Ongoing trials - Institut Myologie

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

Echostim-EFR

Ultrafast ultrasound for the functional assessment of the diaphragm: agreement with standard mechanical and electrophysiological variables, and diagnostic performance.

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

View on ClinicalTrials.gov

AVXS-101-LT-002

Long-term follow-up study of patients who received AVXS-101 in the clinical trials for spinal muscular atrophy.

Code: AVXS-101-LT-002
Disease: Spinal muscular atrophies
Principal investigator: Andreea Seferian
Sponsor: AveXis
Status: Active
Public: Paediatric
Trial site: I-Motion Pediatric

View on ClinicalTrials.gov

AZA-001-301

An 18-month, multicentre, phase 3, double-blind, randomised, placebo-controlled study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick disease type C and in late-infantile and juvenile forms of GM1 or GM2 gangliosidosis.

Code: AZA-001-301
Disease: Gangliosidosis
Principal investigator: Benedicte Heron
Sponsor: Azafaros
Status: In preparation
Public: Paediatric
Phase: Phase 3
Trial site: I-Motion Pediatric

ONYX

An open-label, multicentre extension study to evaluate the long-term safety and efficacy of apitegromab in patients with type 2 and type 3 spinal muscular atrophy who took part in previous apitegromab studies.

Code: ONYX
Disease: Spinal muscular atrophies
Principal investigator: Andreea Seferian
Sponsor: Scholar Rock
Status: Active
Public: Paediatric
Trial site: I-Motion Pediatric

View on ClinicalTrials.gov

GSD3 – Prospective patient follow-up

An observational study with prospective follow-up of patients with glycogen storage disease type III.

Code: GSD3
Disease: Glycogen storage disease type III
Principal investigator: P. Laforet et P. Labrune
Sponsor: AIM
Status: Active
Public: Adults

PHENO SMART

Characterisation of new phenotypes in patients with type 1, 2 and 3 infantile or juvenile spinal muscular atrophy treated with therapies that restore SMN protein expression.

Code: PHENO SMART
Disease: Spinal muscular atrophies
Principal investigator: Silvana De Lucia
Sponsor: UCL
Status: In preparation
Public: Paediatric
Trial site: I-Motion Pediatric

View on ClinicalTrials.gov

PTA17333 Avaglucosidase

A French multicentre, phase 4, open-label extension study of long-term safety and efficacy in patients with Pompe disease who previously took part in development studies.

Code: PTA17333 Avaglucosidase
Disease: Glycogen storage disease type II (Pompe disease)
Principal investigator: Anthony Behin
Sponsor: Sanofi
Status: Active
Public: Adults
Phase: Phase 4
Trial site: I-Motion Adults

View on ClinicalTrials.gov