EUCTR2020-004000-33-FR进行中（未招募）1 期
A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults with ABCC6 Deficiency Manifesting as Pseudoxanthoma elasticum (PXE)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 9
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
- •1.Must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, following International Conference on Harmonisation (ICH) Good Clinical Practice (GCP).
- •2. Clinical diagnosis of PXE supported by prior or concurrent genetic identification of biallelic Abcc6 mutations
- •3. Male or female, ages 18 to <65 years of age at Screening
- •4. Plasma PPi < lower limit of normal (LLN) at screening
- •5. Women of child-bearing potential (WOCBP as defined in CTFG 2014) must have a negative serum pregnancy test at Screening and negative urine pregnancy tests at all additional pregnancy tests during the study.
- •6. WOCBP and partners of fertile males who are WOCBP must agree to use 2 highly effective forms of contraception (per CTFG 2014) from at least 1 month before the first dose of INZ-701 through 30 days after last dose of INZ-701 (greater than 5 half-lives of INZ-701). WOCBP and partners of fertile males who are WOCBP must also agree to not donate ova from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701
- •7. Males who are sexually active must agree to use condoms from the period following first dose of INZ-701 through 30 days after the last dose of INZ-701. Males must also agree to not donate sperm from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701.
- •8. In the opinion of the Investigator, must be willing and able to complete all aspects of the study
- •9. Agree to provide access to relevant medical records
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 9
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •1. In the opinion of the Investigator and/or Sponsor, presence of any clinically significant disease (outside of those considered associated with the diagnosis of ABCC6 Deficiency) that precludes study participation or may confound interpretation of study results, including uncontrolled thyroid disease or unrelated connective tissue, bone, mineral, ophthalmologic, or muscle disease
- •2. Advanced eye disease requiring anti-VEGF treatment at Screening
- •3. Clinically significant abnormal laboratory result at screening, including but not limited to elevations of aspartate aminotransferase, alanine aminotransferase, bilirubin, or creatinine greater than 2 times the upper limit of normal, 25 (OH) Vitamin D levels <20 ng/mL, parathyroid hormone (PTH) more than 20% above the upper limit of normal, and hyper- or hypocalcemia
- •4. Known active fungal, bacterial, and/or viral infection including human immunodeficiency virus, hepatitis B virus, hepatitis C virus, or COVID-19 virus. A negative COVID-19 test result is required within 5 days of first dose of INZ-701
- •5. Known intolerance to any INZ-701 excipient
- •6. Unable or unwilling to discontinue the use of any prohibited medication (examples include bisphosphonates, calcimimetics, antacids, systemic corticosteroids, pyrophosphate containing medications), as provided in protocol Section 9.4
- •7. Receipt of any other investigational new drug within 5 half-lives of the last dose of the other investigational drug or from 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device through completion of participation in the study
- •8. Last symptoms from a COVID-19 vaccination within 14 days prior to the first dose of INZ-701 or as described in the Inozyme COVID-19 Vaccine Guidance Document
- •9. Women who are breastfeeding
研究者
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