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Trial record 3 of 8 for:    angelman syndrome
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Minocycline in the Treatment of Angelman Syndrome

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Edwin Weeber, Ph.D., University of South Florida
ClinicalTrials.gov Identifier:
NCT01531582
First received: February 5, 2012
Last updated: June 11, 2012
Last verified: June 2012
History of Changes
  Purpose

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 Intervention
Angelman Syndrome
 Drug: minocycline

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: The Efficacy of Minocycline in the Treatment of Angelman Syndrome

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by University of South Florida:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
    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:
  • Normalization of the EEG (electroencephalogram) signature [ Time Frame: Baseline, 8 and 16 weeks ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    The CGI is a brief assessment used by the clinician to describe the participants condition before and after the administration of a study medication.


Estimated Enrollment: 24
Study Start Date: April 2012
Estimated Study Completion Date: May 2013
Estimated Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   4 Years to 12 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01531582

Locations
United States, Florida
Univeristy of South Florida
Tampa, Florida, United States, 33613
Sponsors and Collaborators
University of South Florida
Investigators
Principal Investigator: Edwin J Weeber, Ph.D. University of South Florida
  More Information

Additional Information:
If your interested in participating, click this link and submit your contact information.  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: Edwin Weeber, Ph.D., Professor, University of South Florida
ClinicalTrials.gov Identifier: NCT01531582     History of Changes
Other Study ID Numbers: WEEBER001
Study First Received: February 5, 2012
Last Updated: June 11, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by University of South Florida:
Angelman
Minocycline
cognitive
children
tetracycline

Additional relevant MeSH terms:
Angelman Syndrome
Movement Disorders
Central Nervous System Diseases
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Tetracycline
 Minocycline
Protein Synthesis Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Anti-Bacterial Agents
Anti-Infective Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on May 22, 2013