EUCTR2017-002541-29-ES进行中（未招募）1 期
A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) - MOVE Trial
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 80
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Subjects must meet all of the following criteria to be eligible for enrollment:
- •1. Written, signed, and dated informed subject/parent consent; and for subjects who are minors, age-appropriate assent (performed according to local
- •regulations).
- •2. Male or female at least 4 years of age.
- •3. Clinically diagnosed with FOP, with the R206H ACVR1 mutation.
- •4. No flare-up symptoms within the past 4 weeks, including at the time of enrollment.
- •5. Females of child-bearing potential must have a negative blood or urine pregnancy test (with sensitivity of at least 50 mIU/mL) prior to administration of palovarotene. Male and FOCBP subjects must agree to remain abstinent during treatment and for 1 month after treatment or, if sexually active, to use two highly effective methods of birth control during and for 1 month after treatment. Additionally, sexually active FOCBP subjects must already be using two highly effective methods of birth control 1 month before treatment is to start. Specific risk of the use of retinoids during pregnancy, and the agreement to remain abstinent or use two highly effective methods of birth control will be clearly defined in the informed consent and the subject or legally authorized representatives (eg, parents, caregivers, or legal guardians) must specifically sign this section.
- •6. Must be accessible for treatment and follow-up, and be able to undergo all study procedures. Subjects living at distant locations from the
- •investigational site must be able and willing to travel to a site for the initial and all on-site follow-up visits. Subjects must be able to undergo low-dose,
- •WBCT (excluding head).
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 20
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 60
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Subjects meeting any of the following criteria are not eligible for enrollment:
- •1. Weight <10 kg.
- •2. If currently using vitamin A or beta carotene, multivitamins containing vitamin A or beta carotene, or herbal preparations, fish oil, and unable or
- •unwilling to discontinue use of these products during palovarotene treatment.
- •3. Exposure to synthetic oral retinoids other than palovarotene within 4 weeks prior to screening.
- •4. Concurrent treatment with tetracycline or any tetracycline derivatives due to the potential increased risk of pseudotumor cerebri.
- •5. History of allergy or hypersensitivity to retinoids or lactose (note that lactose intolerance is not exclusionary).
- •6. Concomitant medications that are strong inhibitors or inducers of cytochrome P450 (CYP450) 3A4 activity; or kinase inhibitors such as
- •imatinib (see Section 5.2.1).
- •7. Amylase or lipase >2x above the upper limit of normal (ULN) or with a history of chronic pancreatitis.
- •8. Elevated aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >2.5x ULN.
- •9. Fasting triglycerides >400 mg/dL with or without therapy.
- •10. Female subjects who are breastfeeding.
- •11. Subjects with uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic,
- •psychiatric, or other significant disease.
- •12. Subjects experiencing suicidal ideation (type 4 or 5) or any suicidal behavior within the past month as defined by the Columbia-Suicide Severity
- •Rating Scale (C-SSRS).
- •13. Simultaneous participation in another clinical research study within 4 weeks prior to Screening; or within five half-lives of the investigational agent,
- •whichever is longer.
- •14. Any reason that, in the opinion of the Investigator, would lead to the inability of the subject and/or family to comply with the protocol.
研究者
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