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Single-Dose Acamprosate, Lovastatin, Minocycline and Placebo in Fragile X Syndrome (LAM)

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ClinicalTrials.gov Identifier: NCT02998151
Recruitment Status : Enrolling by invitation
First Posted : December 20, 2016
Last Update Posted : March 4, 2020
Sponsor:
Collaborator:
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

Brief Summary:
The aim of this study is to utilize neurophysiologic assessments, behavioral measures and clinical measures to assess how much deficits associated with Fragile X Syndrome from pre-dose to post-dose Acamprosate, lovastatin, minocycline and placebo.

Condition or disease Intervention/treatment Phase
Fragile X Syndrome Drug: Acamprosate Drug: Lovastatin Drug: Minocycline Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Evaluating the Neurophysiologic and Clinical Effects of Single-Dose Acamprosate, Lovastatin, Minocycline and Placebo in Fragile X Syndrome
Study Start Date : January 2016
Estimated Primary Completion Date : July 2020
Estimated Study Completion Date : July 2021


Arm Intervention/treatment
Placebo Comparator: Placebo
Placebo pill
Drug: Placebo
Experimental: Acamprosate Drug: Acamprosate
two 666 pills

Experimental: Lovastatin Drug: Lovastatin
two 20mg pills

Experimental: Minocycline Drug: Minocycline
two 135mg pills




Primary Outcome Measures :
  1. Change in EEG from baseline [ Time Frame: Pre-dose, 4-hour post-dose ]
    EEG will be used to assess the electrophysiologic aspects of auditory processing

  2. Clinical Global Impressions Improvement [ Time Frame: 4-hour post-dose ]
    The CGI-I requires the clinician to assess how much the patient's illness has changed relative to pre-dose, from 1 (very much improved) to 7 (very much worse).


Secondary Outcome Measures :
  1. Clinical Global Impressions Severity [ Time Frame: Pre-dose ]
    . The CGI-S is a 7-point scale designed to measure global severity of illness over the previous seven days.

  2. Aberrant Behavior Checklist [ Time Frame: Baseline and beginning of each visit to assess previous 2 weeks ]
    The recently validated Aberrant Behavior Checklist (ABC) using the 6-factor FXS model will be used as a secondary outcome of clinical status.

  3. Woodcock Johnson Test of cognitive abilities - auditory attention task [ Time Frame: Baseline visit, 4-hour post-dose ]
    Identify parts needed to form a complete shape and Identify orally presented words amid increasingly intense background noise

  4. Repeatable Battery for the Assessment of Neuropsychological Status [ Time Frame: Pre-dose, 4-hour post dose ]
    A 10-item list of unrelated words is presented orally to the examinee who is then required to immediately recall words presented.

  5. Test of Attentional Performance for Children (KiTAP) [ Time Frame: Predose, 4-hour post-dose ]
    Computerized task comprised of 8 subtests where an examinee is required to push a key when a target stimulus is presented on the screen.

  6. Grooved Pegboard Test [ Time Frame: Baseline visit, 4-hour post-dose ]
    Pegs, with a key along one side, must be rotated to match the holes in the pegboard before they can be inserted.



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Ages Eligible for Study:   15 Years to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subjects ages 15-55, with FXS who completed the study entitled "Mechanisms and brain circuits underlying fragile X syndrome (IRB # 2015-8425). FXS is defined as full FMR1 mutations (>200 CGG repeats) confirmed by genetic testing.
  • General good health as determined by physical exam, medical history and laboratory work up.

Exclusion Criteria:

  • Subjects with a history of intolerance to acamprosate, lovastatin, or minocycline will be excluded.
  • Subjects will also be excluded if they have taken any investigational drug within 3 months, have a history of substance abuse or dependence within 6 months, or significant psychiatric or CNS neurological disease unrelated to FXS.
  • Uncontrolled seizures impact EEG data as do anticonvulsants, barbiturates, lithium and benzodiazepines and are exclusions (within 5 half-lives). Those taking other psychiatric medications must be on stable doses for 4 weeks before any testing.
  • For female subjects of child bearing potential, a positive urine pregnancy test.
  • Potential subjects with a creatinine clearance < 50 mL/min will be excluded.
  • Identified medical issues, inability to tolerate study procedures or study drug per the discretion of the Principal Investigator.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02998151


Locations
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United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Investigators
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Principal Investigator: Craig A Erickson, M.D. Children's Hospital Medical Center, Cincinnati
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Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT02998151    
Other Study ID Numbers: CIN001 - {LAM}
First Posted: December 20, 2016    Key Record Dates
Last Update Posted: March 4, 2020
Last Verified: March 2020
Additional relevant MeSH terms:
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Fragile X Syndrome
Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Minocycline
Acamprosate
Lovastatin
L 647318
Dihydromevinolin
Anti-Bacterial Agents
Anti-Infective Agents
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Enzyme Inhibitors
Alcohol Deterrents