Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome

Study Overview
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Brief Summary
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RANDOMIZED CLINICAL TRIAL, PLACEBO COMPARED TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY)

Detailed Description
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STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME

Official Title
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Randomized Clinical Trial, Placebo Compared to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome

Conditions
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Angelman Syndrome

Intervention / Treatment
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* Drug: MINOCYCLINE
* Drug: PLACEBO (for Minocycline)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 30 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MINOCYCLINE 8 weeks<br>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. | Drug: MINOCYCLINE<br>* Pill Minocycline 50 mg capsule<br>* Other names: Administration routes: Oral use;Drug: PLACEBO (for Minocycline)<br>* Pill manufactured to mimic Minocycline 50 mg capsule<br>* Other names: Administration routes: Oral use;|
| Placebo Comparator: PLACEBO 8 weeks<br>Pill manufactured to mimic Minocycline 50 mg capsule | Drug: MINOCYCLINE<br>* Pill Minocycline 50 mg capsule<br>* Other names: Administration routes: Oral use;Drug: PLACEBO (for Minocycline)<br>* Pill manufactured to mimic Minocycline 50 mg capsule<br>* Other names: Administration routes: Oral use;|
| Experimental: MINOCYCLINE 16 weeks<br>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. | Drug: MINOCYCLINE<br>* Pill Minocycline 50 mg capsule<br>* Other names: Administration routes: Oral use;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Increased on the equivalent age of development | Increased on the equivalent age of development, obtained through Development Scale R Merrill-Palmer (MP-R) | 8, 16 and 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior | Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior obtained through Development Scale R Merrill-Palmer (MP-R) | 8, 16 and 24 weeks | 
| Improvement of EEG. | 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 | 8, 16 and 24 weeks | 
| Safety and tolerability | 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. | 8, 16 and 24 weeks | 
| Clinical Global impression (CGI) | Improvement of CGI. . Measure based on changes in the Clinical Global Impression through the perception of parents or guardians, you neurologists and therapist | 8, 16 and 24 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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SAFETY, Angelman Syndrome, EFFICACY, MINOCYCLINE, Pediatrics population, Rare diseases