Randomized Clinical Trial, Placebo Compared to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 32
- 试验地点
- 1
- 主要终点
- Increased on the equivalent age of development
研究概览
简要总结
RANDOMIZED CLINICAL TRIAL, PLACEBO COMPARED TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY)
详细描述
STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
研究组 & 干预措施
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.
干预措施: MINOCYCLINE (Drug)
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.
干预措施: PLACEBO (for Minocycline) (Drug)
PLACEBO 8 weeks
Pill manufactured to mimic Minocycline 50 mg capsule
干预措施: MINOCYCLINE (Drug)
PLACEBO 8 weeks
Pill manufactured to mimic Minocycline 50 mg capsule
干预措施: PLACEBO (for Minocycline) (Drug)
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.
干预措施: MINOCYCLINE (Drug)
结局指标
主要结局
Increased on the equivalent age of development
时间窗: 8, 16 and 24 weeks
Increased on the equivalent age of development, obtained through Development Scale R Merrill-Palmer (MP-R)
次要结局
- Improvement of EEG.(8, 16 and 24 weeks)
- Safety and tolerability(8, 16 and 24 weeks)
- Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior(8, 16 and 24 weeks)
- Clinical Global impression (CGI)(8, 16 and 24 weeks)
研究者
BELEN RUIZ-ANTORAN
DR
Puerta de Hierro University Hospital