EUCTR2021-002244-70-SE进行中（未招募）1 期
Rollover Study; Multicentre, Phase III, Open-label Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged =14 Years with Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed Study PVO-1A-301 or PVO-1A-202/PVO-1A-204 and May Benefit from Palovarotene Therapy. - PIVOINE
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Ipsen Pharma
- 入组人数
- 61
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
- •Participants are eligible to be included in the study only if all of the following criteria apply:
- •1. Participant has completed the EOS or End of Treatment Visit of Study PVO-1A-301 or PVO-1A-202 (PVO-1A-202 Parts C and D correspond to Study PVO-1A-204 in France). There must be a current parent study signed ICF form for the participant to be eligible for Study CLIN-60120-452.
- •2. In the investigator’s judgment, the participant may benefit from continued participation in a palovarotene study.
- •3. Participant must be =14 years of age (aligned with the age of treated participants in the ongoing parent studies PVO-1A-301 and PVO-1A-202/PVO-1A-204) and qualify as 100% skeletally mature (if <18 years, based on assessments carried out at parent EOS Visit; if =18 years, automatically considered 100% skeletally mature) or have reached final adult height based on investigator’s assessment*, at the time the Study CLIN60120-452 informed consent is signed.
- •*The above criteria can be reached at any point after parent study end for the participant to be eligible for Study CLIN-60120-452, as long as it is prior to palovarotene becoming commercially available in the country where the study is being conducted.
- •4. Male, or female who is not pregnant or breastfeeding, and has met all of the following conditions:
- •Females of child-bearing potential (FOCBP) (defined in Section 10.2.1 of the protocol) must have a negative blood or urine pregnancy test (with sensitivity of at least 50 mIU/mL) prior to administration of palovarotene;
- •FOCBP must agree to remain abstinent from heterosexual sex during treatment and for one month after treatment or, if sexually active, to use two effective methods of birth control (described in Section 10.2.2 of the protocol) during and for one month after treatment;
- •Additionally, sexually active FOCBP must already be using two effective methods of birth control one month before treatment is to start;
- •Specific risk of the use of retinoids during pregnancy, and the agreement to remain abstinent or use two effective methods of birth control will be clearly defined in the informed consent and the participant or legally authorised representatives (e.g. parents, caregivers or legal guardians) must specifically sign this section.
- •Contraceptive use by women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- •Informed Consent and Assent
- •5. Capable of giving signed informed consent or providing assent as described in Section 10.1.3 of the protocol.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 14
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 47
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Participants will not be included in the study if any of the following criteria apply:
- •Medical conditions
- •1. History of allergy or hypersensitivity to retinoids, gelatin, lactose (note that lactose intolerance is not exclusionary) or palovarotene, or unresponsiveness to prior treatment with palovarotene.
- •2. Uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric, or other significant disease.
- •3. Intercurrent known or suspected non-healed fracture at any location;
- •4. Any other medical condition/clinically significant abnormalities that would expose the participant to undue risk or interfere with study assessments.
- •5. Amylase or lipase >2× above the upper limit of normal (ULN) or with a history of chronic pancreatitis.
- •6. Elevated aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >2.5× ULN.
- •7. Fasting triglycerides >400 mg/dL with or without therapy.
- •8. Suicidal ideation (type 4 or 5) or any suicidal behaviour within the past month as defined by the Columbia-Suicide Severity Rating Scale (C-SSRS).
- •Prior/concomitant therapy
- •9. Current use of 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.
- •10. Exposure to synthetic oral retinoids other than palovarotene within 4 weeks of the Inclusion Visit.
- •11. Concurrent treatment with tetracycline or any tetracycline derivatives due to the potential increased risk of pseudotumor cerebri.
- •12. Use of concomitant medications that are strong inhibitors or inducers of cytochrome P450 (CYP450) 3A4 activity (see Section 6.8 and 10.5 of protocol); or kinase inhibitors such as imatinib.
- •Other exclusion criteria
- •13. Palovarotene is commercially available in the country where the study is being conducted.
- •14. Any reason that, in the opinion of the investigator, would lead to the inability of the participant and/or family to comply with the protocol.
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