Trial record 4 of 14 for:    angelman syndrome

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by Puerta de Hierro University Hospital
Sponsor:
Information provided by (Responsible Party):
BELEN RUIZ-ANTORAN, Puerta de Hierro University Hospital
ClinicalTrials.gov Identifier:
NCT02056665
First received: February 5, 2014
Last updated: NA
Last verified: February 2014
History: No changes posted
  Purpose

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


Condition Intervention Phase
Angelman Syndrome
Drug: MINOCYCLINE
Drug: PLACEBO (for Minocycline)
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Randomized Clinical Trial, Placebo Compared to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome

Resource links provided by NLM:


Further study details as provided by Puerta de Hierro University Hospital:

Primary Outcome Measures:
  • Increased on the equivalent age of development [ Time Frame: 8, 16 and 24 weeks ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    Improvement of CGI. . Measure based on changes in the Clinical Global Impression through the perception of parents or guardians, you neurologists and therapist


Estimated Enrollment: 32
Study Start Date: January 2014
Estimated Study Completion Date: November 2014
Estimated Primary Completion Date: September 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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
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
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
Other Names:
  • Commercial name: Aknemin 50
  • Active substance: MINOCYCLINE
  • Administration routes: Oral use

Detailed Description:

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

  Eligibility

Ages Eligible for Study:   6 Years to 30 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02056665

Contacts
Contact: Belen Ruiz-Antoran 34911917479

Locations
Spain
Puerta de Hierro University Hospital Recruiting
Madrid, Spain, 28222
Contact: Belen Ruiz-Antoran    34911917479    mariabelen.ruiz@salud.madrid.org   
Principal Investigator: Belen Ruiz-Antorán         
Sponsors and Collaborators
Puerta de Hierro University Hospital
Investigators
Principal Investigator: Belen Ruiz-Antorán Clinical Pharmacology. Puerta de Hierro University Hospital
  More Information

No publications provided

Responsible Party: BELEN RUIZ-ANTORAN, DR, Puerta de Hierro University Hospital
ClinicalTrials.gov Identifier: NCT02056665     History of Changes
Other Study ID Numbers: A-MANECE
Study First Received: February 5, 2014
Last Updated: February 5, 2014
Health Authority: Spain: Spanish Agency of Medicines

Keywords provided by Puerta de Hierro University Hospital:
Angelman Syndrome
EFFICACY
SAFETY
MINOCYCLINE
Pediatrics population
Rare diseases

Additional relevant MeSH terms:
Angelman Syndrome
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
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 28, 2014