Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome

NCT02056665 | Completed Phase 2

• 1 other identifier: A-MANECE
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Angelman Syndrome

Keywords

Angelman Syndrome; EFFICACY; SAFETY; MINOCYCLINE; Pediatrics population; Rare diseases

Outcome Measures

Primary Outcomes (1)

• Increased on the equivalent age of development

  Increased on the equivalent age of development, obtained through Development Scale R Merrill-Palmer (MP-R)

  8, 16 and 24 weeks

Secondary Outcomes (4)

• Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior

  8, 16 and 24 weeks

• Improvement of EEG.

  8, 16 and 24 weeks

• Safety and tolerability

  8, 16 and 24 weeks

• Clinical Global impression (CGI)

  8, 16 and 24 weeks

Study Arms (3)

MINOCYCLINE 8 weeks

EXPERIMENTAL

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; Drug: PLACEBO (for Minocycline)

PLACEBO 8 weeks

PLACEBO COMPARATOR

Pill manufactured to mimic Minocycline 50 mg capsule

Drug: MINOCYCLINE; Drug: PLACEBO (for Minocycline)

MINOCYCLINE 16 weeks

EXPERIMENTAL

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

Interventions

MINOCYCLINE DRUG

Pill Minocycline 50 mg capsule

Also known as: Commercial name: Aknemin 50, Active substance: MINOCYCLINE, Administration routes: Oral use

MINOCYCLINE 16 weeks; MINOCYCLINE 8 weeks; PLACEBO 8 weeks

PLACEBO (for Minocycline) DRUG

Pill manufactured to mimic Minocycline 50 mg capsule

Also known as: Commercial name: NA, Non Active substance:, Administration routes: Oral use

MINOCYCLINE 8 weeks; PLACEBO 8 weeks

Eligibility Criteria

• Age: 6 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Puerta de Hierro University Hospital: lead

Study Sites (1)

Puerta de Hierro University Hospital

Madrid, Madrid, 28222, Spain

Location

Related Publications (1)

• 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.

  PMID: 30126448 DERIVED

MeSH Terms

Conditions

Angelman Syndrome; cyclopia sequence; Rare Diseases

Interventions

Minocycline

Condition Hierarchy (Ancestors)

Movement Disorders; Central Nervous System Diseases; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Tetracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds

Study Officials

• Belen Ruiz-Antorán

  Clinical Pharmacology. Puerta de Hierro University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: DR

Study Record Dates

• First Submitted: February 5, 2014
• First Posted: February 6, 2014
• Study Start: January 1, 2014
• Primary Completion: September 1, 2014
• Study Completion: November 1, 2014
• Last Updated: October 21, 2015

Record last verified: 2014-10

Locations

ES (1)