Study of Acamprosate in Fragile x Syndrome

NCT01911455 | Completed Phase 1 DMC

• 1 other identifier: CIN001- Acamprosate in FX
• Study Type: interventional
• Target: 46
• Locations: 1 country
• Sites: 2

Brief Summary

In this research study we want to understand the effectiveness of a drug treatment, acamprosate, for interfering symptoms (i.e., inattention/hyperactivity, social impairment) associated with Fragile X Syndrome (FXS).

Conditions

Fragile X Syndrome

Keywords

Fragile X Syndrome

Outcome Measures

Primary Outcomes (1)

• Aberrant Behavior Checklist-Social Withdrawal subscale

  The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.

  Change from baseline to week 10

Secondary Outcomes (3)

• Clinical Global Impressions- Improvement (CGI-I)

  week 10

• Aberrant Behavior Checklist- Hyperactivity (ABC-H)

  Change from baseline to week 10

• Aberrant Behavior Checklist-Social Avoidance (ABC-SA)

  Change from baseline to week 10

Study Arms (2)

acamprosate

ACTIVE COMPARATOR

The maximum dose of acamprosate to be used in this study is 666 mg three times daily (total 1998 mg/day) for subjects weight ≥ 50 kg and 1332mg for subjects that weigh \< 50 kg.

Drug: acamprosate

Placebo

PLACEBO COMPARATOR

Placebo will be prescribed with the same frequency and duration as the acamprosate group.

Drug: Placebo

Interventions

acamprosate DRUG

Also known as: Campral

acamprosate

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 5 Years - 23 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnostic confirmation of full mutation FXS
• Age ≥5 years and \<23 years
• General good health as determined by physical exam, medical history and laboratory work up.

You may not qualify if:

• Use of more than two psychotropic medications (medications affecting behavior).
• Unstable dosing of any psychotropic medication (medication affecting behavior)
• Problems with kidney functioning
• Unstable seizure disorder
• Change in any anti-convulsant drug dosing in the 60 days prior to study entry
• Prior adequate treatment trial with acamprosate as determined by the study doctor
• Pregnant or lactating females

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Rush University Medical Center: collaborator

Study Sites (2)

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Fragile X Syndrome

Interventions

Acamprosate

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

Intervention Hierarchy (Ancestors)

Taurine; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Organic Chemicals; Sulfonic Acids; Sulfur Acids; Sulfur Compounds

Study Officials

• Craig Erickson, MD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

• Elizabeth Berry-Kravis, MD

  Rush University Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 26, 2013
• First Posted: July 30, 2013
• Study Start: August 27, 2013
• Primary Completion: November 8, 2018
• Study Completion: November 8, 2018
• Last Updated: February 3, 2021

Record last verified: 2020-03

Locations

US (2)