-NM-301.

Code: PIP7 (MEX-NM-303)
Disease: Myotonic dystrophies
Principal investigator: Arnaud Isapof
Sponsor: Lupin
Status: Ongoing
Public: Paediatric
Trial site: I-Motion Pediatric

REN001-201 / STRIDE

A double-blind, placebo-controlled study to evaluate the efficacy and safety of 24 weeks of treatment with REN001 in patients with primary mitochondrial myopathy.

Code: REN001-201 / STRIDE
Disease: Mitochondrial diseases
Principal investigator: Tanya Stojkovic
Sponsor: Reneo
Status: Active
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

ArthemiR CT-ATX-01-DM1-1.1

A phase 1/2a, double-blind, placebo-controlled, single-ascending-dose and multiple-ascending-dose study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary clinical efficacy of intravenous ATX-01 in participants aged 18 to 64 years with myotonic dystrophy type 1.

Code: ArthemiR CT-ATX-01-DM1-1.1
Disease: Myotonic dystrophies
Principal investigator: Guillaume Bassez
Sponsor: ARTHEx Biotech S. L.
Status: Active
Public: Adults
Phase: Phase 1
Trial site: I-Motion Adults

View on ClinicalTrials.gov

REN001-202

An open-label, multicentre study to evaluate the long-term safety and tolerability of REN001 in participants with primary mitochondrial myopathy.

Code: REN001-202
Disease: Mitochondrial diseases
Principal investigator: Tanya Stojkovic
Sponsor: Reneo
Status: Active
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

Vertex VX23-670-001

A phase 1/2, randomised, double-blind, placebo-controlled, single-dose and multiple-dose escalation study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of VX-670 in adult patients with myotonic dystrophy type 1.

Code: Vertex VX23-670-001
Disease: Myotonic dystrophies
Principal investigator: Guillaume Bassez
Sponsor: Vertex
Status: Active
Public: Adults
Phase: Phase 1
Trial site: I-Motion Adults

View on ClinicalTrials.gov

Exo-KGO1

Assessment of the safety and immediate effects of a robotic lower-limb dermoskeleton in patients with neuromuscular diseases.

Code: Exo-KGO1
Disease: Cross-disease studies
Principal investigator: Damien Bachasson
Sponsor: AIM
Status: Active
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov

Avidity AOC 1001-CS3

A phase 3, randomised, double-blind, placebo-controlled study to evaluate the efficacy and safety of intravenous AOC 1001 in the treatment of myotonic dystrophy type 1.

Code: Avidity AOC 1001-CS3
Disease: Myotonic dystrophies
Principal investigator: Guillaume Bassez
Sponsor: Avidity Biosciences
Status: Active
Public: Adults
Phase: Phase 3
Trial site: I-Motion Adults

View on ClinicalTrials.gov

Exo-NMD1

Assessment of the safety and acute effects of a soft knee- and hip-actuated exoskeleton in patients with neuromuscular disorders.

Code: Exo-NMD1
Disease: Cross-disease studies
Principal investigator: Damien Bachasson
Sponsor: AIM
Status: Active
Public: Adults
Trial site: I-Motion Adults

View on ClinicalTrials.gov