Research

Current Clinical Trial Research

The MD Center at The University of Minnesota is working hard to provide clinical research trials in the various muscular dystrophies. It is our hope and our goal to provide clinical research trials for all those in our MD Community.

You can find the current available studies offered at the University of Minnesota below. Some of these clinical trials are through MD Center Researchers at the U of MN and some are through pharmeceutical partners. You can reach out to our clinical research team to learn more and to inquire about enrolling in specific studies by emailing mdcenter@umn.edu or calling (612) 626-0822.

Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

The current MD Center Clinical Trials for Duchenne Muscular Dystrophy, including enrollment criteria, are below. If you are interested in participating in a Clinical Trial at the U of MN, please contact mdcenter@umn.edu or call (612) 626-0822.

Study Name
Description
Diagnosis
Enrollment Criteria
Outcome Measures for Neuromuscular Disease Urine metabolomic study in all neuromusuclar disease. DMD Samples collected from both MD patients as well as control patients.
 New tools used to study effects of growth hormone therapy on bone health and muscle function in boys with Duchenne muscular dystrophy Bone health study looking at DMD boys on growth hormone vs. no growth hormone DMD Enrollment Closed.
 An Open-Label, Multi-Center, 48-Week Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy FDA approved                                Exon skipping trial in Duchenn using Sarepta drug Eteplirsen. DMD Enrollment Closed.
 A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF PF-06252616 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY Myostatin inhibitor drug trail in ambulatory Duchenne males. DMD Enrollment Closed.
A multicenter, open-label extension study to evaluate the long term safety of PF-06252616 in boys with Duchenne Muscular Dystrophy Myostatin inhibitor drug trail in ambulatory Duchenne males. DMD Must have completed Pfizer Study B5161002 through Week 97. Normal hepatic function on screening lab assessments, in range GLDH values, and normal range of iron content in the liver.
Role of Hyposxia in the heart disease of DMD - Biomarkers Heart disease in DMD patients DMD Documented and geneticall confirmed diagnosis of DMD, male, > 6 years of age (no maximum age)
Role of Hyposxia in the heart disease of DMD Heart disease in DMD patients DMD Documented and geneticall confirmed diagnosis of DMD, male, > 6 years of age (no maximum age), no prior use of nocturnal ventilatory support, clinically determined to require the initiation of ventilatory support within 180 days
Study Name Description Diagnosis Enrollment Criteria
Givinostat   DMD Ambulant males >6  years with DMD characteristic clinicl symptoms or signs, confirmed DMD diagnosis via genetic testing, able to complete 2 Four Stairs Climb Tests, rise to floor time of <10 seconds, manual muscle testing of quadricepts > Grade 3, in range of the protocol specific functional algorithm predictive of vastus lateralis muscle fat fraction, have used corticosteroids for a minimum of 6 months.
An Open-Label, Safety Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy Ataluren open access trials for studies 007 and 008 DMD Genetically confirmed DMD males previously treated with Ataluren
PITT0112: Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping Becker natural history study. DMD Genetically confirmed BMD ambulatory males
A PHASE 3 EFFICACY AND SAFETY STUDY OF ATALUREN (PTC124) IN PATIENTS WITH NONSENSE MUTATION DYSTROPHINOPATHY Ataluren trial for ambulatory boys with nonsense mutations DMD Genetically confirmed DMD males previously treated with Ataluren
Hypophosphatasemia in DMD Alkaline phsphatase in bone health DMD Diagnosis of DMD, male, ages 6-18 yrs old. Specific growth hormone criteria. 
A phase 3, randomized, dbl-blind, placebo-controlled efficacy and safety study of Ataluren in Patients with nonsense mutation DMD and Open-Label extension Ataluren trial for ambulatory boys with nonsense mutations DMD Ages ≥6 yrs ambulatory males with nonsense DMD

Charcot Marie Tooth (CMT)

Charcot Marie Tooth (CMT)

The current MD Center Clinical Trials for Charcot Marie Tooth (CMT), including enrollment criteria, are below. If you are interested in participating in a Clinical Trial at the U of MN, please contact mdcenter@umn.edu or call (612) 626-0822.

Study Name
Description
Diagnosis
Enrollment Criteria
A phase 2 randomized, dbl-bl, placebo-controlled study of ACE-083 in patients with CMT To evaluate safety, tolerability, PK and PD effects on muscle using ACE-083 (recombinant fusion protein form of human follistatin linked to a human IgG2 Fc domain) CMT Ages ≥18 yrs with CMT1 or CMTX
Muscle cramps in CMT (FLX-787-204) Muscle cramp treatment with FLX-787-ODT agent CMT Genetically confrimed ALS  male/female ages ≥18 yrs. Presense of cramps. 

Facioscapulohumeral Muscular Dystrophy (FSHD)

Facioscapulohumeral Muscular Dystrophy (FSHD)

The current MD Center Clinical Trials for Facioscapulohumeral (FSHD), including enrollment criteria, are below. If you are interested in participating in a Clinical Trial at the U of MN, please contact mdcenter@umn.edu or call (612) 626-0822.

Study Name
Description
Diagnosis
Enrollment Criteria
Muscle biopsy for the study of FSHD (Kyba) To determine molecular & cellular defect on the cells of FSHD muscle FSHD Patients: Ages ≥18 yrs with FSHD
Controls: Ages ≥18 yrs healthy first degree family member/sibling
A phase 2 randomized, dbl-bl, placebo-controlled study of ACE-083 in patients with FSHD  To evaluate safety, tolerability, PK and PD effects on muscle using ACE-083 (recombinant fusion protein form of human follistatin linked to a human IgG2 Fc domain) FSHD Ages ≥18 yrs with FSHD1 or FSHD2

Amytrophic Lateral Sclerosis (ALS)

Amytrophic Lateral Sclerosis (ALS)

The current MD Center Clinical Trials for Amytrophic Lateral Sclerosis (ALS), including enrollment criteria, are below. If you are interested in participating in a Clinical Trial at the U of MN, please contact mdcenter@umn.edu or call (612) 626-0822.

Study Name
Description
Diagnosis
Enrollment Criteria
Muscle cramps in ALS (FLX-787-203) Muscle cramp treatment with FLX-787-ODT agent ALS Genetically confrimed CMT male/female ages ≥18 yrs. Presense of cramps. 

The most up-to-date and comprehensive place to find a description of clinical trials available in the US for those living with a Neuromuscular Disease is on http://www.clinicaltrials.gov, as required by U.S. Law.