Neurophysiological and Acute Pharmacological Studies in FXS Patients
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|ClinicalTrials.gov Identifier: NCT02998151|
Recruitment Status : Completed
First Posted : December 20, 2016
Results First Posted : November 24, 2021
Last Update Posted : November 26, 2021
|Condition or disease||Intervention/treatment||Phase|
|Fragile X Syndrome||Drug: Acamprosate Drug: Lovastatin Drug: Minocycline Drug: Placebo Drug: Baclofen||Early Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||29 participants|
|Intervention Model:||Crossover Assignment|
|Intervention Model Description:||This study was designed as a 4-intervention crossover, with all study participants receiving all possible interventions. These were originally placebo, acamprosate, minocycline, and lovastatin. Midway through the study (n=16) it was determined that acamprosate was undetectable in serum and this intervention was replaced by baclofen. Remaining participants (n=13) received baclofen and 5 participants were re-enrolled to receive baclofen or a second round of placebo, so investigators and participants would remain blinded to drug status during the baclofen visit. The second round of placebo was not analyzed.|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Primary Purpose:||Basic Science|
|Official Title:||Neurophysiological and Acute Pharmacological Studies in FXS Patients|
|Study Start Date :||January 2016|
|Actual Primary Completion Date :||November 2020|
|Actual Study Completion Date :||November 2020|
Experimental: All Study Participants
Participants received, in random order, a single dose of placebo, acamprosate, lovastatin, minocycline, or baclofen, with a two-week washout period between doses. Midway through the study (n=16) it was determined that acamprosate was undetectable in serum and this intervention was replaced by baclofen. Remaining participants (n=13) received baclofen and 5 participants were re-enrolled to receive baclofen or a second round of placebo, so investigators and participants would remain blinded to drug status during the baclofen visit. The second round of placebo was not analyzed.
two 666mg pills
two 20mg pills
two 135mg pills
one 30mg pill
- Change in EEG Relative Gamma Power [ Time Frame: Pre-dose, 4-hour post-dose ]EEG relative gamma power at rest was calculated as the percent of power in the gamma frequencies relative to the sum of power in all frequency bands, averaged across electrodes, and calculated separately at pre-dose and post-dose timepoints. To assess the impact of drug, the pre-dose relative gamma power was subtracted from post-dose relative gamma power. Higher numbers indicate more relative gamma power post-dose; lower numbers indicate more relative gamma power pre-dose.
- Clinical Global Impressions-Improvement [ Time Frame: 4-hour post-dose ]The Clinical Global Impressions - Improvement (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).
- Woodcock Johnson Test of Cognitive Abilities - Auditory Attention Task [ Time Frame: 4-hour post-dose ]Woodcock Johnson Test of Cognitive Abilities III Auditory Attention subscale. Participants must identify orally presented words amid increasingly intense background noise. The scores for this subtask range from 0-50, with higher scores indicating a better outcome. Raw scores for this subscale are reported (rather than standard scores, or age- or grade-equivalents).
- Change From Pre-dose in the Repeatable Battery for the Assessment of Neuropsychological Status at 4 Hours Post Dose [ Time Frame: Pre-dose, 4-hour post dose ]Four 10-item lists of unrelated words were presented orally to the examinee who was then required to immediately recall words presented, at both pre-dose and post-dose timepoints. The impact of drug was assessed by subtracting the number of words remembered post-dose from the number of words remembered pre-dose. Lower numbers indicate more words remembered post-dose; higher numbers indicate more words remembered pre-dose.
- Test of Attentional Performance for Children (KiTAP) Test of Alertness [ Time Frame: Predose, 4-hour post-dose ]Computerized task where an examinee is required to push a key when a target stimulus is presented on the screen. Scores are presented as change in median reaction time (RT), in milliseconds.
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
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|Principal Investigator:||Craig A Erickson, M.D.||Children's Hospital Medical Center, Cincinnati|