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

NCT01325740 | Suspended Phase 2 DMC

• 1 other identifier: 107FX201
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Fragile X Syndrome

Outcome Measures

Primary Outcomes (1)

• Adverse events will be monitored for tolerability

  physical exam, laboratories, collection of adverse events

  1 day

Secondary Outcomes (1)

• Blood levels of STX107 will be measured to define pharmacokinetics

  1 day

Study Arms (3)

STX107 10 mg

ACTIVE COMPARATOR

Drug: STX107

Placebo

PLACEBO COMPARATOR

Drug: STX107

STX107 30 mg

ACTIVE COMPARATOR

Drug: STX107

Interventions

STX107 DRUG

STX107 10 mg single dose po capsule, STX107 30 mg single dose po capsule, Placebo single po capsule

Placebo; STX107 10 mg; STX107 30 mg

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Seaside Therapeutics, Inc.: lead

Study Sites (5)

University of California-Los Angeles Neuropsychiatric Institute

Los Angeles, California, 90024, United States

Location

University of California-Davis, M.I.N.D. Institute

Sacramento, California, 95817, United States

Location

University of Colorado Denver, Children's Hospital

Aurora, Colorado, 80045, United States

Location

Emory University School of Medicine

Decatur, Georgia, 30033, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

MeSH Terms

Conditions

Fragile X Syndrome

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 28, 2011
• First Posted: March 30, 2011
• Study Start: May 1, 2011
• Primary Completion: September 1, 2013
• Study Completion: September 1, 2013
• Last Updated: December 20, 2012

Record last verified: 2012-12

Locations

US (5)