XClose

Queen Square Centre for Neuromuscular Diseases

Home
Menu

Clinical Trials

We run an active translational research programme.

If you are a doctor interested in making a referral, or someone with mitochondrial disease with questions concerning research or clinical trials, please email our research nurses, Louise or Iwona, at l.germain@nhs.net or iwona.skorupinska@nhs.net.

Stride study
  • Recruitment: active
  • Description: This is a trial testing the effectiveness and safety of a new medication (REN001) for mitochondrial muscle disease.
  • Eligibility: patients with muscle symptoms and a mutation in mitochondrial DNA
  • Contact: l.germain@nhs.net
MRI for mitochondrial myopathy
  • Recruitment: Active
  • Description: This is a study looking at the effectiveness of new MRI techniques to monitor disease progression in mitochondrial disease
  • Eligibility: patients with mitochondrial disease due to m.3243A>G mutations or single deletions in mitochondrial DNA
  • Contact: l.germain@nhs.net
Falcon study
  • Recruitment: Trial is in ‘set up’, accepting expressions of interest from doctors and patients
  • Description: This is a trial testing the effectiveness and safety of a new medication (KL1333) for mitochondrial muscle disease.
  • Eligibility: patients with muscle symptoms and a mutation in mitochondrial DNA
  • Contact: l.germain@nhs.net
SPIMD
  • Recruitment: Trial is in ‘set up’, accepting expressions of interest from doctors and patients
  • Description: This is a trial testing the effectiveness and safety of a new medication (elamipretide) for mitochondrial muscle disease.
  • Eligibility: patients with muscle symptoms and a mutation in nuclear mitochondrial genes (e.g., POLG) or mitochondrial DNA
  • Contact: l.germain@nhs.net