Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome (A-MANECE)

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ClinicalTrials.gov Identifier: NCT02056665

Recruitment Status : Completed

First Posted : February 6, 2014

Last Update Posted : October 21, 2015

Sponsor:

Information provided by (Responsible Party):

BELEN RUIZ-ANTORAN, Puerta de Hierro University Hospital

Study Description

Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Angelman Syndrome	Drug: MINOCYCLINE Drug: PLACEBO (for Minocycline)	Phase 2

Detailed Description:

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

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	32 participants
Allocation:	Randomized
Intervention Model:	Crossover Assignment
Masking:	Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:	Treatment
Official Title:	Randomized Clinical Trial, Placebo Compared to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome
Study Start Date :	January 2014
Actual Primary Completion Date :	September 2014
Actual Study Completion Date :	November 2014

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Angelman syndrome

Drug Information available for: Minocycline Minocycline hydrochloride

Genetic and Rare Diseases Information Center resources: Angelman Syndrome

U.S. FDA Resources

Arms and Interventions

Arm	Intervention/treatment
Experimental: MINOCYCLINE 8 weeks 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 Pill Minocycline 50 mg capsule Other Names: Commercial name: Aknemin 50 Active substance: MINOCYCLINE Administration routes: Oral use Drug: PLACEBO (for Minocycline) Pill manufactured to mimic Minocycline 50 mg capsule Other Names: Commercial name: NA Non Active substance: Administration routes: Oral use
Placebo Comparator: PLACEBO 8 weeks Pill manufactured to mimic Minocycline 50 mg capsule	Drug: MINOCYCLINE Pill Minocycline 50 mg capsule Other Names: Commercial name: Aknemin 50 Active substance: MINOCYCLINE Administration routes: Oral use Drug: PLACEBO (for Minocycline) Pill manufactured to mimic Minocycline 50 mg capsule Other Names: Commercial name: NA Non Active substance: Administration routes: Oral use
Experimental: MINOCYCLINE 16 weeks 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 Pill Minocycline 50 mg capsule Other Names: Commercial name: Aknemin 50 Active substance: MINOCYCLINE Administration routes: Oral use

Outcome Measures

Primary Outcome Measures :

Increased on the equivalent age of development [ Time Frame: 8, 16 and 24 weeks ]
Increased on the equivalent age of development, obtained through Development Scale R Merrill-Palmer (MP-R)

Secondary Outcome Measures :

Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior [ Time Frame: 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. [ Time Frame: 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 [ Time Frame: 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) [ Time Frame: 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

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:	6 Years to 30 Years (Child, Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02056665

Locations

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Spain
Puerta de Hierro University Hospital
Madrid, Spain, 28222

Sponsors and Collaborators

Puerta de Hierro University Hospital

Investigators

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Principal Investigator:	Belen Ruiz-Antorán	Clinical Pharmacology. Puerta de Hierro University Hospital

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Ruiz-Antoran B, Sancho-López A, Cazorla-Calleja R, López-Pájaro LF, Leiva Á, Iglesias-Escalera G, Marín-Serrano ME, Rincón-Ortega M, Lara-Herguedas J, Rossignoli-Palomeque T, Valiente-Rodríguez S, González-Marques J, Román-Riechmann E, Avendaño-Solá 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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Responsible Party:	BELEN RUIZ-ANTORAN, DR, Puerta de Hierro University Hospital
ClinicalTrials.gov Identifier:	NCT02056665
Other Study ID Numbers:	A-MANECE
First Posted:	February 6, 2014 Key Record Dates
Last Update Posted:	October 21, 2015
Last Verified:	October 2014

Keywords provided by BELEN RUIZ-ANTORAN, Puerta de Hierro University Hospital:


Angelman Syndrome EFFICACY SAFETY	MINOCYCLINE Pediatrics population Rare diseases

Additional relevant MeSH terms:

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Angelman Syndrome Syndrome Disease Pathologic Processes Movement Disorders Central Nervous System Diseases Nervous System Diseases	Abnormalities, Multiple Congenital Abnormalities Chromosome Disorders Genetic Diseases, Inborn Minocycline Anti-Bacterial Agents Anti-Infective Agents

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