XClose

UCL Great Ormond Street Institute of Child Health

Home

Great Ormond Street Institute of Child Health

Menu

Clinical Trials

Message to the families about trials and their recruitment

Current Trials

 

Skip E (18NM19)

Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen 

Further information:

Baby Skip Extension (19NM20)

An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 

Further information:

IONIS/Shine (15NM30)

An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102

Further information: 

Jewelfish (18NM48)

An open-label study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of Risdiplam (RO7034067) in adult and pediatric patients with spinal muscular atrophy

Further information: 

SMA-REACH

Spinal Muscular Atrophy Research and Clinical Hub UK (SMA REACH UK)

Further information: 

North STAR

The U.K. NorthStar Clinical Network - Natural history and standards of care in Duchenne Muscular Dystrophy
Further information: 

CMT natural history

A Natural History Study of CMT1B, CMT2A, CMT4A and CMT4C
Further information:

Rare Disease Study 

Neuromuscular Rare Disease Translational Research in patients with Duchenne Muscular Dystrophy
Further information:

KineDMD 

KINEDMD: A study of kinematics in Duchenne Muscular Dystrophy
Further information:

 

PTC Therapeutics STRIDE

Long-Term Observational Study of Translarna (Ataluren) Safety and Effectiveness in Usual Care
Further information:

BIND WP5 part 1 (questionnaire study)

Brain Involvement in Dystrophinopathies
Further information:

AFM Natural History Study

Outcome measures in Duchenne Muscular Dystrophy: A Natural History Study
Further information:

Brain DMD

Restoring Dystrophin Expression In Brain To Improve Behavioural And Emotional Problems In Duchenne Muscular Dystrophy
Further information:

 

 

Closed Trials

SIDEROS  16NM17 :  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.

Sideros-E  18BB36 :  A Phase lii Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ldebenone in Patients with Duchenne Muscular Dystrophy {DMD) who completed the SIDEROS study

BMS Spitfire WN40227 17NM18 : A RANDOMIZED, DOUBLE BLIND, PLACEBO-CONTROLLED, STUDY TO ASSESS THE EFFICACY, SAFETY, AND TOLERABILITY OF RO7239361 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY

AveXis LTFU (STR1VE) 17NM10 :   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

SPR1NT (18NM18) 18NM18 : A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-Symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2.

Italfarmaco 16NM23 : Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.

Baby Skip (17NM07) 17NM07 :  An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping.

FOR-DMD  12DN09 : Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen.

Vision-DMD (18BB17) : Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD).

Muscle MRI in CMT study 18NM33

EmoDe 17NM38 : A Study of Emotional Function in Duchenne Muscular Dystrophy (EmoDe Study)

 

 

 

 

 

 

 

 

 

 

Title placeholder

Content placeholder