RECRUITING

Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

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Conditions

Spinal Muscular Atrophy

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This is an observational study to investigate the improvement of NMJ defects in adult patients with SMA following treatment with Risdiplam. Eligible patients will have received treatment with daily oral Risdiplam after receiving approval through their commercial insurance or drug assistance program. All subjects will be evaluated at one visit. Eligible subjects must have been receiving risdiplam for at least 12 months.

Official Title

Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

Quick Facts

Study Start:2021-10-13
Study Completion:2024-12-31
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05219487

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 70 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Signed Informed Consent Form
  2. * Age 18-70 years at time of signing Informed Consent Form
  3. * Able and willing to provide written informed consent and to comply with the program protocol according to ICH and local regulations
  4. * Ability to comply with the study protocol, in the investigator's judgment
  5. * Adequately recovered from any acute illness at the time of screening, and considered clinically well enough to participate, in the opinion of the treating physician
  6. * Patients with retinopathy of prematurity should have evidence of stable disease
  7. * Genetic confirmation of 5q SMA documented on standard genetic tests for the disorder
  8. * Treated with risdiplam for a duration of 12 months or longer
  1. * Treatment with an investigational therapy within 180 days prior to initiation of study drug
  2. * History of established diagnosis of neuromuscular junction disorder
  3. * Co-morbid conditions that preclude travel or testing
  4. * Patients who are, in the investigator's opinion, mentally or legally incapacitated to provide an informed consent

Contacts and Locations

Study Contact

Gia Cinkay
CONTACT
614-366-9050
Georgia.Cinkay@osumc.edu
Marco Tellez, CCRP
CONTACT
614-688-7837
Marco.Tellez@osumc.edu

Principal Investigator

Bakri Elsheikh, MBBS
PRINCIPAL_INVESTIGATOR
Ohio State University

Study Locations (Sites)

The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
United States

Collaborators and Investigators

Sponsor: Bakri Elsheikh

  • Bakri Elsheikh, MBBS, PRINCIPAL_INVESTIGATOR, Ohio State University

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-10-13
Study Completion Date2024-12-31

Study Record Updates

Study Start Date2021-10-13
Study Completion Date2024-12-31

Terms related to this study

Additional Relevant MeSH Terms

  • Spinal Muscular Atrophy