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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, 350 patients are included in ongoing trials, and 23 clinical trials in progress or under development are being conducted at I-Motion on these diseases:

Spinal Muscular Atrophy

  • IONIS-CS11-SHINE: An Open-label extension study for patients with spinal muscular atrophy who previously participated in investigational studies of ISIS 396443
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • Registry SMA (in preparation): European Registry of Patients With Infantile-onset Spinal Muscular Atrophy
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • FIREFISH: A two part seamless multicenter study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in infants with spinal muscular atrophy type 1
    . contact: Odile Boespflug-Tanguy
    . 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 spinal muscular atrophy patients
    . contact: Odile Boespflug-Tanguy
    . more on Institute of Myology website
  • STR1VE (AveXis): Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
    . contact: Odile Boespflug-Tanguy
    . more on Institute of Myology website

Duchenne Muscular Dystrophy

  • NatHis-DMD: Outcome measures in Duchenne Muscular Dystrophy: a Natural History Study
    . contact: Silvana De Lucia
    . 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
  • SRP4658-407 (in preparation): A Natural History Study in Patients with Duchenne Muscular Dystrophy Managed in Routine Clinical Practice
    . contact: Odile Boespflug-Tanguy
    . 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
  • 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 Duchenne Muscular Dystrophy
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • SRP4658-302 (in preparation): Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy
    Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • SRP 4658-102: An open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with DMD Amenable to Exon 51 Skipping
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • SRP 4856-102-OLE: An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • SRP4658-402 (in preparation): 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 Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping
    . contact: Andreea Seferian
    . more on Institute of Myology website
  • SIDEROS: A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid steroids
    . contact: Odile Boespflug-Tanguy
    . more on Institute of Myology website
  • SIDEROS E: A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study
    . contact: Odile Boespflug-Tanguy
    . more on Institute of Myology website
  • SPITFIRE (Anti-Myostatin): A Phase 2/3, Randomized,Parallel, Double-blind, Placebo-controlled, to assess the Efficacy, Safety and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy
    . contact: Odile Boespflug-Tanguy
    . more on Institute of Myology website
  • Italfarmaco: 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-E (in preparation): 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
  • WAVE Dystance: A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients with Duchenne Muscular Dystrophy
    . contact: Odile Boespflug-Tanguy
    . more on Institute of Myology website
  • WAVE-OLE: A Multicenter, Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001
    . contact: Odile Boespflug-Tanguy
    . more on Institute of Myology website

Myotubular Myopathy

  • INCEPTUS: A Prospective, Non-Interventional Clinical Assessment Study in X Linked Myotubular Myopathy (XLMTM) Subjects Aged 3 Years and Younger
    . contact : Andreea Seferian
    . more on Institute of Myology website
  • NatHis-MTM: Prospective Natural History Study of Patients With Myotubular Myopathy and Other CentroNuclear Myopathies
    . contact : Andreea Seferian
    . more on Institute of Myology website
  • 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
  • Dyn-101 (in preparation): 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 : Andreea Seferian
    . more on Institute of Myology website
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