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

Majadahonda Iron Gate University

Status and phase: Completed; Phase 2

Conditions: Angelman Syndrome

Treatments: Drug: MINOCYCLINE; Drug: PLACEBO (for Minocycline)

Study type: Interventional

Funder types: Other

Identifiers

NCT02056665

A-MANECE

Details and patient eligibility

About

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

Full description

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

Enrollment: 32 patients

Sex: All

Ages: 6 to 30 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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32 participants in 3 patient groups, including a placebo group

MINOCYCLINE 8 weeks

Experimental group

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.

Treatment:

Drug: PLACEBO (for Minocycline)

Drug: MINOCYCLINE

PLACEBO 8 weeks

Placebo Comparator group

Description:

Pill manufactured to mimic Minocycline 50 mg capsule

Treatment:

Drug: PLACEBO (for Minocycline)

Drug: MINOCYCLINE

MINOCYCLINE 16 weeks

Experimental group

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.

Treatment:

Drug: MINOCYCLINE