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The number of ongoing clinical trials in which the I-Motion Institute is involved is only increasing.

I-Motion performs various roles in the clinical trials in which it is involved. When I-Motion is a “promoter”, it coordinates the trials from A to Z. Thus, I-Motion develops its own clinical studies through the trials promotion by the Institute of Myology. When it is an “investigator”, it organizes the inclusion and follow-up of patients in clinical trials conducted by industrialists, private laboratories (Genethon, for example) or even public organizations.

Today, clinical trials in progress or under development being conducted at I-Motion concern:

Spinal Muscular Atrophy

  • IONIS-CS11-SHINE: An Open-label extension study for patients with SMA who previously participated in investigational studies of ISIS 396443
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • SUNFISH: A two-part seamless, multi-center randomized, placebo-controlled, doubleblind study to investigate the safety, tolerabilty, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 SMA patients
    . contact: Odile Boespflug-Tanguy
    . more on Institute of Myology website
  • AVXS-101-LT-002: A Long-term Follow-up Study of Patients in the Clinical Trials for SMA Receiving AVXS-101
    . contact: Andreea Seferian
    . more on Institute of Myology website

Duchenne Muscular Dystrophy

  • NatHis-DMD: Outcome measures in DMD: a Natural History Study
    . contact: Silvana De Lucia
    . more on Institute of Myology website
  • PTC-PASS: Long-Term Observational Study of Translarna safety and effectiveness in routine care
    . contact: Silvana De Lucia
    . more on Institute of Myology website
  • SRP-4045-302: Long-term, Open-label Extension Study for Patients With DMD Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • Essence: A Double-blind, Placebo-Controlled, Multicenter Study With and Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With DMD
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • GNT-014-MDYF: Natural History of Duchenne Muscular Dystrophy – A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 6 Years
    . contact: Silvana De Lucia
    . more on Institute of Myology website
  • GNT-016-MDYF: Microdystrophin (GNT0004) Gene Therapy Clinical Trial in DMD
    . contact: Silvana De Lucia
    . more on Institute of Myology website
  • Italfarmaco 48: Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With DMD
    . contact: Odile Boespflug-Tanguy
    . more on Institute of Myology website
  • Italfarmaco 51: Open label, long-term safety, tolerability, and efficacy study of Givinostat in all DMD patients who have been previously treated in one of the Givinostat studies
    . contact: Odile Boespflug-Tanguy
    . more on Institute of Myology website
  • MIS51ON: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with DMD With Deletion Mutations Amenable to Exon 51 Skipping
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • SRP4658-407: A Natural History Study in Patients with DMD Managed in Routine Clinical Practice
    . contact: Odile Boespflug-Tanguy
    . more on Institute of Myology website
  • SRP 4856-102-OLE: An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with DMD Who Have Completed Study 4658-102
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • FGCL-3019-093: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory DMD
    . contact: Silvana De Lucia
    . more on Institute of Myology website
  • FGCL-3019-094: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in ambulatory subjects with DMD
    . contact: Silvana De Lucia
    . more on Institute of Myology website
  • C3391002: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study To Evaluate The Safety And Efficacy Of PF-06939926 For The Treatment Of Non-Ambulatory Male Participants With DMD
    . contact: Arnaud Isapof
    . more on Institute of Myology website

Myotonic Dystrophies and Non Dystrophic Myotonies

  • 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
    . contact: Arnaud Isapof
    . more on Institute of Myology website
  • PIP7 (MEX-NM-303): Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders Who Have Completed MEX-NM-301 Study
    . contact: Arnaud Isapof
    . more on Institute of Myology website

Myotubular Myopathy and other Centronuclear Myopathies

  • ASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • NatHis-CNM: Prospective, Longitudinal Study of the Natural History and functional status of patients with MyoTubular Myopathy and other centronuclear myopathies
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • UNITE-CNM: A Phase 1/2 trial on the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of DYN101 in patients ≥ 16 years of age with centronuclear myopathies caused by mutations in DNM2 or MTM1
    . contact: Anthony Behin
    . more on Institute of Myology website
  • DynamicA Phase 1/2, multicenter, open-label, dose-confirmation trial to evaluate safety and preliminary efficacy of DYN101 in subjects 2 to 17 years of age with centronuclear myopathy caused by mutations in MTM1 or DNM2
    . contact: Emmanuelle Lagrue
    . more on Institute of Myology website

GM1 Gangliosidosis

  • P1-GM-101: An open-label adaptive design study of intracisternal administration of adeno-associated viral vector serotype rh.10 carrying the human β-galactosidase cDNA for the treatment of GM1 gangliosidosis
    . contact: Bénédicte Héron
    . more on Institute of Myology website

AADC Deficiency

  • AADCAware: An International, Real-World, Observational Registry Of The Natural History Of Subjects Diagnosed With Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
    . contact: Claudia Ravelli
    . more on Institute of Myology website
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