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Published on: October 20, 2023
An open-label pilot trial of minocycline in children as a treatment for Angelman syndrome
Joseph C Grieco1, Stephanie L Ciarlone2, Maria Gieron-Korthals3,4
1Department of Molecular Pharmacology and Physiology, University of South Florida, Morsani College of Medicine, 12901 Bruce B Downs Boulevard, Tampa, FL, 33612, USA. jgrieco1@health.usf.edu.
Background:
Minocycline, a member of the tetracycline family, has a low risk of adverse effects and an ability to improve behavioral performance in humans with cognitive disruption. We performed a single-arm open-label trial in which 25 children diagnosed with Angelman syndrome (AS) were administered minocycline to assess the safety and tolerability of minocycline in this patient population and determine the drug's effect on the cognitive and behavioral manifestations of the disorder.
Methods:
Participants, age 4-12 years old, were randomly selected from a pool of previously screened children for participation in this study. Each child received 3 milligrams of minocycline per kilogram of body weight per day for 8 weeks. Participants were assessed during 3 study visits: baseline, after 8-weeks of minocycline treatment and after an 8-week wash out period. The primary outcome measure was the Bayley Scales of Infant and Toddler Development 3rd Edition (BSID-III). Secondary outcome measures included the Clinical Global Impressions Scale (CGI), Vineland Adaptive Behavior Scales 2nd Edition (VABS-II), Preschool Language Scale 4th Edition (PLS-IV) and EEG scores. Observations were considered statistically significant if p < 0.05 using ANOVA and partial eta squared (η(2)) was calculated to show effect size. Multiple comparisons testing between time points were carried out using Dunnett's post hoc testing.
Results:
Significant improvement in the mean raw scores of the BSID-III subdomains communication and fine motor ability as well as the subdomains auditory comprehension and total language ability of the PLS-IV when baseline scores were compared to scores after the washout period. Further, improvements were observed in the receptive communication subdomain of the VABS-II after treatment with minocycline. Finally, mean scores of the BSID-III self-direction subdomain and CGI scale score were significantly improved both after minocycline treatment and after the wash out period.
Conclusion:
The clinical and neuropsychological measures suggest minocycline was well tolerated and causes improvements in the adaptive behaviors of this sample of children with Angelman syndrome. While the optimal dosage and the effects of long-term use still need to be determined, these findings suggest further investigation into the effect minocycline has on patients with Angelman syndrome is warranted.
Trial Registration:
NCT01531582 - clinicaltrials.gov.
Insights
Minocycline treatment improved communication and motor skills in children with Angelman syndrome (AS). This study found the drug safe and beneficial for adaptive behaviors, warranting further investigation.
Area of Science:
- Neuroscience
- Pediatrics
- Pharmacology
Background:
- Angelman syndrome (AS) is a genetic disorder affecting development.
- Minocycline, a tetracycline antibiotic, has shown potential in improving cognitive function.
- Previous research indicates minocycline's safety and efficacy in managing cognitive disruptions.
Purpose of the Study:
- To assess the safety and tolerability of minocycline in children with AS.
- To evaluate minocycline's impact on cognitive and behavioral aspects of Angelman syndrome.
- To determine the efficacy of minocycline in improving adaptive behaviors in pediatric AS patients.
Main Methods:
- A single-arm, open-label trial involving 25 children diagnosed with AS (ages 4-12).
- Minocycline administered at 3 mg/kg/day for 8 weeks, followed by an 8-week washout period.
- Primary outcome: Bayley Scales of Infant and Toddler Development (BSID-III); Secondary outcomes: CGI, VABS-II, PLS-IV, and EEG scores.
Main Results:
- Significant improvements observed in BSID-III communication and fine motor subdomains.
- Enhancements noted in PLS-IV auditory comprehension and total language ability.
- VABS-II receptive communication and BSID-III self-direction subdomains showed improvement, as did CGI scores.
Conclusions:
- Minocycline was well-tolerated in children with Angelman syndrome.
- The study suggests minocycline positively impacts adaptive behaviors in this population.
- Further research is warranted to determine optimal dosage and long-term effects of minocycline in AS patients.
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