Angelman Syndrome MedDRA version: 16.0 Level: PT Classification code 10049004 Term: Angelman's syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Aknemin 50
Pharmaceutical Form: Capsule
INN or Proposed INN: MINOCYCLINE
CAS Number: 13614-98-7
Current Sponsor code: PL 33016/0005
Other descriptive name: MINOCYCLINE HYDROCHLORIDE
Concen
Sponsors
Dra. Cristina Avendaño Solá
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: (1) Male or female between 6 and 30 years old. (2) Clinical diagnosis of Angelman Syndrome and molecular confirmation of diagnosis. (3) The participant has an acceptable guardian can give consent on behalf of the participant. Are the trial subjects under 18? yes Number of subjects for this age range: 32 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: (1) Patients with hypersensitivity to tetracyclines. (2) Patients with impaired hepatic or renal function and in those with mainly drug allergy history. (3) 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 |
|---|---|
| Main Objective: To explore the efficacy of Mynocicline in the treatment of patients affected of Angelman Syndrome, in terms of increase in the age of development;Secondary Objective: 1) To compare mynocicline efficay vs placebo in terms of: -specific improvement of cognitive development, language and comunication, motor development, social-emotional development and adaptative conduct. - improvement of adaptive behaviour, , the ability to adapt to changes in their environment, learn new skills everyday and the level of independence 2) To evaluate the safety of mynociclne;Primary end point(s): Increased on the equivalent age of development, obtained through Development Scale R Merrill-Palmer (MP-R);Timepoint(s) of evaluation of this end point: 8, 16 and 24 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior obtained through Development Scale R Merrill-Palmer (MP-R), at 8, 16 and 24 weeks. - Improved adaptive behaviors, the ability to adapt to changes in their environment, learn new life skills and level of independence in the 8, 16 and 24 weeks obtained through the Vineland Adaptive Behavior Scale, Fourth Edition ( Vin-II). - 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, at 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.;Timepoint(s) of evaluation of this end point: 8, 16 and 24 weeks | — |
Countries
Spain
Contacts
Public ContactDra. Belén Ruiz Antorán
No
Outcome results
None listed