A Randomized, Double-blind, Placebo-controlled Phase II/III Trial to Evaluate the Efficacy and Safety of 2 Doses of Oral Liarozole (75 mg od and 150 mg od) Given During 12 Weeks in Lamellar Ichthyosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 98
- 试验地点
- 20
- 主要终点
- Efficacy: Investigator's Global Assessment
研究概览
简要总结
Lamellar ichthyosis is a congenital disease of the skin with a generalized scaling. The primary activity of liarozole is considered to be the inhibition of the degradation of a substance called retinoic acid, which is the principal endogenous regulator of growth and differentiation of epithelial tissues in mammals. The current study intends to evaluate the efficacy and safety in patients with lamellar ichthyosis.
详细描述
Lamellar ichthyosis is an autosomal recessive disorder that is apparent at birth and is present throughout life. Although the disorder is not life threatening, it is quite disfiguring and causes considerable psychological stress to affected patients. Prevalence is less than 1 case per 300,000 individuals. Treatment is mainly symptomatic i.e. emollients with or without keratolytic agents. Treatment with systemic retinoids is reserved for those patients, refractory to conventional therapy, because of the long-term adverse effects and teratogenicity of systemic retinoids.
Liarozole may provide a new concept for the treatment of this condition. Because of its mechanism of action, retinoic acid (RA) levels will only be increased in tissues that are targets for RA production.
The proposed Phase II/III study intends to evaluate the efficacy of liarozole compared with placebo, in patients with lamellar ichthyosis.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 14 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects of either sex aged 14 years or older.
- •Clinical diagnosis of lamellar ichthyosis
- •Women of childbearing potential should use appropriate contraception
- •Women of childbearing potential should have a negative pregnancy test at screening visit.
- •Subjects are, except for their lamellar ichthyosis, in good general health.
- •Subjects and legal representative(s), if applicable, signed informed consent.
排除标准
- •Subject is receiving topical (except emollient), UV treatment or systemic treatment for ichthyosis.
- •Subject is pregnant or breast feeding.
- •History or suspicion of alcohol or drug abuse.
- •Significant co-existing diseases.
- •Clinically significant abnormal ECG
- •History of hypersensitivity to retinoids or any of the ingredients in the trial medication.
- •Clinically relevant laboratory abnormalities at screening.
- •Use of immune-suppressive drugs including topical or systemic corticosteroids.
- •Participation in an investigational trial 30 days prior to the start of the trial.
结局指标
主要结局
Efficacy: Investigator's Global Assessment
次要结局
- Severity scores of other symptoms
- Quality of Life
- Safety and tolerability
- Pharmacokinetics
- Overall Scaling Score