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 – Bria Jensen, 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 – Bria Jensen, 801-585-9399

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 – Bria Jensen, 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 – Bria Jensen, 801-585-9399

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 – Bria Jensen, 801-585-9399

Study Sponsor – Marathon Pharmaceuticals

Facioscapulohumeral Dystrophy Trials


Evaluate Safety and Biological Activity of ATYR1940 in Patients With Early Onset Facioscapulohumeral Muscular Dystrophy – Safety and biological activity study of ATRY1940

Principal Investigator – Russell Butterfield, MD, PhD

Study Coordinator – Mary Pautler, 801-585-9717

Study Sponsor – aTyr Pharmaceuticals

Myotonic Dystrophy Trials


ISIS DMPK- A Phase 1/2a Blinded, Placebo-Controlled Study to Assess the Safety, Tolerability, and Dose-range Finding of Multiple Ascending Doses of ISIS 598769 Administered Subcutaneously to Adult Patients with Myotonic Dystrophy Type I – Safety, tolerability, and pharmacodynamic study of multiple escalating doses of ISIS-DMPKRx.

Principal Investigator – Nicholas Johnson, MD

Study Coordinator – Caren Trujillo, 801-585-0892

Study Sponsor – IONIS Pharmaceuticals

Spinal Muscular Atrophy


An Open-label Safety and Tolerability Study of IONIS SMNRx in Patients With Spinal Muscular Atrophy Who Previously Participated in IONIS SMNRx-CS2 or IONIS SMNRx-CS10 – Safety and tolerability study of multiple doses of IONIS-SMNRx.

Principal Investigator – Nicholas Johnson, MD

Study Coordinator – Nicole Rausch, 801-585-9055

Study Sponsor – IONIS Pharmaceuticals


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 – Nicole Rausch, 801-585-9055

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 – Nicole Rausch, 801-585-9055

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 – Nicole Rausch, 801-581-5522

Study Sponsor – IONIS Pharmaceuticals