A Study to Assess the Tolerability of a Single Dose of STX107 in Adults With Fragile X Syndrome

A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of a Single Dose of STX107 in Adults With Fragile X Syndrome

The study will consist of a Screening period (up to 14 days), a Treatment period, and a Follow-Up period. Sixteen subjects will be enrolled into two sequential dose cohorts - 10 or 30 mg (or matching placebo) across four study centers.

Study Overview

• Status: Suspended
• Conditions: Fragile X Syndrome
• Intervention / Treatment: Drug: STX107

• Study Type: Interventional
• Enrollment (Anticipated): 16
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90024
      • University of California-Los Angeles Neuropsychiatric Institute
    • Sacramento, California, United States, 95817
      • University of California-Davis, M.I.N.D. Institute
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado Denver, Children's Hospital
  • Georgia
    • Decatur, Georgia, United States, 30033
      • Emory University School of Medicine
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 50 years (ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

Inclusion Criteria:

• Male subjects 18 to 50 years of age, inclusive.
• Molecular documentation of the full fragile X mutation.

Exclusion Criteria:

• Subjects with a history of seizure disorder who are, in the opinion of the Investigator and Medical Monitor, not currently considered to be well controlled.
• Subjects currently being treated with psychoactive medications (including stimulants and anxiolytics).
• Subjects with any condition, including alcohol and drug abuse, which might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. 4. Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
• Subjects who, in the Investigator's opinion, might not be suitable for the study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
PLACEBO_COMPARATOR: Placebo	Drug: STX107; STX107 10 mg single dose po capsule, STX107 30 mg single dose po capsule, Placebo single po capsule
ACTIVE_COMPARATOR: STX107 10 mg	Drug: STX107; STX107 10 mg single dose po capsule, STX107 30 mg single dose po capsule, Placebo single po capsule
ACTIVE_COMPARATOR: STX107 30 mg	Drug: STX107; STX107 10 mg single dose po capsule, STX107 30 mg single dose po capsule, Placebo single po capsule

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse events will be monitored for tolerability	physical exam, laboratories, collection of adverse events	1 day

Secondary Outcome Measures

Outcome Measure	Time Frame
Blood levels of STX107 will be measured to define pharmacokinetics	1 day

Collaborators and Investigators

• Sponsor: Seaside Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start: May 1, 2011
• Primary Completion (ANTICIPATED): September 1, 2013
• Study Completion (ANTICIPATED): September 1, 2013

Study Registration Dates

• First Submitted: March 28, 2011
• First Submitted That Met QC Criteria: March 28, 2011
• First Posted (ESTIMATE): March 30, 2011

Study Record Updates

• Last Update Posted (ESTIMATE): December 20, 2012
• Last Update Submitted That Met QC Criteria: December 19, 2012
• Last Verified: December 1, 2012

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Chromosome Disorders; Sex Chromosome Disorders; Syndrome; Fragile X Syndrome
• Other Study ID Numbers: 107FX201