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, bcrockett@genetics.utah.edu

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, bryang@genetics.utah.edu

Study Sponsor – Santhera/Orion


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, liz.nathalie.diaz@utah.edu

Study Sponsor – PTC Bio Pharmaceuticals


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

Principal Investigator – Russell Butterfield, MD, PhD

Study Coordinator – Bryan Gardner, 801-585-9399, bryang@genetics.utah.edu

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, brittneyh@genetics.utah.edu

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, bryang@genetics.utah.edu

Study Sponsor – Marathon Celerion


An Open-Label Extension Study to Evaluate the Safety of PF-06252616 in Boys with Duchenne Muscular Dystrophy – Safety and efficacy study of PF-06252616.

Principal Investigator – Russell Butterfield, MD, PhD

Study Coordinator – Bryan Gardner, 801-585-9399, bryang@genetics.utah.edu

Study Sponsor – Pfizer


An Open-Label, Safety Study for Previously Treated Ataluren Patients with Nonsense Mutation Dystrophinopathy – Protocol PTC124-GD-016-DMD.

Principal Investigator – Russell Butterfield, MD, PhD

Study Coordinator – Liz Diaz, 801-581-5522, liz.nathalie.diaz@utah.edu

Study Sponsor – PTC Bio


SRP-4053 and SRP-4045 in Patients with Duchenne Muscular Distrophy

Principal Investigator – Russell Butterfield, MD, PhD

Study Coordinator – Brittney Holmberg, 801-585-9055, brittneyh@genetics.utah.edu

Study Sponsor – Sarepta


Activity & Safety of Utrophin Modulation in Abulatory Paedeatric Males with Duchene Muscular Distrophy -Phase 2 Study of SMT C1100

Principal Investigator – Russell Butterfield, MD, PhD

Study Coordinator – Bryant Gordon, 801-585-5052, bgordon@genetics.utah.edu

Study Sponsor – Summit


Facioscapulohumeral Dystrophy Trials


Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy -Phase 2 Study

Principal Investigator – Nicholas Johnson, MD

Study Coordinator – Bryant Gordon, 801-585-5052, bgordon@genetics.utah.edu

Study Sponsor – Acceleron


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, liz.nathalie.diaz@utah.edu

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, liz.nathalie.diaz@utah.edu

Study Sponsor – IONIS Pharmaceuticals


Dose Comparison Study of AVXS-101 for Sitting but Non-ambulatory Patients with Spinal Muscular Atrophy – Phase I, Open-Label.

Principal Investigator – Nicholas Johnson, MD

Study Coordinator – Bryant Gordon, 801-585-5052 bgordon@genetics.utah.edu

Study Sponsor – Avexis


Gene Replacement Therapy for Patients with Spinal Muscular Atrophy – Phase 3, Open-Label, Single-Arm, Single-Dose.

Principal Investigator – Nicholas Johnson, MD

Study Coordinator – Teresa Janecki, 801-585-9717 teresaj@genetics.utah.edu

Study Sponsor – Avexis