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
A Phase 3 study of Arimoclomol in inclusion body myositis (IBM)
For more information, contact Research Coordinator Crystal Neate at: crystal.neate@hsc.utah.edu
Healey ALS Platform Trial
Population: Adult
Disease: Inclusion Body Myositis
Enrolling: Not at this time
The Healey Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
For more information, contact Research Coordinator Mike Papadakis at: m.papadakis@hsc.utah.edu
Biogen ATXN2
Population: Adult
Disease: Amyotrophic Lateral Sclerosis (ALS)
Enrolling: Yes
A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB105 Administered Intrathecally to Adults with Amyotrophic Lateral Sclerosis with or Without Poly-CAG Expansion in the Ataxin-2 Gene.
For more information, contact Research Coordinator Mike Papadakis at: m.papadakis@utah.edu
Duchenne Muscular Dystrophy (DMD)
Pfizer C3391001
Population: Pediatric
Disease: Duchenne Muscular Dystrophy (DMD)
Enrolling: Yes
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)
Enrolling: Not at this time
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)
Enrolling: Yes
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)
Enrolling: Not at this time
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 Carly Straley at: carly.straley@hsc.utah.edu
Sarepta 4045-302
Population: Pediatric
Disease: Duchenne Muscular Dystrophy (DMD)
Enrolling: Not at this time
Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen.
For more information, contact Research Coordinator Becky Crockett at: bcrockett@genetics.utah.edu
Spirinolactone NWCH DMD
Population: Pediatric
Disease: Duchenne Muscular Dystrophy (DMD)
Enrolling: Yes
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 Shelby Murdock at: shelby.murdock@hsc.utah.edu
Sarepta 9001-301 EMBARK
Population: Pediatric
Disease: Duchenne Muscular Dystrophy
Enrolling: Not at this time
A phase 3 study for an investigational gene therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.
For more information, contact Research Coordinator Teresa Janecki at: teresaj@genetics.utah.edu
Sarepta 9001-303 ENVISION
Population: Pediatric
Disease: Duchenne Muscular Dystrophy
Enrolling: Not at this time
This study is still in the pre-start-up phase.
For more information, contact Research Coordinator Sarah Moldt at:
Catabasis POLARIS
Population: Pediatric
Disease: Duchenne Muscular Dystrophy (DMD)
Enrolling: Closed to enrollment
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
FOR-DMD
Population: Pediatric
Disease: Duchenne Muscular Dystrophy
Enrolling: Yes
***.
For more information, contact Research Coordinator Becky Crockett at: becky.crockett@hsc.utah.edu
Sarepta DMD-NHS (SRP-9001-501)
Population: Pediatric
Disease: Duchenne Muscular Dystrophy
Enrolling: Not at this time
***.
For more information, contact Research Coordinator Shelby Murdock at: shelby.murdock@hsc.utah.edu
Facioscapulohumeral Muscular Dystrophy (FSHD)
Fulcrum
Population: Adult
Disease: Facioscapulohumeral Muscular Dystrophy (FSHD)
Enrolling: Not at this time
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
Leukodystrophy
Passage Bio
Population: Pediatric
Disease: Krabbe
Enrolling: Not at this time
A phase 1/2 open-label, multicenter, dosing ranging and confirmatory study to assess the safety, tolerability, and efficacy of PBKR03 administered to pediatric subjects with Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy). PBKR03 is being developed to treat early infantile Krabbe disease, with a single dose of PBKR03 administered by ICM injection. PBKR03 has the potential to address unmet needs in early infantile Krabbe disease patients by providing a fast-acting, disease-modifying treatment to patients for whom there exists limited options for therapy.
For more information, contact Research Coordinator Kelsee Parry at: kelsee.parry@hsc.utah.edu
Myasthenia Gravis
RA 101495-201
Population: Adult
Disease: Myasthenia Gravis
Enrolling: Not at this time
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of RA101495 in Subjects with Generalized Myasthenia Gravis. Subjects will be randomized in a 1:1:1 ratio to receive daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 weeks.
For more information, contact Research Coordinator Teresa Janecki at: teresaj@genetics.utah.edu
RA 101495 Phase 3 301 (RAISE)
Population: Adult
Disease: Myasthenia Gravis
Enrolling: Currently Enrolling
RA 101495 Phase 3 301 (RAISE): 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. Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg Zilucoplan or placebo for 12 weeks.
For more information, contact Research Coordinator Teresa Janecki at: teresaj@genetics.utah.edu
RA 101495 Phase 3 Ext. (RAISE-XT)
Population: Adult
Disease: Myasthenia Gravis
Enrolling: Open to Enrollment
RA 101495 Phase 3 Ext. (RAISE-XT): A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects with Generalized Myasthenia Gravis.
For more information, contact Research Coordinator Teresa Janecki at: teresaj@genetics.utah.edu
MG Viela Bio
Population: Adult
Disease: Myasthenia Gravis
Enrolling: Yes
A Randomized, Double-Blind, Multicenter, Placebo-Controlled Phase 3 Study with Open-Label Period to Evaluate the Efficacy and Safety of Inebilizumab in Adults with Myasthenia Gravis. The study will enroll approximately 252 subjects >18 years, comprising 172 subjects (86 per treatment group) with AChR-Ab+MG and 80 subjects (40 per treatment group) with MuSK-Ab+MG, randomized 1:1 to receive 300mg of inebilizumab or placebo via IV on three separate days. Subjects will be followed for 52 weeks for observation and evaluation. ACTIVELY SCREENING AND ENROLLING PATIENTS
For more information, contact Research Coordinator Crystal Neate at: crystal.neate@hsc.utah.edu
Spinal Muscular Atrophy (SMA)
Avexis Strong
Population: Pediatric
Disease: Spinal Muscular Atrophy (SMA)
Enrolling: Not at this time
Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting but Non-Ambulatory Patients with Spinal Muscular Atrophy. On regulatory pause, but anticipate restart in late 2020 or early 2021 for patients with type 2 SMA.
For more information, contact Research Coordinator Teresa Janecki at: teresaj@genetics.utah.edu
Avexis LT-002
Population: Pediatric
Disease: Spinal Muscular Atrophy (SMA)
Enrolling: Only open to patients previously treated with Avexis
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
CS11
Population: Pediatric
Disease: Spinal Muscular Atrophy (SMA)
Enrolling: Not at this time
An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of 396443 – Type I, II, & III.
For more information, contact Research Coordinator Kelsee Parry at:
Nurture
Population: Pediatric
Disease: Spinal Muscular Atrophy (SMA)
Enrolling: Not at this time
An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy Type I.
For more information, contact Research Coordinator Kelsee Parry at:
Biogen RESPOND SMA Spinraze
Population: Pediatric
Disease: Spinal Muscular Atrophy (SMA)
Enrolling: Not at this time
A Phase IV study enrolling Summer 2021 for patients 3 to 36 months with previous gene therapy treatment.
For more information, contact Research Coordinator Shelby Murdock at: