Please reach out to the Research Coordinator listed as the contact for studies of interest to determine if they are currently enrolling. 

Amyotrophic Lateral Sclerosis (ALS) Studies

IBM Arimoclomol

Population: Adult

Disease: Inclusion Body Myositis

Enrolling: Not at this time

This is a Phase 2 of 3 study of Arimoclomol in the treatment of IBM. There is, additionally, and extension of this study currently active.

For more information, contact Research Coordinator Crystal Neate at: crystal.neate@hsc.utah.edu

IBM Extension

Population: Adult

Disease: Inclusion Body Myositis

Enrolling: Not at this time

An extension of the IBM Arimoclomol study.

For more information, contact Research Coordinator Crystal Neate at: crystal.neate@hsc.utah.edu

Orion REFALS

Population: Adult

Disease: Amyotrophic Lateral Sclerosis (ALS)

Enrolling: Not at this time

Effects of oral Levosimendan (ODM-109) on respirator function in patients with ALS: Open-label extension for patients completing Study 3119002 (REFALS). This study is active, but is not currently enrolling additional patients at this time.

For more information, contact Research Coordinator Mike Papadakis at: m.papadakis@utah.edu

Alexion CHAMPION ALS

Population: Adult

Disease: Amyotrophic Lateral Sclerosis (ALS)

Enrolling: Not at this time

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients with Amyotrophic Lateral Sclerosis (ALS); Adult neuro, inactive (activation planned for ~November); CHAMPION ALS is a phase 3 clinical trial of Ravulizumab in patients with ALS.

For more information, contact Research Coordinator Mike Papadakis at: m.papadakis@utah.edu

AXN005-GBS-03

Population: Adult

Disease: Amyotrophic Lateral Sclerosis (ALS)

Enrolling:

For more information, contact Research Coordinator Crystal Neate at: crystal.neate@hsc.utah.edu

MG (Viela Bio) Medpace

Population: Adult

Disease: Amyotrophic Lateral Sclerosis (ALS)

Enrolling:

 

For more information, contact Research Coordinator Crystal Neate at: crystal.neate@hsc.utah.edu

Duchenne Muscular Dystrophy (DMD)

FOR-DMD

Population: Pediatric

Disease: Duchenne Muscular Dystrophy (DMD)

Finding the Optimum Steroid Regime for Duchenne Muscular Dystrophy.

For more information, contact Research Coordinator Becky Crockett at: bcrockett@genetics.utah.edu

Pfizer C3391001

Population: Pediatric

Disease: Duchenne Muscular Dystrophy (DMD)

A Phase 1B Multicenter, Open-Label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of PF-06939926 in Ambulatory Subjects with Duchenne Muscular Dystrophy (DMD).

For more information, contact Research Coordinator Sterling Meisner at: sterling.meisner@hsc.utah.edu

Pfizer C3391003

Population: Pediatric

Disease: Duchenne Muscular Dystrophy (DMD)

A Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of PF 06939926 for the treatment of Duchenne Muscular Dystrophy. PF-06939926 is an investigational gene therapy that may restore functional dystrophin expression in the muscle cells by replacing the mutated or deleted parts of the patient’s dystrophin gene with a shortened/functional version of dystrophin called mini-dystrophin. The mini-dystrophin is packaged into a viral vector called AAV9 and then distributed to the cells using an IV infusion.

For more information, contact Research Coordinator Sterling Meisner at: sterling.meisner@hsc.utah.edu

PTC-016

Population: Pediatric

Disease: Duchenne Muscular Dystrophy (DMD)

An Open-Label, Safety Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy.

For more information, contact Research Coordinator Sarah Moldt at: sarah.moldt@hsc.utah.edu

PTC-041

Population: Pediatric

Disease: Duchenne Muscular Dystrophy (DMD)

A Phase 3, Randomized, Double-Blind, Placebo Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension. 

For more information, contact Research Coordinator Cameron Tear at: cameron.tear@hsc.utah.edu 

Sarepta I 4658

Population: Pediatric

Disease: Duchenne Muscular Dystrophy (DMD)

An Open-Label, Multi-Center 48 Week Study with a Concurrent Untreated Control Arm to Evaluate the Safety of Eteplirsen in Duchenne Muscular Dystrophy. 

Spirinolactone NWCH DMD

Population: Pediatric

Disease: Duchenne Muscular Dystrophy (DMD)

The purpose of this study is to see if Spirinolactone is safe to use as an alternative to steroid treatment in boys with Duchenne Muscular Dystrophy. 

For more information, contact Research Coordinator Becky Crockett at: bcrockett@genetics.utah.edu

Catabasis GALAXY

Population: Pediatric

Disease: Duchenne Muscular Dystrophy (DMD)

This study is still in the early stages of startup. 

For more information, contact Research Coordinator Sterling Meisner at: sterling.meisner@hsc.utah.edu

Catabasis POLARIS

Population: Pediatric

Disease: Duchenne Muscular Dystrophy (DMD)

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric.

For more information, contact Research Coordinator Sterling Meisner at: sterling.meisner@hsc.utah.edu

Capricor Hope 2

Population: Pediatric

Disease: Duchenne Muscular Dystrophy (DMD)

A Phase 2, randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of intravenous delivery of allogeneic cardiosphere-derived cells in subjects with Duchenne Muscular Dystrophy. This study is currently in the close out stage. 

For more information, contact Research Coordinator John Doyle at: john.doyle@hsc.utah.edu

Fulcrum

Population: Adult

Disease: Facioscapulohumeral Muscular Dystrophy (FSHD)

A Phase 2 Randomized, Double Blind, Placebo Controlled, 24 week, Parallel design Study of Efficacy and Safety of FTX-1821 in Treating Subjects with FSHD.

For more information, contact Research Coordinator Mike Papadakis at: m.papadakis@utah.edu

RAPharma RAISE

Population: Adult

Disease: Myasthenia Gravis

A Phase 3, multicenter, randomized, double-blind, placebo-controlled, study to confirm the safety, tolerability, and efficacy of Zilucoplan in subjects with Generalized Myasthenia Gravis.

For more information, contact Research Coordinator Teresa Janecki at: teresaj@genetics.utah.edu

RAPharma Phase 3 Extension

Population: Adult

Disease: Myasthenia Gravis

An extension of RAPharma Phase 3. 

For more information, contact Research Coordinator Teresa Janecki at: teresaj@genetics.utah.edu

Avexis Strong

Population: Pediatric

Disease: Spinal Muscular Atrophy (SMA)

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

For more information, contact Research Coordinator Teresa Janecki at: teresaj@genetics.utah.edu

Avexis LT-002

Population: Pediatric

Disease: Spinal Muscular Atrophy (SMA)

A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101.

For more information, contact Research Coordinator Teresa Janecki at: teresaj@genetics.utah.edu

Sarepta 9001-301

Population: Pediatric

Disease: Duchenne Muscular Dystrophy

A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy.

For more information, contact Research Coordinator Teresa Janecki at: teresaj@genetics.utah.edu