RECRUITING

Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)

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Conditions

Fragile X SyndromeFragile XFXS

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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 a single dose, placebo-controlled study. Male subjects aged 18 to 40 years (inclusive) with a diagnosis of FXS. Eligible subjects may enroll in this study comprised of two in home and two in clinic visits each 14 days apart, for a total of four visits. Subjects will be given single dose gaboxadol (10 mg) or matched placebo at each of these visits to take orally. Thus, all enrolled subjects will receive placebo at home and in clinic and receive gaboxadol at home and in clinic in a blinded fashion.

Official Title

Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)

Quick Facts

Study Start:2024-01-29
Study Completion:2024-09-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06334419

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 40 Years
Sexes Eligible for Study:MALE
Accepts Healthy Volunteers:No
Standard Ages:ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Subject consents to participate, or if they are not their own legal guardian, offers assent supported by legally authorized representative consent
  2. 2. Subject is willing and able to comply with the study procedures as specified in the protocol and to comply with the study drug administration. Caregiver also commits to the study requirements prior to any study-related procedures
  3. 3. Subject and caregiver are both able to understand the spoken national language clearly and caregiver can read and write to complete study assessments
  4. 4. Males age 18 to 40 years (inclusive)
  5. 5. Has FXS with molecular genetic confirmation of the full FMR1 mutation (\>200 cysteine-guanine-guanine \[CGG\] repeats). May have been confirmed historically or at Screening
  6. 6. Is in general good health as deemed by the Investigator, determined by physical examination, medical history, and laboratory tests
  7. 7. If receiving serotonin-selective reuptake inhibitor (SSRI), serotonin-norepinephrine reuptake inhibitor (SNRI), or serotonin antagonist and reuptake inhibitor (SARI), is on a stable, well-tolerated dose for the previous 3 months with no further changes anticipated
  8. 8. Is not sexually active or can confirm at least one form of contraceptive
  1. 1. Any chronic major medical comorbid condition deemed by the Investigator as presenting added risk to the subject, including but not limited to, refractory hypertension, kidney disease, or liver disease
  2. 2. Diagnosed with diabetes (Type 1 or II) or receiving any anti-diabetic medication
  3. 3. Unstable seizure disorder, defined by any seizure within 6 months prior to baseline visit and/or a change in any anti-convulsant drug dosing in the 60 days prior to study consent
  4. 4. Changes in psychotropic or anti-convulsant (where taken for reasons other than seizure control) drug treatment within 30 days prior to Screening
  5. 5. Significant changes in any educational, behavioral, and/or dietary interventions the month prior to Screening
  6. 6. Planned initiation of new, or modification of ongoing, interventions during the study
  7. 7. Unable or unwilling to take oral medication (whole capsule, despite assistance with a spoonful of applesauce, yoghurt, or equivalent liquid food)
  8. 8. Consumption of liver enzyme inducers or inhibitors including and not limited to foods, medicines, herbal remedies and supplements three days prior to any Visit. Foods or beverages containing CYP3A4/5 inhibitors (e.g., grapefruit, pomegranate, pomelo, and star fruit) should be avoided before taking study medication and for up to 1 hour post dose throughout the study
  9. 9. Has abnormal baseline laboratory assessments including, but not limited to, alanine aminotransferase (ALT) or aspartate aminotransferase (AST) or total bilirubin \>1.5 × the upper limit of normal (ULN), serum creatinine \>1.5 x ULN or other clinically relevant laboratory abnormality
  10. 10. Has a clinically significant heart rate or blood pressure (BP) at Screening as judged by the Investigator
  11. 11. Has received an investigational drug in any prior clinical study within 30 days or 5 half-lives (whichever is longer) prior to Screening

Contacts and Locations

Study Contact

Ashley Dapore
CONTACT
513-517-1580
ashley.dapore@cchmc.org
Hannah J Sachs, MPA
CONTACT
513-636-2592
hannah.sachs@cchmc.org

Principal Investigator

Cragi A Erickson, MD
PRINCIPAL_INVESTIGATOR
Children's Hospital Medical Center, Cincinnati

Study Locations (Sites)

Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States

Collaborators and Investigators

Sponsor: Craig Erickson

  • Cragi A Erickson, MD, PRINCIPAL_INVESTIGATOR, Children's Hospital Medical Center, Cincinnati

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 Date2024-01-29
Study Completion Date2024-09-30

Study Record Updates

Study Start Date2024-01-29
Study Completion Date2024-09-30

Terms related to this study

Keywords Provided by Researchers

  • Fragile X
  • Fragile X Syndrome
  • FXS

Additional Relevant MeSH Terms

  • Fragile X Syndrome