Gene Therapy Clinical Research

Investigators at the Center for Gene Therapy in The Research Institute at Nationwide Children's Hospital are currently conducting numerous clinical research studies, described in detail below. The Neurosciences Center at Nationwide Children's offers patients and families a comprehensive approach to the care for children and adolescents with disorders of the brain, spine and nervous system; from initial diagnosis and treatment to rehabilitation and long-term follow-up care.

Learn more about the SMA type 1 clinical trial. Please note: this trial is not recruiting participants at this time. Information for parents of children with SMA1 who want to learn more about study participation can visit: studysmanow.com.

Batten Disease

Batten CLN-6 Gene Therapy Trial

Principal Investigator

Jerry Mendell, MD, Nationwide Children's Hospital

Study Title and Phase

Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscionosis, Delivering the CLN6 Gene by Self-Complementary AAV9, Phase I/IIa

Study Duration

2 years (14 visits, including one pre-screening visit prior to treatment)

Inclusion Criteria

  • Diagnosis of CLN6 disease determined by genotype available at screening

  • A score of ≥3 on the quantitative clinical assessment of the Hamburg motor-language aggregate score at screening on CLN2 disease motor-language scale, as defined in the Ratings Assessment Guideline

  • Age ≥ 1

  • Must be able to walk or walk with assistance

Exclusion Criteria

  • Presence of another inherited neurologic disease, e.g., other forms of CLN or seizures unrelated to CLN6 disease (patients with febrile seizures may be eligible)

  • Presence of another neurological illness that may have caused cognitive decline (e.g., trauma, meningitis, hemorrhage) before screening

  • Active viral infection (includes HIV or serology positive for hepatitis B or C)

  • Has received stem cell or bone marrow transplantation for CLN6 disease

  • Contraindications for spinal tap procedure (e.g. spina bifida, meningitis, impairment, or clotting abnormalities)

  • Contraindications for MRI scans (e.g., cardiac pacemaker, metal fragment or chip in the eye, aneurysm clip in the brain)

  • Episode of generalized motor status epilepticus within 4 weeks before the First Dose visit

  • Severe infection (e.g., pneumonia, pyelonephritis, or meningitis) within 4 weeks before the First Dose visit (enrollment may be postponed)

  • Has received any investigational medication within 30 days before the first infusion of study drug

  • Patients with Anti-AAV9 antibody titers > 1:50 as determined by ELISA binding immunoassay.

  • Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject’s ability to comply with the protocol required testing or procedures or compromise the subject’s wellbeing, safety, or clinical interpretability

  • Pregnancy any time during the study; pregnancy test will be given to females of childbearing age

  • Abnormal laboratory values considered clinically significant (GGT > 3XULN, Bilirubin ≥ 3.0 mg/dL , Creatinine ≥ 1.8 mg/dL, Hgb < 8 or > 18 g/Dl; WBC > 15,000 per cmm)

  • Family does not want to disclose patient's study participation with primary care physician and other medical providers.

  • Those with bleeding disorders or any other medical conditions or circumstances in which intrathecal (IT) administration of the product or lumbar puncture (for collection of CSF) are contradicted according to local institutional policy.

  • History of or current chemotherapy, radiotherapy or other immunosuppression therapy within the past 30 days. Corticosteroid treatment may be permitted at the discretion of the PI.

  • Patients with two consecutive abnormal liver tests (>2 times the upper limit of normal). Liver enzymes will be re-tested once if abnormal upon initial screening.

Coordinator Contact Information

Alana Mahley
Alana.Mahley@NationwideChildrens.org
614-355-2606

Congenital Myasthenic Syndromes (CMS)

Phase 3 Study to Evaluate the Efficacy and Safety of Amifampridine Phosphate in Patients with Congenital Myasthenic Syndromes (CMS)

Principal Investigator

Samiah A. Al-Zaidy, MD, Nationwide Children’s Hospital

Study Title and Phase

Multicenter, Double-blind, Placebo-controlled, Randomized, Outpatient Two-period Two-Treatment Crossover Study to Evaluate the Efficacy and Safety of Amifampridine Phosphate (3,4-Diaminopyridine Phosphate) in Patients with Congenital Myasthenic Syndromes (CMS), Phase 3

Study Duration

63 days (screening period excluded, can last up to 14 days)

Inclusion Criteria

  • Male or female age 2 years or older

  • Body weight ≥ 10 kg

  • Genetically-confirmed CMS (involving acetylcholine receptor defect, Rapsyn deficiency, MuSK deficiency, Dok-7 deficiency, SYT2 mutations, SNAP25B deficiency, and fast channel syndrome)

  • MFM 20 or 32 score equal or less than 48 to 76, respectively, at screening

  • If patient is naïve to amifampridine, improvement of >20% in MFM20 or MFM32 scores after open label period of uptitration of dose.

  • If patient was previously on 3,4- DAP, history of meaningful improvement in motor function.

  • Willingness to remain on stable dose of medications throughout study interval (medications include: pyridostigmine, prednisone, albuterol, ephedrine, or fluoxetine)

  • Females of childbearing age must have negative pregnancy test and be willing to use reliable contraceptives throughout the study.

  • Ability to participate in study based on overall health of patient and disease prognosis, as determined by the investigator.

Exclusion Criteria

  • CMS subtype diagnosis of acetylcholinesterase deficiency, slow-channel syndrome, LRP4 deficiency, and plectin deficiency

  • Cardiac conduction defects on screening electrocardiogram (ECG)

  • Seizure disorder

  • Clinical significant abnormal laboratory values at screening

  • Pregnant or breastfeeding

  • Any systemic bacterial or other infection

  • Treatment with investigational drug (other than amifampridine or amifampridine phosphate), device, biological agent within 30 days before screening or during study

  • Any other medical condition that might interfere with the study, in the opinion of the investigator

  • History of drug allergy to any pyridine-containing substances or any amifampridine or amifampridine phosphate excipient(s)

Coordinator Contact Information

Alana Mahley
Alana.Mahley@NationwideChildrens.org
614-355-2606

Duchenne Muscular Dystrophy (DMD)

Study to Evaluate Eteplirsen (Sarepta) in Early-Stage Duchenne Muscular Dystrophy (DMD)

Principal Investigator

Jerry Mendell, MD, Nationwide Children's Hospital

Study Title and Phase

An Open-Label, Multi-Center Phase III Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early-Stage Duchenne Muscular Dystrophy (DMD)

Study Duration

One year. 20 centers in the United States are participating in this trial.

Inclusion Criteria

  • Age four to six years old

  • Confirmed Duchenne Muscular Dystrophy (DMD) with out-of-frame deletion amenable to exon 51 skipping

  • Two intact upper arm muscle groups

  • Stable corticosteroid use, for at least 12 weeks prior to study

    • Or no steroid use for at least 12 weeks prior to the study and not expected to start use within the first 12 weeks of the trial

Exclusion Criteria

  • Left ventricular ejection fraction (LVEF) < 50%

  • QTcF > 450 msec

  • Drug treatment that could affect muscle function, within previous 12 weeks

    • Growth hormone

    • Anabolic steroids

  • Other experimental treatment, within previous 12 weeks

  • Systemic aminoglycoside antibiotic use, within previous 12 weeks

  • Clinically significant illness (cardiac, hepatic, renal, etc. diseases)

  • Major surgery, within 3 months prior to trial

  • Ongoing illness or need for treatment in which principal investigator deems unfit for trial

Coordinator Contact Information

Roush, Kandice 
Kandice.Roush@nationwidechildrens.org 
(614) 722-2558

Study to Evaluate Eteplirsen (Sarepta) in Older Children with Duchenne Muscular Dystrophy (DMD)

Principal Investigator

Jerry Mendell, MD, Nationwide Children's Hospital

Study Title and Phase

An Open-Label, Multi-Center 48-Week Phase III Study with a Concurrent Untreated Control Arm to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Duchenne Muscular Dystrophy (DMD)

Study Duration

One year. 39 international sites are participating in this trial.

Inclusion Criteria

  • Age 7 to 16 years old

  • Treated Group

    • Confirmed Duchenne Muscular Dystrophy (DMD)

    • Out-of-frame deletion

    • Amenable to exon 51 skipping

  • Untreated Group

    • Confirmed Duchenne Muscular Dystrophy (DMD)

    • Out-of-frame deletion

    • Only amenable to other exon skipping (not exon 51)

  • Two intact upper arm muscle groups

  • Stable corticosteroid use, for at least 24 weeks prior to study

    • ACE inhibitors, ARBs, beta blockers and potassium are permitted

  • Ambulatory > 300m on 6MWT (2-day average, within 15%)

  • Cardiac and pulmonary function unlikely to deteriorate over the study period

    • Forced vital capacity (FVC) % of predicted > 50%

    • No nocturnal ventilation

  • Use of reliable method of contraception (if sexually active)

Exclusion Criteria

  • Drug treatment that could affect muscle function, within previous 12 weeks

    • Growth hormone

    • Anabolic steroids

  • Previous experimental treatment

    • SMT C1100/BMN 195

    • Drisapersen (PRO051), within previous 6 months

    • Shock Training System (STS), during study

  • Systemic aminoglycoside antibiotic use, within previous 12 weeks

  • Clinically significant illness (cardiac, hepatic, renal, etc. diseases)

  • Major surgery, within 3 months prior to trial

Coordinator Contact Information

Roush, Kandice 
Kandice.Roush@nationwidechildrens.org 
(614) 722-2558

Research Study for Males 10 Years and Older with Duchenne Muscular Dystrophy

What is the purpose of the study?

The purpose of this study is to see if both spironolactone and eplerenone (Inspra), generic drugs used in other types of heart conditions, help maintain heart and lung function in boys with Duchenne Muscular Dystrophy (DMD).

Who is eligible for the study?

If you are a male with DMD, at least 10 years old, AND:

  • Non-ambulatory

  • Do not have kidney problems

  • Not taking eplerenone or spironolactone

  • Can undergo a cardiac magnetic resonance imaging (MRI) scan without sedation

  • Have preserved heart function (left ventricular ejection fraction ≥45%±5%)

How long will this study last and how many study visits are there?

  • The study lasts for 12 months.

  • After the initial (baseline) screening visit, there are five (5) study visits during the 12-month period.

  • You will receive a follow-up phone call two weeks after enrollment. 

  • The study visits after the baseline visit will happen during the following time periods: 1 month, 2 months, 3 months, 6 months, 9 months and 12 months.

Where do the study visits take place?

  • The baseline visit will take place at one of the following:

    • The Ohio State University (OSU) in Columbus, OH 

    • Mattel Children’s Hospital in Los Angeles, CA

    • University of Colorado Hospital in Aurora, CO

    • University of Utah Hospital in Salt Lake City, UT

  • Blood draw at 1, 2, 3, 6, and 9 months can be done at a local laboratory where you reside.

  • The 12-month visit will be done at the same site where you had your baseline visit.

What will happen during this study?

  • Participants will be randomly assigned to receive either eplerenone or spironolactone.

  • Participants will take one tablet of the assigned drug once daily for duration of the study.

  • All participants will have blood drawn at the baseline visit as well as at months 1, 2, 3, 6, 9 and 12.

  • All participants will have a cardiac MRI scan and pulmonary function tests at baseline and 12 months.

There is no payment for participating in this study, but you will be reimbursed for travel, food and lodging expenses at the baseline and 12-month visit. The study drug will be provided by the study, and the blood draws will be paid for by the study. The cardiac MRI scan and pulmonary functional test at baseline and 12-month visit will be paid for by the study, if it is not part of your regular clinical visit.

What are the benefits of participating?

If the results are promising, this medication may be available to you or your child after the study has ended. While no personal benefit can ever be guaranteed, there are other benefits to participating in a research study. You may be able to:

  • Play an active role in your own health care (or that of your child)

  • Gain access to new research treatments before they are widely available

  • Have access to medical specialists who may not normally be available

  • Help others by contributing to the better understanding and treatment of DMD-associated heart disease

How do I learn more about this study?

You can learn more about this study at ClinicalTrials.gov. You may also contact:

Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD

Principal Investigator

Samiah A. Al-Zaidy, MD, Nationwide Children’s Hospital

Study Title and Phase

Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD

Study Duration

2 years (3 visits over 24 months)

Inclusion Criteria

  • Age > 18

  • Cohort A requires confirmed mutation in the DMD gene with an affected son

  • Cohort B included DMD/BMD mothers with no somatic mutation in the DMD gene

  • Cohort C age-matched healthy controls with a normal CK level

  • Able to complete testing in English

  • Able to consent

Exclusion Criteria

  • Contraindication to cardiac MRI

  • Subjects on heart failure medication at the time of enrollment

  • Subjects on steroid medications

  • Presence of an inherited neurologic disease or comorbidity that may affect their ability to complete the study

  • Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject’s ability to comply with the protocol required testing or procedures or compromise the subject’s well-being, safety, or clinical interpretability

  • Pregnancy at any time during the study

Coordinator Contact Information

Daniel Jenkins
Daniel.Jenkins@NationwideChildrens.org
614-355-3424

FOR Duchenne Muscular Dystrophy Study (FOR DMD Study)

Purpose of Study

The Newcastle University and the University of Rochester, in collaboration with the United States National Institutes of Health, and the National Institute of Neurological Disorders and Stroke, are conducting a phase III study with corticosteroids in boys with Duchenne Muscular Dystrophy (FOR DMD study).

Corticosteroids are currently the only medicine that has been shown to increase muscle strength in boys with DMD. Doctors have tried different ways of prescribing corticosteroids in order to decrease undesirable side effects. Currently, different doctors in different countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at all.

The FOR DMD study aims to compare three ways of giving corticosteroids to boys with DMD to determine which increases muscle strength the most, and which causes the fewest side effects.

Using the results of this study, we aim to provide patients and families with clearer information about the best way to take these drugs.

This study will look at three ways of taking corticosteroids by the mouth:

  • Prednisone 0.75mg/kg/day
  • Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment
  • Deflazacort 0.9mg/kg/day

All three dosages are commonly used in boys with DMD and have shown to be beneficial.

In this study there is no placebo group, which means that all participants will receive active drugs (Prednisone or Deflazacort). However, neither the boys nor the clinicians will know which treatment or regime the boy is taking.

The study will recruit 300 boys around Europe, United States and Canada.

In North America, 16 centers will take part in the study:

  • Alberta Children's Hospital Penn State Children's Hospital
  • Children's Memorial Hospital, Chicago SUNY Downstate Med Center
  • Health Sciences Centre Winnipeg University of California, Davis
  • Kansas University Medical Center University of California Los Angeles
  • Kennedy Krieger Institute University of New Mexico
  • London Health Sciences Center University of Rochester Medical Center
  • Nemours Children’s Hospital, Orlando University of Texas Southwestern Medical Center
  • Nationwide Children’s Hospital Vanderbilt Children's Hospital

Patients who do not attend one of these hospitals for their routine follow-up can also participate, but will have to travel to their closest participating site to receive the study drug and for the check-ups.

Participants will receive study medication for a minimum of three years and a maximum of five years (depending on how early the boy was recruited into the study) and participation involves visits to the study hospital every three months for the first 6 months and every six months thereafter. At these visits we will be repeating many of the tests your child usually has in clinic for his routine DMD follow up.

Who can participate:

In order to take part in the study boys need to fulfil a number of criteria. These can only be checked when you come into the clinic. However, at this stage if your child may be eligible if he:

  • Has a genetically confirmed diagnosis of DMD
  • Is aged 4-7 years (before 8th birthday)
  • Has never taken steroids (by mouth)

Who to contact:

If you feel that your child might be able to participate in this trial, please feel free to discuss it with your doctor locally. Alternatively, if you would like further information, please contact the University of Rochester Medical Center: Kim Hart | Phone: 1 (585) 275-3767.

Limb Girdle Muscular Dystrophy (LGMD)

Gene Transfer Clinical Trial for Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)

Principal Investigator

Jerry Mendell, MD, Nationwide Children's Hospital

Study Title and Phase

Phase I/II gene transfer clinical trial for Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)

Procedure

The vector delivered via direct intravascular delivery to a lower limb artery to a single leg (cohort 1A) or to both legs (cohort 1B and 2). Study participants will receive general anesthesia for the procedure.

Study Duration

Two years. Follow-up visits after 1 week, 2 weeks, 1 month, 2 months, 3 months, 6 months, 1 year and 2 years. 

Inclusion Criteria

  • Confirmed alpha-sarcoglycan deficiency

  • Males or females (of any ethnic group) eligible

  • Age 7 years or older

    • Cohort 1A: Adult, wheelchair dependent

    • Cohort 1B and 2: Ambulant, Gower's, uses handrail for stairs, performs 6MWT with >2SD below average

  • Onset of weakness by age 5

  • Able to cooperate with muscle testing

  • Use of reliable method of contraception (if sexually active)

Exclusion Criteria

  • Active viral infection

  • No weakness or loss of function (despite LGMD-2D diagnosis)

  • Symptoms of cardiomyopathy

    • Dyspnea on exertion

    • Shortness of breath when supine (lying down)

    • Echocardiogram with ejection fraction < 40%

    • Pedal edema

    • Rales at base of lungs

  • Ongoing illness or need for drug treatment which principal investigator deems unsafe for gene transfer

  • Clinically significant, abnormal lab values for GGT, creatinine, WBC, bilirubin and Hgb

  • Pregnancy

  • HIV or autoimmune disease

  • Hep A, B, or C infection

  • rAAVrh74 or AAV8 antibody titer >1.50 by ELISA testing

Coordinator Contact Information

Beverly Galliers
Beverly.Galliers@NationwideChildrens.org
614-355-3424

Gene Therapy Trial for Limb Girdle-Muscular Dystrophy Type 2B (LGMD2B)

Principal Investigator

Jerry Mendell, MD, Nationwide Children's Hospital

Study Title and Phase

Intramuscular Gene Transfer Clinical Trial for Dysferlin Deficiency Delivering the Dysferlin Gene by AAVrH74, Phase I

Study Duration

2 years (11 visits, including one pre-screening visit prior to treatment)

Inclusion Criteria

  • 18 years or older

  • Non-ambulant (cannot walk 10 meters in ≤ 30 seconds)

  • Established mutations of the Dysferlin gene on both alleles

  • Impaired muscle function, but with sufficient muscle preservation to ensure muscle transfection based on MRI or ultrasound

  • Willingness to practice reliable contraception methods for subjects who are sexually active with reproductive capacity, during the first 6 months after gene transfer

Exclusion Criteria

  • Active viral infection (includes HIV or serology positive for hepatitis B or C)

  • Presence of Dysferlin mutations without weakness or loss of function

  • Symptoms or signs of cardiomyopathy

  • Diagnosis of (or ongoing treatment for) an autoimmune disease

  • Persistent leukopenia or leukocytosis (WBC ≤ 3.5 K/µL or ≥ 20.0 K/µL) or an absolute neutrophil count < 1.5K/µL

  • Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer

  • Pregnancy

  • AAVrh74 or AAV8 binding antibody titers > 1:50 as determined by ELISA immunoassay

  • Abnormal laboratory values in the clinically significant range as designated by the PI for kidney function, liver function and hemologic studies

Coordinator Contact Information

Beverly Galliers
Beverly.Galliers@NationwideChildrens.org
614-355-3424

Mucopolysaccharidosis (MPS) IIIA

Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

Principal Investigator

Kevin Flanigan, MD, Nationwide Children's Hospital

Study Title and Phase

Phase I/II gene transfer clinical trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

Procedure

Adeno-associated virus serotype 9 carrying the human SGSH gene under the control of an U1a promoter will be delivered one time through a venous catheter inserted into a peripheral limb vein while under sedation.

Study Duration

Two years. Follow-up visits include Days 7, 14, 30, 60, 90, and 180 as well as Months 6, 12, 18 and 24.

Inclusion Criteria

  • Age 2 years old or greater

  • Confirmed diagnosis of MPS IIIA by either of two methods:

  • No detectable or significantly reduced* SGSH enzyme activity by leukocyte or fibroblast assay.

  • Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene

  • Clinical history or examination features of neurologic dysfunction

Exclusion Criteria

  • Inability to participate in the clinical evaluation as determined by PI

  • Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics

  • Inability to be safely sedated in the opinion of the clinical anesthesiologist

  • Active viral infection based on clinical observations

  • Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer

  • Subjects with anti-AAV9 antibody titers ≥ 1:50 as determined by ELISA binding immunoassay

  • Serology consistent with exposure to HIV, or serology consistent with active hepatitis A, B or C infection

  • Bleeding disorder or any other medical condition or circumstance in which a lumbar puncture (for collection of CSF) is contraindicated according to local institutional policy

  • Visual or hearing impairment sufficient to preclude cooperation with neurodevelopmental testing

  • Uncontrolled seizure disorder, due to the requirement for multiple MRI examinations as part of the study protocol. Subjects who are stable on anticonvulsive medications may be included

  • Any item (braces, etc.) which would exclude the patient from being able to undergo MRI according to local institutional policy

  • Any item (braces, etc.) which would exclude the patient from being able to undergo MRI according to local institutional policy

  • Patients with cardiomyopathy or significant congenital heart abnormalities

  • The presence of significant non-MPS IlIA related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study

  • Abnormal, clinically significant laboratory values based upon normal values in the Nationwide Children's Hospital Laboratory as listed in Table 1 of the protocol.

  • Due to the nature of enzyme activity testing, normal ranges and reported units vary from lab to lab. Many laboratories utilize control samples rather than normal ranges, to account for the influence of small day-to-day fluctuations in the laboratory environment.

    • For the purposes of invitation to a screening visit, we will accept "significantly reduced" results as those interpreted as such by any clinical laboratory approved to perform this diagnostic test.

    • For uniformity of data for analysis, and confirmation of accurate diagnosis before gene transfer, subjects who consent to complete the screening visit will have their blood drawn for confirmatory enzyme activity level to be performed by Greenwood Genetics Center Biochemical Laboratory. Subjects must have an enzyme activity level considered to be in the affected range by Greenwood Genetic Center Biochemical Laboratory to proceed within the study.

Coordinator Contact Information

Krista Kunkler
Krista.Kunkler@NationwideChildrens.org

Spinal Muscular Atrophy Rich Text

Phase I/IIa Trial of scAAV1.tMCK.NTF3 for CMT1A

Principal Investigator
Zarife Sahenk, MD, PhD, Nationwide Children’s Hospital

Study Title and Phase
Phase I/IIa Trial of scAAV1.tMCK.NTF3 for CMT1A

Procedure
An open-label, one-time injection ascending dose study in which scAAV1.tMCK.NTF3, Neutrophin Factor 3 Vector, will be administered by intramuscular injections into muscles in both legs in Charcot-Marie-Tooth disease type 1A subjects with PMP22 gene duplication. Cohort 1 will include three subjects ages 18 to 35 years receiving (2e12 vg/kg), and Cohort 2 will include six subjects ages 15 to 35 years old receiving (6e12 vg/kg).

Study Duration
2 years. Post-gene transfer monitoring will include follow up visits on days 7, 14, 30, 60, 90, 120, and months 6, 9, 12, 15, 18 and 24 following gene transfer. 

Inclusion Criteria
  • Subjects 15- 35 years old inclusive with CMT1A will be enrolled (Cohort 1 will only include subjects that are 18 to 35 years of age)
  • Must exhibit a 1.5 Mb duplication at 17p11.2 inclusive of the peripheral myelin protein 22 (PMP22) gene
  • Males and females of any ethnic or racial group
  • Must exhibit weakness of the ankle dorsiflexion muscle (but has full ROM against gravity and is able to stand on heels 3 seconds or greater)
  • Abnormal nerve conduction velocities
  • Ability to cooperate for clinical evaluation and repeat nerve conduction studies
  • Willingness of sexually active subjects to practice a reliable method of contraception during the study

Contact
Stephanie Diemer, 614-355-2679, Stephanie.Diemer@NationwideChildrens.org.

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