Angelman Syndrome
Conditions
Keywords
Angelman Syndrome, EFFICACY, SAFETY, MINOCYCLINE, Pediatrics population, Rare diseases
Brief summary
RANDOMIZED CLINICAL TRIAL, PLACEBO COMPARED TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY)
Detailed description
STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME
Interventions
DRUGMINOCYCLINE
Pill Minocycline 50 mg capsule
Pill manufactured to mimic Minocycline 50 mg capsule
Sponsors
Puerta de Hierro University Hospital
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to 30 Years
Healthy volunteers
No
Inclusion criteria
* Male or female between 6 and 30 years old. * Clinical diagnosis of Angelman Syndrome and molecular confirmation of diagnosis. * The participant has an acceptable guardian can give consent on behalf of the participant.
Exclusion criteria
* Patients with hypersensitivity to tetracyclines. * Patients with impaired hepatic or renal function and in those with mainly drug allergy history. * Any other condition that in the opinion of the investigator is considered clinically relevant and that administration of minocycline contraindicated
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Increased on the equivalent age of development | 8, 16 and 24 weeks | Increased on the equivalent age of development, obtained through Development Scale R Merrill-Palmer (MP-R) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior | 8, 16 and 24 weeks | Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior obtained through Development Scale R Merrill-Palmer (MP-R) |
| Improvement of EEG. | 8, 16 and 24 weeks | Improvement of EEG. Measure based on changes in the background activity, type, number and duration of crises, widespread tendency to crises, paroxysmal abnormalities recorded types and the overall evaluation of clinical neurophysiologist |
| Safety and tolerability | 8, 16 and 24 weeks | a) Physical Examination b) Vital signs c) Laboratory Tests d) Adverse effects (AEs) list for treatment, laboratory values, values outside the reference range and descriptive statistics. |
| Clinical Global impression (CGI) | 8, 16 and 24 weeks | Improvement of CGI. . Measure based on changes in the Clinical Global Impression through the perception of parents or guardians, you neurologists and therapist |
Countries
Spain
Outcome results
None listed