Minocycline in the Treatment of Angelman Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 24 April 2023
• Main ID: NCT01531582
• Date of registration: 05/02/2012
• Prospective Registration: Yes
• Primary sponsor: University of South Florida
• Public title: Minocycline in the Treatment of Angelman Syndrome
• Scientific title: The Efficacy of Minocycline in the Treatment of Angelman Syndrome
• Date of first enrolment: April 2012
• Target sample size: 25
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01531582
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: Edwin J Weeber, Ph.D.
• Affiliation: University of South Florida

Key inclusion & exclusion 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.



Age minimum: 4 Years
Age maximum: 12 Years
Gender: All

Health Condition(s) or Problem(s) studied

Angelman Syndrome

Intervention(s)

Drug: minocycline

Primary Outcome(s)

Bayley Scales of Infant and Toddler Development, 2nd Edition (BSID-II) Score at Baseline, 8 Weeks After Treatment, and at 16 Week Follow-up [Time Frame: Baseline, 8 weeks & 16 weeks]

Secondary Outcome(s)

Normalization of the EEG (Electroencephalogram) Signature [Time Frame: Baseline, 8 and 16 weeks]

Clinical Global Impressions Severity Scale Score at Baseline, 8 Weeks After Treatment, and at 16 Week Follow-up [Time Frame: Baseline, 8 & 16 weeks]

Preschool Language Scale, Fourth Edition (PLS-4)Score at Baseline, 8 Weeks After Treatment, and at 16 Week Follow-up [Time Frame: Baseline, 8 and 16 weeks]

Vineland Adaptive Behavior Scale, 4th Edition (Vineland-II)Score at Baseline, 8 Weeks After Treatment, and at 16 Week Follow-up [Time Frame: Baseline, 8 and 16 weeks]

Secondary ID(s)

WEEBER001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 18/04/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01531582