NL-OMON48469已完成2 期
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects with Multiple Osteochondromas - MO-Ped
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 10
研究概览
简要总结
Trial ended prematurely
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 17（—）
入选标准
- •1. Written, signed, and dated informed subject/parent consent and age
- •appropriate assent (performed according to local regulations)., 2. A clinical
- •diagnosis of MO with a disease-causing Ext1 or Ext2 mutations confirmed by a
- •central laboratory., 3. Male and female subjects with a chronological age of
- •2-14 years, inclusive., 4. Female subjects must be premenarchal at screening.,
- •5. Bone age at screening of <=14 years, 0 months per the Greulich-Pyle method as
- •assesed by a central reader., 6. Symptomatic MO, defined as the occurrence of
- •any one of the following at screening:, • Five or more clinically-evident OCs
- •and the presence of a new or enlarging OC in the preceding 12 months.
- •Five or more clinically-evident OCs and the presence of a painful OC.
- •A skeletal deformity.
- •A joint limitation.
- •Prior surgery for a MO-related complication., 7. If a subject had a prior
- •surgery for MO, the subject should not be screened until at least 8 weeks
- •post-surgery to allow for at least 12 weeks of stabilization of symptoms prior
- •to first dose. Surgical orthopedic implants are allowed if they were in situ
- •for >=12 weeks prior to the baseline MRI., 8. If a subject is currently
- •receiving pain medications, the dose must be stable (ie, <20% variance) for 2
- •weeks prior to screening., 9. The ability to undergo whole body MRI with or
- •without sedation/general anesthesia., 10. Male and female subjects of child
- •bearing potential who are heterosexually active must agree to use two highly
- •effective methods of birth control, one of which must be highly effective
- •during treatment, and for 1 month after treatment discontinuation, unless they
- •commit to true abstinence from heterosexual sex. Heterosexually active females
- •of child bearing potential (FOCBP) must also agree to start effective methods
- •of birth control at screening. An FOCBP is defined as a female who is 13 or
- •older of age or is post-menarchal, whichever is earlier, 11. Subjects must be
- •accessible for treatment with study drug and follow-up.
排除标准
- •1. A weight <10 kg., 2. Other known syndromic conditions such as Langer-Giedion
- •or Potocki Shaffer., 3. Any subject with neurologic signs suggestive of spinal
- •cord impingement., 4. If subject is 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. For eligibility, no washout is required prior to the
- •first dose of study drug., 5. Exposure to synthetic oral retinoids within 4
- •weeks prior to enrollment., 6. Concurrent treatment with tetracycline or any
- •tetracycline derivatives, due to the potential increased risk of pseudotumor
- •cerebri., 7. History of allergy or hypersensitivity to retinoids, gelatin or
- •lactose (other than lactose intolerance)., 8. Concomitant medications that are
- •strong inhibitors or inducers of cytochrome P450 (CYP450) 3A4 activity., 9.
- •Amylase or lipase >2 times the above the upper limit of normal (>2×ULN) or with
- •a history of chronic pancreatitis., 10. Elevated aspartate aminotransferase
- •(AST) or alanine aminotransferase (ALT) >;2.5x ULN., 11. Fasting triglycerides
- •>400 mg/dL with or without therapy., 12. Subjects with uncontrolled
- •cardiovascular, renal, hepatic, pulmonary, gastrointestinal, endocrine,
- •metabolic, ophthalmologic, immunologic, psychiatric, or other significant
- •disease. These include subjects requiring glucocorticoid at doses >0.2mg/kg or
- •up to 10 mg prednisone equivalent daily. , 13. Subjects experiencing suicidal
- •ideation (type 4 or 5) or any suicidal behavior within the past month or any
- •suicidal behavior within the past year as defined by the Columbia-Suicide
- •Severity Rating Scale (C SSRS)., 14. Subjects unable or unwilling to complete
- •the study or all study-related procedures, including imaging., 15. Any surgical
- •implant that is contraindicated for MRI. Dental braces are permitted., 16.
- •Participation in any clinical research study within 4 weeks prior to enrollment
- •or simultaneous participation in any clinical research study., 17. 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.
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