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Trial record 1 of 1 for:    NCT03492346
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Limb Girdle Muscular Dystrophy Type 2E Recruitment Study

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ClinicalTrials.gov Identifier: NCT03492346
Recruitment Status : Recruiting
First Posted : April 10, 2018
Last Update Posted : December 14, 2018
Sponsor:
Collaborator:
Myonexus Therapeutics
Information provided by (Responsible Party):
Jerry R. Mendell, Nationwide Children's Hospital

Tracking Information
First Submitted Date April 3, 2018
First Posted Date April 10, 2018
Last Update Posted Date December 14, 2018
Actual Study Start Date March 28, 2018
Estimated Primary Completion Date March 28, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 3, 2018)
Baseline Measurements [ Time Frame: 2 years ]
Establish baseline measurements for potential subjects that may be enrolled into a gene therapy trial.
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT03492346 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: April 3, 2018)
  • Disease Progression [ Time Frame: 2 years ]
    Better define the rate of disease progression and skeletal muscle involvement
  • Registry [ Time Frame: 2 years ]
    Generate a registry of well-characterized LGMD2E patients
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Limb Girdle Muscular Dystrophy Type 2E Recruitment Study
Official Title Limb Girdle Muscular Dystrophy Type 2E Recruitment Study
Brief Summary This study is to recruit and establish baseline measurements for potential subjects that may be eligible for a gene therapy trial. Specifically, this trial is recruiting individuals who are suspected or have been confirmed to have Limb Girdle Muscular Dystrophy type 2E (LGMD2E).
Detailed Description

This is a longitudinal observational study. It is a 24-month study with the possibility of extending the data time points. Visits will occur monthly. However, at the discretion of the PI, subjects may not be required to return monthly. These subjects may return at intervals ranging from 2 months to a max of 6 months apart.

In the situation that the subjects would fall out of the inclusion criteria or not be eligible for the LGMD2E gene therapy trial, they will be given the opportunity to roll over into the Natural History for LGMD (IRB17-01086). If a subject is invited to screen for the gene therapy trial they will discontinue this trial. By being in this study, it is not a guarantee that subjects will be invited to screen for the LGMD2E gene therapy trial.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population
  • Confirmed LGMD2E diagnosis by genetic testing or
  • Suspected of having LGMD type 2E due to symptoms and a diagnosed family member or a member of a community with a large population of one of these two types
Condition Limb-Girdle Muscular Dystrophy, Type 2E
Intervention Not Provided
Study Groups/Cohorts LGMD2E Subject Population

Individuals:

  • Confirmed LGMD2E diagnosis by genetic testing or
  • Suspected of having LGMD type 2E due to symptoms and a diagnosed family member or a member of a community with a large population of one of these two types
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: April 3, 2018)
25
Original Estimated Enrollment Same as current
Estimated Study Completion Date March 28, 2020
Estimated Primary Completion Date March 28, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Age 3-15 inclusive
  • Males or females of any ethnic group
  • SGCB DNA gene mutations at both alleles or suspected to have LGMD2E based on family and medical history. If suspected, genetic testing will be performed to confirm diagnosis.
  • Weakness demonstrated based on history of difficulty running, jumping and climbing stairs
  • Ability to complete 100MW timed test within 30-90% predicted
  • Perform assessments to the best of their ability with reliable results as deemed by the evaluator.
  • Ability to attend scheduled appointments
  • Ability to provide informed consent (or assent for ages 9-15)

Exclusion Criteria:

  • Confirmed diagnosis of neuromuscular disorder other than LGMD2E
  • 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
  • Subjects with AAVrh74 binding antibody titers > 1:400 as determined by ELISA immunoassay. If endpoint titer is positive at screening, testing may be repeated in 1 month. Antibody testing will be performed on a separate study (IRB17-01101).
  • Diagnosis of (or ongoing treatment for) an autoimmune disease
Sex/Gender
Sexes Eligible for Study: All
Ages 3 Years to 15 Years   (Child)
Accepts Healthy Volunteers No
Contacts
Contact: Sikder Hassan 614-355-2602 Sikder.Hassan@nationwidechildrens.org
Contact: Amanda Nicholl, RN 614-355-2765 Amanda.Nicholl@nationwidechildrens.org
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03492346
Other Study ID Numbers IRB18-00224
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Jerry R. Mendell, Nationwide Children's Hospital
Study Sponsor Nationwide Children's Hospital
Collaborators Myonexus Therapeutics
Investigators
Principal Investigator: Jerry R Mendell, M.D. Nationwide Children's Hospital
PRS Account Nationwide Children's Hospital
Verification Date December 2018