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RANDOMIZED CLINICAL TRIAL, PLACEBO COMPARED TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY)
STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MINOCYCLINE 8 weeks | Experimental | Duration of treatment: 8 weeks
|
|
| PLACEBO 8 weeks | Placebo Comparator | Pill manufactured to mimic Minocycline 50 mg capsule |
|
| MINOCYCLINE 16 weeks | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MINOCYCLINE | Drug | Pill Minocycline 50 mg capsule |
|
| 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 |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Belen Ruiz-Antorán | Clinical Pharmacology. Puerta de Hierro University Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Puerta de Hierro University Hospital | Madrid | Madrid | 28222 | Spain |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30126448 | Derived | Ruiz-Antoran B, Sancho-Lopez A, Cazorla-Calleja R, Lopez-Pajaro LF, Leiva A, Iglesias-Escalera G, Marin-Serrano ME, Rincon-Ortega M, Lara-Herguedas J, Rossignoli-Palomeque T, Valiente-Rodriguez S, Gonzalez-Marques J, Roman-Riechmann E, Avendano-Sola C. A randomized placebo controlled clinical trial to evaluate the efficacy and safety of minocycline in patients with Angelman syndrome (A-MANECE study). Orphanet J Rare Dis. 2018 Aug 20;13(1):144. doi: 10.1186/s13023-018-0891-6. |
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| Type | Date | Date Unknown |
|---|---|---|
| Release | Sep 7, 2018 | |
| Reset | Feb 8, 2019 |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Sep 7, 2018 | Feb 8, 2019 |
| ID | Term |
|---|---|
| D017204 | Angelman Syndrome |
| C562573 | cyclopia sequence |
| D035583 | Rare Diseases |
| ID | Term |
|---|---|
| D009069 | Movement Disorders |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D000015 | Abnormalities, Multiple |
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| ID | Term |
|---|---|
| D008911 | Minocycline |
| ID | Term |
|---|---|
| D013754 | Tetracyclines |
| D009279 | Naphthacenes |
| D011084 | Polycyclic Aromatic Hydrocarbons |
| D006841 | Hydrocarbons, Aromatic |
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| PLACEBO (for Minocycline) | Drug | Pill manufactured to mimic Minocycline 50 mg capsule |
|
|
| 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 |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D000096803 | Imprinting Disorders |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D006844 |
| Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D011083 | Polycyclic Compounds |