Current trials at I-Motion

The number of ongoing clinical trials in which the I-Motion Pediatric platform 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: Andreea Seferian
  . 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
• Nusi-MFM: Responsiveness and validity study of MFM-32 in SMA patients treated with Nusinersen
  . contact : Andreea Seferian
  . more on Institute of Myology website
• MFM Play: Development and validation study of a Motor Function Measure digitalized playful completion modules: MFM-Play study
  . contact : Marta Gomez
  . more on Institute of Myology website
  
• REALITY: A randomized crossover pragmatic study to evaluate virtual reality for the mitigation of anxiety during intrathecal administration in patients with spinal muscular atrophy
  . contact: Marie-Christine Nougues
  . more on Institute of Myology website
•  SAPPHIRE: Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy
  . contact: Andreea Seferian
  . more on Institute of Myology website
• RESILIENT: A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension
  . contact: Marina Colella
  . more on Institute of Myology website

Duchenne Muscular Dystrophy

• 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 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
• 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: Andreea Seferian
  . 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: Andreea Seferian
  . 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

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

• PTC-AADC-MA-406: An Two Part, International, Real-World, Observational Registry Of Participants Diagnosed With Aromatic L-Amino Acid Decarboxylase Deficiency (AADC) With or Without Treatment With Eladocagene Exuparvovec
  . contact: Claudia Ravelli
  . more on Institute of Myology website