Loupe Loupe Logo Sans titre - 2 Logo color EN I-motion_VA_Q Logo White EN I-motion_VA_BL Logo Logo Logo Logo Logo Logo Logo

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 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
  • 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

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
Logo white en