Fragile X SyndromePhase 1NCT01911455
Study of Acamprosate in Fragile x Syndrome
A Phase 1 study testing acamprosate in people with Fragile X Syndrome, run by Children's Hospital Medical Center, Cincinnati at 2 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 5 to 23 yearsHealthy volunteers can take part
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- acamprosate
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 46 participants
- Where
- 2 sitesUnited States
- Study dates
- 27 Aug 2013to 8 Nov 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 23 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Rush University Medical Center
Chicago, Illinois, 60612
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With Fragile X Syndrome
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).
Read the detailed description
Each subject with FXS will receive 10 weeks of blinded treatment with acamprosate or matching placebo. After completion of the double-blind phase, all subjects will have an opportunity to receive acamprosate as part of the study procedures for 16 weeks.
- Conditions
- Fragile X Syndrome
- Being tested
- acamprosate, Placebo
- Keywords
- Fragile X Syndrome
- Medical subject headings
- Fragile X Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
acamprosateDrug
Also called Campral
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: acamprosate |
| Placebo | Placebo comparator | Placebo will be prescribed with the same frequency and duration as the acamprosate group. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Change from baseline to week 10
Secondary outcomes (3)
Clinical Global Impressions- Improvement (CGI-I)
The CGI-I global improvement item is a 7-point Likert scale designed to measure symptomatic change at a specific time as compared to baseline. CGI-I is a gold standard global measure of potential change with treatment in placebo-controlled pharmacotherapy trials in developmental disabilities.
Measured: week 10
Aberrant Behavior Checklist- Hyperactivity (ABC-H)
The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
Measured: Change from baseline to week 10
Aberrant Behavior Checklist-Social Avoidance (ABC-SA)
The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
Measured: Change from baseline to week 10
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2013 (estimated)
- Study start27 Aug 2013
- Primary completion8 Nov 2018
- Study completion8 Nov 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fragile X SyndromeStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Rush University Medical Center
- Responsible party
- Sponsor
- Investigators
- Craig Erickson, MD, Principal investigator, Children's Hospital Medical Center, CincinnatiElizabeth Berry-Kravis, MD, Principal investigator, Rush University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01911455
- Sponsor's protocol ID
- CIN001- Acamprosate in FX
- Status last verified
- Mar 2020
- First submitted
- 26 Jul 2013
- Last updated
- 3 Feb 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With Fragile X Syndrome
- ClinicalTrials.gov ID
- NCT01911455
- Acronym / sponsor ID
- CIN001- Acamprosate in FX
- Conditions
- Fragile X Syndrome
- Keywords
- Fragile X Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Rush University Medical Center
- Study start
- 27 Aug 2013 (actual)
- Primary completion
- 8 Nov 2018 (actual)
- Study completion
- 8 Nov 2018 (actual)
- First posted
- 30 Jul 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
Each subject with FXS will receive 10 weeks of blinded treatment with acamprosate or matching placebo. After completion of the double-blind phase, all subjects will have an opportunity to receive acamprosate as part of the study procedures for 16 weeks.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| acamprosate | Active comparator | Drug: acamprosateThe 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. |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo will be prescribed with the same frequency and duration as the acamprosate group. |
Who may qualify
- Ages
- 5 to 23 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| acamprosateAlso: Campral | Drug | acamprosate |
| Placebo | Drug | Placebo |
Primary outcome
Aberrant Behavior Checklist-Social Withdrawal subscale
Time frame: Change from baseline to week 10
The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
3 secondary outcomes
Clinical Global Impressions- Improvement (CGI-I)
Time frame: week 10
Aberrant Behavior Checklist- Hyperactivity (ABC-H)
Time frame: Change from baseline to week 10
Aberrant Behavior Checklist-Social Avoidance (ABC-SA)
Time frame: Change from baseline to week 10
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Feb 2021.
Related trials for Fragile X Syndrome
Not yet recruitingPhase 2
Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome
Aggressive Behavior in Fragile X Syndome
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Not yet recruiting
Single Session Support for Parents of Children With Neurodevelopmental Disabilities Struggling With Anxious Avoidance
Neurodevelopmental Disorders, Autism Spectrum Disorder (ASD)
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Not yet recruitingPhase 2/3
A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Craig Erickson, MD principal investigator
Children's Hospital Medical Center, Cincinnati
Elizabeth Berry-Kravis, MD principal investigator
Rush University Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing acamprosate in people with Fragile X Syndrome, run by Children's Hospital Medical Center, Cincinnati at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Aberrant Behavior Checklist-Social Withdrawal subscale (time frame: Change from baseline to week 10). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Feb 2021.