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Fragile X syndrome (FXS) is the most common inherited form of developmental disability. FXS is inherited from the carrier parent, most often the mothers. FXS is associated with severe interfering behavioral symptoms which include anxiety related symptoms, attention deficit hyperactivity, and aggressive behaviors. Approximately 25-33% of individuals with FXS also meet criteria for autistic disorder. The hypothesis of this study is that treatment with acamprosate will reduce inattention/hyperactivity, language impairment, irritability, social deficits, and cognitive delay in youth with FXS. The purpose of this study is to investigate the effectiveness and tolerability of acamprosate in youth with Fragile X Syndrome and to assess the potential psychophysiological differences between FXS and autism spectrum disorders.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Acamprosate | Active Comparator | The maximum dose of acamprosate to be used in this study is 1998 mg per day for those subjects weighing greater than 60kg and 1332 mg per day for those less weighing less than 60kg. |
|
| Autism Spectrum Disorder | No Intervention | This baseline comparison group will participated in only the psychophysiological and biomarker portion of subject characterization. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Acamprosate | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Global Impression- Severity Scale (CGI-S) | The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating 1, normal, not at all ill; 2, borderline mentally ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. | Week 10 |
| Measure | Description | Time Frame |
|---|---|---|
| The Aberrant Behavior Checklist (ABC) | The Aberrant Behavior Checklist (ABC) is a 58-item rating scale used to assess maladaptive behaviors across five original subscales: Irritability (15 items from 0-45), Social Withdrawal (16 items from 0-48), Stereotypy (7 items from 0-21), Hyperactivity (16 items from 0-48), Inappropriate Speech (4 items from 0-12). Additionally, Social Avoidance, a newly developed four-item subscale (from 0-12) of the ABC that captures core social avoidance aspects of Fragile X Syndrome is reported. All items on the ABC are rated from 0 (not at all a problem) to 3 (the problem is severe in degree). Higher scores indicate greater maladaptive behaviors. Differences between Baseline and Week 10 are used as an indicator of change. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Craig A. Erickson, M.D. | Indiana University School of Medicine - Department of Psychiatry | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Riley Child and Adolescent Psychiatry Clinic - Riley Hospital for Children | Indianapolis | Indiana | 46202 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23436129 | Background | Erickson CA, Wink LK, Ray B, Early MC, Stiegelmeyer E, Mathieu-Frasier L, Patrick V, Lahiri DK, McDougle CJ. Impact of acamprosate on behavior and brain-derived neurotrophic factor: an open-label study in youth with fragile X syndrome. Psychopharmacology (Berl). 2013 Jul;228(1):75-84. doi: 10.1007/s00213-013-3022-z. Epub 2013 Feb 24. |
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The psychophysiological measures proved non-feasible as subjects could not tolerate the procedures. The paradigms were tried on two subjects in the Autism Spectrum Disorder arm; however, the data that was collected was not usable. Subsequently, neurobiological characterization was halted.
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| ID | Title | Description |
|---|---|---|
| FG000 | Acamprosate | The maximum dose of acamprosate to be used in this study is 1998 mg per day for those subjects weighing greater than 60kg and 1332 mg per day for those less weighing less than 60kg. Acamprosate |
| FG001 | Autism Spectrum Disorder (ADS) | This baseline comparison group will participated in only and biomarker portion of subject characterization. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Acamprosate Treatment Group | Twelve subjects received open-label acamprosate, mean final of 1,054 mg/day (range: 666-1,998 mg/day) |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Clinical Global Impression- Severity Scale (CGI-S) | The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating 1, normal, not at all ill; 2, borderline mentally ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. | Posted | Mean | Standard Deviation | units on a scale | Week 10 |
|
Ten Weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Acamprosate | The maximum dose of acamprosate to be used in this study is 1998 mg per day for those subjects weighing greater than 60kg and 1332 mg per day for those less weighing less than 60kg. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Irritability | Psychiatric disorders |
Psychophysiological measures proved non-feasible as subjects could not tolerate the procedures. The paradigms were tried on 2 subjects in the ADS arm, as data collected was not usable. Subsequently, neurobiological characterization was halted.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Craig Erickson | Cincinnati Childrens Hospital | 5136366265 | craig.erickson@cchmc.org |
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| ID | Term |
|---|---|
| D005600 | Fragile X Syndrome |
| D000067877 | Autism Spectrum Disorder |
| ID | Term |
|---|---|
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
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| ID | Term |
|---|---|
| D000077443 | Acamprosate |
| ID | Term |
|---|---|
| D013654 | Taurine |
| D017738 | Alkanesulfonic Acids |
| D000473 | Alkanes |
| D006839 | Hydrocarbons, Acyclic |
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| Week 10 |
| Social Responsiveness Scale | The 65-item SRS is a standardized measure of the core symptoms of autism. Each item is scored on a 4-point Likert scale. The score of each individual item is summed to create a total raw score. A total scores results are as follows: 0-62: Within normal limits 63-79: Mild range of impairment 80-108: Moderate range of impairment 109-149: Severe range of impairment | Week 10 |
| Children's Yale-Brown Obsessive Compulsive Scale Modified for PDD | The Children's Yale-Brown Obsessive Compulsive Scales-Modified (CY-BOCS) is a 5-item, semi-structured clinician rating scale modified designed to rate the current severity of repetitive behavior in children and adolescents with PDD. Once the current repetitive behaviors are identified, they are separately rated on 5 items: Time Spent, Interference, Distress, Resistance, and Control. Each of these items is scored on a 5-point scale form 0 (least symptomatic) to 4 (most symptomatic). The CY-BOCS yields a Total Score from 0 to 20 and is sensitive to change. | Week 10 |
| ADHD Rating Scale 4th Edition | The ADHD Rating Scale is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder. The ADHD Rating Scale-IV is completed by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. The total score can range from 0 to 54, with a higher score indicating greater severity. | Week 10 |
| Vineland Adaptive Behavior Scales-II (VABS-II) Communication Domain | The VABS-II is a semi-structured interview designed to assess adaptive functioning in communication, daily living, socialization and motor skills. Recognizing that language is a major area of impairment in the study population, the Communication Domain (99 Items from 0-198), in particular the Expressive Subdomain (54 Items from 0-108) are of interest in this study. Items arranged in a developmental sequence are rated on a 3-point scale. Each item is scored from 0 (never performs the behavior) to 3 (usually performs the behavior independently). Higher scores indicate higher adaptive functioning. Differences between Baseline and Week 10 are used as an indicator of change. | Week 10 |
| Peabody Picture Vocabulary | The Peabody Picture Vocabulary Test is one of the most commonly used assessment tests that measure verbal ability in standard American English vocabulary. This test has been nationally standardized using examinees from various age groups, from children to adults. Thus, the raw scores are equated to mental age, using the norms obtained from standardization. The total standard scores range from 40 (worse receptive vocabulary) to 160 (better receptive vocabulary). The scores can also be converted to percentile rank. | Week 10 |
| Brain-derived Neurotrophic Factor (BDNF) | BDNF is a protein that supports the survival of existing neurons and growth and differentiation of new neurons and synapses. | Screen and Week 10 |
| Participants |
|
| Age, Continuous | Mean | Full Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Aberrant Behavior Checklist | Aberrant Behavior Checklist (ABC) is a 58-item rating scale assessing maladaptive behaviors across 5 original subscales: Irritability(15 items from 0-45), Social Withdrawal(16 items from 0-48), Stereotypy(7 items from 0-21), Hyperactivity(16 items from 0-48), Inappropriate Speech(4 items from 0-12). Also reported is Social Avoidance, a newly developed 4-item subscale (from 0-12) that captures core social avoidance aspects of Fragile X Syndrome. Items on the ABC are rated from 0 (not at all a problem) to 3 (the problem is severe in degree). Higher scores indicate greater maladaptive behaviors. | Mean | Standard Deviation | units on a scale |
|
| Clinical Global Impression -Severity | The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating 1, normal, not at all ill; 2, borderline mentally ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. | Mean | Standard Deviation | units on a scale |
|
| Children's Yale-Brown Obsessive Compulsive Scale Modified for PDD | The Children's Yale-Brown Obsessive Compulsive Scales-Modified (CY-BOCS) is a 5-item, semi-structured clinician rating scale modified designed to rate the current severity of repetitive behavior in children and adolescents with PDD. Once the current repetitive behaviors are identified, they are separately rated on 5 items: Time Spent, Interference, Distress, Resistance, and Control. Each of these items is scored on a 5-point scale form 0 (least symptomatic) to 4 (most symptomatic). The CY-BOCS yields a Total Score from 0 to 20 and is sensitive to change. | Mean | Standard Deviation | units on a scale |
|
| Social Responsiveness Scale | The 65-item SRS is a standardized measure of the core symptoms of autism. Each item is scored on a 4-point Likert scale. The score of each individual item is summed to create a total raw score. A total scores results are as follows: 0-62: Within normal limits 63-79: Mild range of impairment 80-108: Moderate range of impairment 109-149: Severe range of impairment | Mean | Standard Deviation | units on a scale |
|
| ADHD Rating Scale 4th Edition | The ADHD Rating Scale is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder. The ADHD Rating Scale-IV is completed by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. The total score can range from 0 to 54, with a higher score indicating greater severity. | Mean | Standard Deviation | units on a scale |
|
| Peabody Picture Vocabulary Test | The Peabody Picture Vocabulary Test is one of the most commonly used assessment tests that measure verbal ability in standard American English vocabulary. This test has been nationally standardized using examinees from various age groups, from children to adults. Thus, the raw scores are equated to mental age, using the norms obtained from standardization. The total standard scores range from 40 (worse receptive vocabulary) to 160 (better receptive vocabulary). The scores can also be converted to percentile rank. | Mean | Standard Deviation | units on a scale |
|
| Vineland Adaptive Behavior Scales-II (VABS-II) Communication Domain | The VABS-II is a semi-structured interview designed to assess adaptive functioning in communication, daily living, socialization and motor skills. Recognizing that language is a major area of impairment in the study population, the Communication Domain (99 Items from 0-198), in particular the Expressive Subdomain (54 Items from 0-108) are of interest to this study. Items arranged in a developmental sequence are rated on a 3-point scale. Each item is rated from 0 (never performs the behavior) to 2 (usually performs behavior independently). Higher scores indicate higher adaptive functioning. | Mean | Standard Deviation | units on a scale |
|
| Brain-derived neurotropic factor | Brain-derived neurotrophic factor (BDNF) is a protein that supports the survival of existing neurons and growth and differentiation of new neurons and synapses. | Mean | Standard Deviation | pg/mL |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Secondary | The Aberrant Behavior Checklist (ABC) | The Aberrant Behavior Checklist (ABC) is a 58-item rating scale used to assess maladaptive behaviors across five original subscales: Irritability (15 items from 0-45), Social Withdrawal (16 items from 0-48), Stereotypy (7 items from 0-21), Hyperactivity (16 items from 0-48), Inappropriate Speech (4 items from 0-12). Additionally, Social Avoidance, a newly developed four-item subscale (from 0-12) of the ABC that captures core social avoidance aspects of Fragile X Syndrome is reported. All items on the ABC are rated from 0 (not at all a problem) to 3 (the problem is severe in degree). Higher scores indicate greater maladaptive behaviors. Differences between Baseline and Week 10 are used as an indicator of change. | Posted | Mean | Standard Deviation | units on a scale | Week 10 |
|
|
|
| Secondary | Social Responsiveness Scale | The 65-item SRS is a standardized measure of the core symptoms of autism. Each item is scored on a 4-point Likert scale. The score of each individual item is summed to create a total raw score. A total scores results are as follows: 0-62: Within normal limits 63-79: Mild range of impairment 80-108: Moderate range of impairment 109-149: Severe range of impairment | Posted | Mean | Standard Deviation | units on a scale | Week 10 |
|
|
|
| Secondary | Children's Yale-Brown Obsessive Compulsive Scale Modified for PDD | The Children's Yale-Brown Obsessive Compulsive Scales-Modified (CY-BOCS) is a 5-item, semi-structured clinician rating scale modified designed to rate the current severity of repetitive behavior in children and adolescents with PDD. Once the current repetitive behaviors are identified, they are separately rated on 5 items: Time Spent, Interference, Distress, Resistance, and Control. Each of these items is scored on a 5-point scale form 0 (least symptomatic) to 4 (most symptomatic). The CY-BOCS yields a Total Score from 0 to 20 and is sensitive to change. | Posted | Mean | Standard Deviation | units on a scale | Week 10 |
|
|
|
| Secondary | ADHD Rating Scale 4th Edition | The ADHD Rating Scale is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder. The ADHD Rating Scale-IV is completed by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. The total score can range from 0 to 54, with a higher score indicating greater severity. | Posted | Mean | Standard Deviation | units on a scale | Week 10 |
|
|
|
| Secondary | Vineland Adaptive Behavior Scales-II (VABS-II) Communication Domain | The VABS-II is a semi-structured interview designed to assess adaptive functioning in communication, daily living, socialization and motor skills. Recognizing that language is a major area of impairment in the study population, the Communication Domain (99 Items from 0-198), in particular the Expressive Subdomain (54 Items from 0-108) are of interest in this study. Items arranged in a developmental sequence are rated on a 3-point scale. Each item is scored from 0 (never performs the behavior) to 3 (usually performs the behavior independently). Higher scores indicate higher adaptive functioning. Differences between Baseline and Week 10 are used as an indicator of change. | Posted | Mean | Standard Deviation | units on a scale | Week 10 |
|
|
|
| Secondary | Peabody Picture Vocabulary | The Peabody Picture Vocabulary Test is one of the most commonly used assessment tests that measure verbal ability in standard American English vocabulary. This test has been nationally standardized using examinees from various age groups, from children to adults. Thus, the raw scores are equated to mental age, using the norms obtained from standardization. The total standard scores range from 40 (worse receptive vocabulary) to 160 (better receptive vocabulary). The scores can also be converted to percentile rank. | Posted | Mean | Standard Deviation | units on a scale | Week 10 |
|
|
|
| Secondary | Brain-derived Neurotrophic Factor (BDNF) | BDNF is a protein that supports the survival of existing neurons and growth and differentiation of new neurons and synapses. | Posted | Mean | Standard Deviation | pg/mL | Screen and Week 10 |
|
|
|
| 0 |
| 12 |
| 9 |
| 12 |
| Increased repetitive behavior | Psychiatric disorders |
|
| Diarrhea | Gastrointestinal disorders |
|
| mild constipation | Gastrointestinal disorders |
|
| Increased anxiety | Psychiatric disorders |
|
| Insomnia | Psychiatric disorders |
|
| Nightmares | Psychiatric disorders |
|
| Rhinitis | Infections and infestations |
|
| Urinary Urgency | Renal and urinary disorders |
|
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| D009422 | Nervous System Diseases |
| D025064 | Sex Chromosome Disorders |
| D025063 | Chromosome Disorders |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D040181 | Genetic Diseases, X-Linked |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D002659 | Child Development Disorders, Pervasive |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
| D006838 |
| Hydrocarbons |
| D009930 | Organic Chemicals |
| D013451 | Sulfonic Acids |
| D013456 | Sulfur Acids |
| D013457 | Sulfur Compounds |
| Title | Measurements |
|---|---|
|
| ABC Hyperactivity |
|
| ABC Inappropriate Speech |
|
| ABC Social Avoidance |
|