Minocycline in the Treatment of Angelman Syndrome
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|ClinicalTrials.gov Identifier: NCT01531582|
Recruitment Status : Unknown
Verified May 2014 by Edwin Weeber, Ph.D., University of South Florida.
Recruitment status was: Active, not recruiting
First Posted : February 13, 2012
Last Update Posted : May 12, 2014
|Condition or disease||Intervention/treatment||Phase|
|Angelman Syndrome||Drug: minocycline||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||The Efficacy of Minocycline in the Treatment of Angelman Syndrome|
|Study Start Date :||April 2012|
|Estimated Primary Completion Date :||December 2014|
|Estimated Study Completion Date :||December 2014|
Experimental: Children with Angelman Syndrome
Children with a molecularly confirmed diagnosis of Angelman Syndrome meeting the protocol requirements will be selected randomly. All participants will receive the study drug, minocycline, over an identical time course. Participants will undergo identical baseline, 8 and 16 week follow up assessments.
The participant's parent or guardian will be instructed to administer minocycline caplets by mouth twice daily. Parents or guardians will be instructed to avoid dairy products, antacids, or any vitamin preparation that contains any divalent or trivalent cations (e.g. Aluminum, Calcium, Magnesium, etc.) for one hour prior to, and two hours after study medication administration.
- A change from baseline in the Bayley Scales of Infant and Toddler Development, 2nd edition (BSID-II)Score [ Time Frame: Baseline, 8 weeks & 16 weeks ]The primary outcome measure consists of improvement in raw and standard scores on the Bayley Scales of Infant and Toddler Development when post Minocycline administration results are compared to baseline results.
- Normalization of the EEG (electroencephalogram) signature [ Time Frame: Baseline, 8 and 16 weeks ]The secondary outcome measures consist of normalization of the EEG signature when comparing post MC administration results to baseline results. Angelman syndrome patients have a characteristic EEG signature and are prone to seizure. It stands to reason then, if the administration of MC decreases the number of seizures, a difference in the EEG signature should be observed as well.
- A change from baseline in the Vineland Adaptive Behavior Scale, 4th edition (Vineland-II)Score [ Time Frame: Baseline, 8 and 16 weeks ]This test is to measure the adaptive behaviors; the ability to adapt to changes in one's environment, learn new everyday skills and level of independence.
- A change from baseline in the Aberrant Behavior Checklist - Community version (ABC - Community)Score [ Time Frame: Baseline, 8 and 16 weeks ]This behavior rating scale utilizes direct observation to measure behavior problems in those with mental retardation. The checklist evaluates irritability, lethargy, stereotypic behavior, hyperactivity, inappropriate speech and provides a raw score for each domain.
- A change from baseline in the Preschool Language Scale, Fourth Edition (PLS-4)Score [ Time Frame: Baseline, 8 and 16 weeks ]This test is used to evaluate the development of expressive and receptive language development. It also can be used to assess behaviors considered to be language precursors.
- A change from baseline in the Clinical Global Impressions Severity Scale Score [ Time Frame: Baseline, 8 & 16 weeks ]The CGI is a brief assessment used by the clinician to describe the participants condition before and after the administration of a study medication.
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): NCT01531582
|United States, Florida|
|Univeristy of South Florida|
|Tampa, Florida, United States, 33613|
|Principal Investigator:||Edwin J Weeber, Ph.D.||University of South Florida|