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The Vigilan CTD observational study is being conducted to learn more about the rare x-linked neurodevelopmental disease: Creatine Transporter Deficiency (CTD)

Lumos Pharma is sponsoring a multi-year observational clinical study of males with Creatine Transporter Deficiency (CTD) that is currently recruiting individuals to participate. It is called the Vigilan CTD Observational Study.

ClinicalTrials.gov Listing:

https://clinicaltrials.gov/show/NCT02931682

The Vigilan CTD study is a multiple site, clinical study that intends to enroll 50 males with CTD.  All 10 sites in North America are enrolling patients. For a listing of the 10 sites, please scroll to the bottom of this page.

Here are some questions and answers to help participants understand more about the Vigilan study.

What is an observational study?

An observational study involves following and collecting information on people with a particular disease or health condition over a period of time.  At specified times, researchers record information about study participants based on what they see and hear and what they learn from tests.  In this way, researchers can learn more about the basic biology of a disease. An observational study also helps researchers understand how disease symptoms and behaviors change over time.

Observational studies are a very important step toward finding effective disease treatments.  Study findings provide a solid foundation for the design of clinically meaningful trials where a treatment is proposed.

Why participate in the Vigilan study?

Creatine Transporter Deficiency (CTD) was first identified in 2001. The condition is rare so it hasn’t received as much attention or investigation as other more common health problems.

To date there have been few published studies where researchers have looked at how the disease develops over time without any treatment. This is one of the challenges of finding an effective medication for CTD—a challenge shared by researchers working to find cures for many rare diseases. Without information about how a disease progresses, researchers cannot know how to test a new drug to find out if it actually leads to improvements in people’s functioning.

The Vigilan study will give researchers a better understanding of the clinical signs and symptoms of CTD, including effects on behavior and intellectual and physical development. By participating, you and your child will do the following:

  • Receive important and detailed health information about your child’s CTD from comprehensive work-ups and tests that are conducted in university-associated clinical settings with medical experts
  • Speed up efforts to find an effective treatment for CTD
  • Become even a stronger patient advocate for your child and join the CTD scientific and patient family

What exactly will this study involve?

Males signed up to participate will undergo a clinical evaluation at a university clinic to determine if they are eligible to participate in the study.   If eligible, and once enrolled in the study, participants will undergo a series of tests of physical and intellectual abilities. Follow-up testing will occur at the test site every 3 months. In addition, parents or caregivers will receive a phone call every 2 months so researchers can keep track of any changes you may notice in your child’s health and behavior.

The study does not involve any experimental treatments for CTD.

Who is eligible to participate?

  • Males diagnosed with CTD by a healthcare professional based on genetic tests showing a mutation in the SLC6A8 gene

Who cannot participate in the Vigilan study?

  • Males with a history of status epilepticus in the 3 months before they begin the study
  • Children or parents/caregivers who for other reasons are unable to carry out procedures the study involves

I’d like to sign up my son/CTD loved one but we probably live too far away from the test sites.

Travel reimbursement is available for study participants and some family members.

There are 10 locations that are geographically distributed throughout the United States and there is one site also in Canada.

Be part of the solution in understanding more about CTD !

Vigilan Study Locations- 10 sites open and recruiting

North America- 10 sites now recruiting

  • United States, Pennsylvania
    • Childrens Hospital of Philadelphia,
      Philadelphia, Pennsylvania United States, 19104

      • Investigator: Judith Miller, PhD
      • Study Coordinator: Rebecca Podell
  • United States, Maryland
    • National Institutes of Health Clinical Center
      Bethesda, Maryland, United States, 20814

      • Investigator: Simona Bianconi, MD
  • United States, North Carolina
    • Duke University Medical Center
      Durham, North Carolina ,United States, 27710

      • Investigator: Dwight Koeberl, MD,PhD
      • Study Coordinator: Crista Walters
  • United States, Illinois
    • Rush University Medical Center
      Chicago, Illinois, United States, 60612

      • Investigator: Elizabeth Berry-Kravis, MD, PhD
      • Study Coordinator: Mariana Hernandez
  • United States, San Diego
    • University of California, San Diego,
      La Jolla, California, United States 92037

      • Investigator: Bruce Barshop, MD, PhD
      • Study Coordinator: David Ashley
  • United States, Boston
    • Boston Children's Hospital,
      Boston, Massachusetts, United States 02115

      • Investigator: Irina Anselm, MD
      • Study Coordinator: Grace Bezin
  • United States, Houston
    • Texas Children's Hospital
      Houston, Texas, United States 77030

      • Investigator: V. Reid Sutton, MD
      • Study Coordinator: Beverly Feldman
  • United States, Cincinnati
    • Cincinnati Children's Children's Hospital
      Cincinnati, Ohio, United States 45229

      • Investigator: Kim Cecil, PhD
      • Study Coordinator: Jacqueline Gill
  • United States, Salt Lake City
    • University of Utah
      Salt Lake City, Utah, United States 84108

      • Investigator: Nicola Longo, MD, PhD
      • Study Coordinator: Carrie Bailey
  • Canada, Toronto
    • The Hospital for Sick Children
      Toronto, Ontario, Canada M5G 1X8

      • Investigator: Saadet Andrews, MD, PhD
      • Study Coordinator: Julie Choi