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

Primary Purpose

Angelman Syndrome

Status
Completed
Phase
Phase 2
Locations
Spain
Study Type
Interventional
Intervention
MINOCYCLINE
PLACEBO (for Minocycline)
Sponsored by
Puerta de Hierro University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Angelman Syndrome focused on measuring Angelman Syndrome, EFFICACY, SAFETY, MINOCYCLINE, Pediatrics population, Rare diseases

Eligibility Criteria

6 Years - 30 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Puerta de Hierro University Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Placebo Comparator

Experimental

Arm Label

MINOCYCLINE 8 weeks

PLACEBO 8 weeks

MINOCYCLINE 16 weeks

Arm Description

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.

Pill manufactured to mimic Minocycline 50 mg capsule

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.

Outcomes

Primary Outcome Measures

Increased on the equivalent age of development
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
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.
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
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)
Improvement of CGI. . Measure based on changes in the Clinical Global Impression through the perception of parents or guardians, you neurologists and therapist

Full Information

First Posted
February 5, 2014
Last Updated
October 19, 2015
Sponsor
Puerta de Hierro University Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT02056665
Brief Title
Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome
Acronym
A-MANECE
Official Title
Randomized Clinical Trial, Placebo Compared to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
October 2014
Overall Recruitment Status
Completed
Study Start Date
January 2014 (undefined)
Primary Completion Date
September 2014 (Actual)
Study Completion Date
November 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Puerta de Hierro University Hospital

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Angelman Syndrome
Keywords
Angelman Syndrome, EFFICACY, SAFETY, MINOCYCLINE, Pediatrics population, Rare diseases

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
32 (Actual)

8. Arms, Groups, and Interventions

Arm Title
MINOCYCLINE 8 weeks
Arm Type
Experimental
Arm Description
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.
Arm Title
PLACEBO 8 weeks
Arm Type
Placebo Comparator
Arm Description
Pill manufactured to mimic Minocycline 50 mg capsule
Arm Title
MINOCYCLINE 16 weeks
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
MINOCYCLINE
Other Intervention Name(s)
Commercial name: Aknemin 50, Active substance: MINOCYCLINE, Administration routes: Oral use
Intervention Description
Pill Minocycline 50 mg capsule
Intervention Type
Drug
Intervention Name(s)
PLACEBO (for Minocycline)
Other Intervention Name(s)
Commercial name: NA, Non Active substance:, Administration routes: Oral use
Intervention Description
Pill manufactured to mimic Minocycline 50 mg capsule
Primary Outcome Measure Information:
Title
Increased on the equivalent age of development
Description
Increased on the equivalent age of development, obtained through Development Scale R Merrill-Palmer (MP-R)
Time Frame
8, 16 and 24 weeks
Secondary Outcome Measure Information:
Title
Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior
Description
Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior obtained through Development Scale R Merrill-Palmer (MP-R)
Time Frame
8, 16 and 24 weeks
Title
Improvement of EEG.
Description
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
Time Frame
8, 16 and 24 weeks
Title
Safety and tolerability
Description
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.
Time Frame
8, 16 and 24 weeks
Title
Clinical Global impression (CGI)
Description
Improvement of CGI. . Measure based on changes in the Clinical Global Impression through the perception of parents or guardians, you neurologists and therapist
Time Frame
8, 16 and 24 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Belen Ruiz-Antorán
Organizational Affiliation
Clinical Pharmacology. Puerta de Hierro University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Puerta de Hierro University Hospital
City
Madrid
ZIP/Postal Code
28222
Country
Spain

12. IPD Sharing Statement

Citations:
PubMed Identifier
30126448
Citation
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.
Results Reference
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Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome

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