Clinical Trials

Below is a list of our current and upcoming Clinical Trials. For more information on Clinical Trials, the University of Utah Department of Neurology Clinical Trials Office has put together an informational page here.

The University of Utah Health Care System has more information here.



Duchenne Muscular Dystrophy Trials


Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD) – Comparison study of corticosteriods.

Principal Investigator – Russell Butterfield, MD, PhD

Study Coordinator – Becky Crockett, 801-585-1676

Study Sponsor – NIH Grant


Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy – Safety and efficacy study of PTC124.

Principal Investigator – Russell Butterfield, MD, PhD

Study Coordinator – Bryan Gardner, 801-585-9399

Study Sponsor – PTC Bio Pharmaceuticals


Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy – Phase 3 extension study of Ataluren.

Principal Investigator – Russell Butterfield, MD, PhD

Study Coordinator – Liz Diaz, 801-585-9717

Study Sponsor – PTC Bio Pharmaceuticals


A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy – Safety and efficacy study of PF-06252616.

Principal Investigator – Russell Butterfield, MD, PhD

Study Coordinator – Bryan Gardner, 801-585-9399

Study Sponsor – Pfizer


Confirmatory Study of Eteplirsen in DMD Patients (PROMOVI) – Confirmatory study of the efficacy of eteplirsen.

Principal Investigator – Russell Butterfield, MD, PhD

Study Coordinator – Brittney Holmberg, 801-585-9055

Study Sponsor – Sarepta Pharmaceuticals


An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort – Extension study of Deflazacort.

Principal Investigator – Russell Butterfield, MD, PhD

Study Coordinator – Bryan Gardner, 801-585-9399

Study Sponsor – Marathon Pharmaceuticals

Facioscapulohumeral Dystrophy Trials

Myotonic Dystrophy Trials

Spinal Muscular Atrophy


A Study to Assess the Efficacy and Safety of IONIS-SMN Rx in Infants With Spinal Muscular Atrophy – Efficacy and safety study of IONIS-SMN Rx.

Principal Investigators – Russell Butterfield, MD, PhD, and Nicholas Johnson, MD

Study Coordinator – Liz Diaz, 801-585-9717

Study Sponsor – IONIS Pharmaceuticals


A Study of Multiple Doses of ISIS SMNRx (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy – Efficacy study of multiple doses of ISIS SMNRx.

Principal Investigators – Russell Butterfield, MD, PhD, and Nicholas Johnson, MD

Study Coordinator – Liz Diaz, 801-585-9717

Study Sponsor – IONIS Pharmaceuticals


A Study of CK-2127107 in Patients With Spinal Muscular Atrophy – Pharmacodynamic study of CK-2127107.

Principal Investigator – Nicholas Johnson, MD

Study Coordinator – Liz Diaz, 801-585-9717

Study Sponsor – Cytokinetics Pharmaceuticals


An Open-Label Study (SHINE) for Patients With Spinal Muscular Atrophy (SMA) Who Participated in Studies With IONIS-SMNRx – Open-label extension study of IONIS-SMNRx.

Principal Investigators – Russell Butterfield, MD, PhD, and Nicholas Johnson, MD

Study Coordinator – Liz Diaz, 801-585-9717

Study Sponsor – IONIS Pharmaceuticals