Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome
- Conditions
- Angelman Syndrome
- Interventions
- Drug: PLACEBO (for Minocycline)
- Registration Number
- NCT02056665
- Lead Sponsor
- Puerta de Hierro University Hospital
- 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
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 32
- 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.
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description MINOCYCLINE 8 weeks PLACEBO (for Minocycline) Duration of treatment: 8 weeks * Patients \<35 kg. 100 mg / day. Administered at a dose of one capsule of 50 mg breakfast and dinner. * Patients 35 - 50 kg. 150 mg / day. Administered at a dose of 2 capsules of 50 mg breakfast and 1 capsule of 50mg dinner. * Patients \> 50 kg. 200 mg / day. Administered at a dose of two capsules of 50 mg of breakfast and dinner. PLACEBO 8 weeks PLACEBO (for Minocycline) Pill manufactured to mimic Minocycline 50 mg capsule PLACEBO 8 weeks MINOCYCLINE Pill manufactured to mimic Minocycline 50 mg capsule MINOCYCLINE 8 weeks MINOCYCLINE Duration of treatment: 8 weeks * Patients \<35 kg. 100 mg / day. Administered at a dose of one capsule of 50 mg breakfast and dinner. * Patients 35 - 50 kg. 150 mg / day. Administered at a dose of 2 capsules of 50 mg breakfast and 1 capsule of 50mg dinner. * Patients \> 50 kg. 200 mg / day. Administered at a dose of two capsules of 50 mg of breakfast and dinner. MINOCYCLINE 16 weeks MINOCYCLINE Duration of treatment: 16 weeks Patients \<35 kg. 100 mg / day. Administered at a dose of one capsule of 50 mg breakfast and dinner. Patients 35 - 50 kg. 150 mg / day. Administered at a dose of 2 capsules of 50 mg breakfast and 1 capsule of 50mg dinner. Patients \> 50 kg. 200 mg / day. Administered at a dose of two capsules of 50 mg of breakfast and dinner.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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.
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)
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
Trial Locations
- Locations (1)
Puerta de Hierro University Hospital
🇪🇸Madrid, Spain