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CLINICAL ACTIVITIES
Ongoing trials
The Institute of Myology leads cutting-edge clinical trials for neuromuscular diseases, in partnership with industry laboratories and academic researchers.
Below you will find the list of ongoing trials at the Institute of Myology
6 clinical trials in progress
DYNE101-DM1-201
A randomised, placebo-controlled, multiple-ascending-dose study evaluating the safety, tolerability, pharmacodynamics, efficacy and pharmacokinetics of DYNE-101 in participants with myotonic dystrophy type 1.
DYNE101-DM1-201
A randomised, placebo-controlled, multiple-ascending-dose study evaluating the safety, tolerability, pharmacodynamics, efficacy and pharmacokinetics of DYNE-101 in participants with myotonic dystrophy type 1.
- Code
- DYNE101-DM1-201
- Disease
- Myotonic dystrophies
- Principal investigator
- Guillaume Bassez
- Sponsor
- DYNE Therapeutics
- Status
- Active
- Public
- Adults
- Trial site
- I-Motion Adults
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.
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.
- Code
- PIP4 (MEX-NM-301)
- Disease
- Myotonic dystrophies
- Principal investigator
- Arnaud Isapof
- Sponsor
- Lupin
- Status
- Ongoing
- Public
- Paediatric
- Trial site
- I-Motion Pediatric
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.
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
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.
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
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.
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
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.
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