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

October 19, 2015 updated by: BELEN RUIZ-ANTORAN, Puerta de Hierro University Hospital

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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

Study Type

Interventional

Enrollment (Actual)

32

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Spain
      • Madrid, Spain, 28222
        • Puerta de Hierro University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 30 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / 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:
  • Administration routes: Oral use
  • Commercial name: NA
  • Non Active substance:
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:
  • Administration routes: Oral use
  • Commercial name: NA
  • Non Active substance:
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
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
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)
8, 16 and 24 weeks
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
8, 16 and 24 weeks
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.
8, 16 and 24 weeks
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
8, 16 and 24 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Puerta de Hierro University Hospital

Investigators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2014

Primary Completion (Actual)

September 1, 2014

Study Completion (Actual)

November 1, 2014

Study Registration Dates

First Submitted

February 5, 2014

First Submitted That Met QC Criteria

February 5, 2014

First Posted (Estimate)

February 6, 2014

Study Record Updates

Last Update Posted (Estimate)

October 21, 2015

Last Update Submitted That Met QC Criteria

October 19, 2015

Last Verified

October 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • A-MANECE

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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