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Minocycline in the Treatment of Angelman Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. 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
Information provided by (Responsible Party):
Edwin Weeber, Ph.D., University of South Florida

Brief Summary:
There is mounting evidence to suggest that a treatment for Angelman syndrome is not just possible, but probable. The lack of known molecular targets associated with AS has hampered the development of specific therapeutics. However, a recent surge of potential therapeutics for other disorders associated with cognitive disruption has begun to be used in human clinical trials. The molecular modes of action for many of these new therapeutic agents have correlates to counter the molecular defects observed in AS. One such agent is minocycline (MC), a drug traditionally used as an antibiotic. This compound administered to a mouse model of AS showed a significant decrease in motor deficit and an increase in long term potentiation. The investigators believe a similar result will be observed when minocycline is administered to the AS patient and may lead to the development of an effective AS therapeutic.

Condition or disease Intervention/treatment Phase
Angelman Syndrome Drug: minocycline Not Applicable

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Arm Intervention/treatment
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.
Drug: minocycline
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.
Other Names:
  • Alti-Minocycline
  • Apo-Minocycline
  • Arestin
  • Dynacin
  • Gen-Minocycline
  • Klinomycin
  • Minociclina [INN-Spanish]
  • Minocin
  • Minocyclin
  • Minocycline HCl
  • Minocyclinum [INN-Latin]
  • Minocyn
  • Minomycin
  • Novo-Minocycline
  • Solodyn
  • Vectrin
  • Tetracycline

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. The participant is between the ages of 4 to 12 years old.
  2. The participant has been previously diagnosed with AS by clinical evaluation.
  3. The participant's diagnosis has molecular confirmation (e.g. karyotyping, fluorescent in situ hybridization (FISH), DNA methylation test or sequencing of the ubiquitin-protein ligase E3A gene) of the diagnosis.
  4. The participant has a CGI-Severity Score of at least 4 indicating a moderate level of behavioral difficulty.
  5. The participant is male or female.
  6. The participant has an acceptable surrogate capable of giving consent on the participant's behalf.

Exclusion Criteria:

  1. The participant was diagnosed with AS with no identifiable molecular abnormality.
  2. The participant has a known allergy to MC or tetracycline.
  3. The participant is currently enrolled in a study in which a drug, vitamin or dietary manipulation is used in the treatment of AS.
  4. The participant suffers from severe or uncontrolled seizures or any other medical condition rendering the patient unstable.
  5. The participant suffers from cardiovascular, respiratory, liver, kidney or hematologic disease.
  6. The participant suffers from liver disease or elevated liver function tests.
  7. The participant has a history of neutropenia, anemia or thrombocytopenia.
  8. The participant has a history of systemic lupus erythematosus or an anti-nuclear antibody (ANA) titer or >1:40.
  9. The participant is pregnant or at risk of becoming pregnant (sexually active females).
  10. The participant experiences persistent psychotic symptoms.
  11. The participant (or a parent/caregiver) is not willing to participate in clinic visits.
  12. The participant experiences severe symptoms judged to likely to endanger the participant's safety or the safety of others.

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 identifier (NCT number): NCT01531582

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United States, Florida
Univeristy of South Florida
Tampa, Florida, United States, 33613
Sponsors and Collaborators
University of South Florida
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Principal Investigator: Edwin J Weeber, Ph.D. University of South Florida
Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Edwin Weeber, Ph.D., Professor, University of South Florida Identifier: NCT01531582    
Other Study ID Numbers: WEEBER001
First Posted: February 13, 2012    Key Record Dates
Last Update Posted: May 12, 2014
Last Verified: May 2014
Keywords provided by Edwin Weeber, Ph.D., University of South Florida:
Additional relevant MeSH terms:
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Angelman Syndrome
Pathologic Processes
Movement Disorders
Central Nervous System Diseases
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Anti-Bacterial Agents
Anti-Infective Agents
Protein Synthesis Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action