CTIS2023-507382-26-00招募中1 期
The ENERGY 3 Study: A Randomized, Controlled, Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Children with Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency - INZ701-106
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 33
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 17（—）
- 性别
- All
入选标准
- •Caregiver’s written or electronic informed consent after the nature of the study has been explained, and prior to any research-related procedures, per International Conference on Harmonisation (ICH) Good Clinical Practice (GCP), 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, WOCBP and partners of fertile males who are WOCBP must be using or agree to use a highly effective form of contraception (as per CTFG 2020 ENREF 4) from at least 1 month before the first dose of INZ-701 through 30 days after the last dose of INZ-701 (greater than 5 half-lives of INZ-701), In the opinion of the Investigator, able to complete all aspects of the study, Study participant’s assent in accordance with local regulations, A confirmed postnatal molecular genetic diagnosis of ENPP1 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory or regional equivalent, Males and females =1 year and <13 years of age at Study Day 1, Open growth plates of the distal femur and proximal tibia in both legs, Plasma PPi concentration of <1400 nM at Screening, 25-hydroxyvitamin D (25[OH]D) levels of =12 ng/mL at Screening, Radiographic evidence of skeletal abnormalities based on an RSS =2, Women of childbearing potential (WOCBP, as defined in Clinical Trials Facilitation and Coordination Group [CTFG 2020]) must have a negative serum pregnancy test at Screening and must not be breastfeeding
排除标准
- •In the opinion of the Investigator, has clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency that will preclude study participation and/or may confound the interpretation of study results, If receiving any of the following prohibited medications as indicated in the protocol: systemic corticosteroids (>5 mg prednisone equivalent per day), anti-fibroblast growth factor 23 (FGF23), and oral and/or IV bisphosphonates, Unable or unwilling to discontinue calcitriol or other active forms of vitamin D3 (or analogs) within 7 days prior to Study Day 1 and/or oral phosphate supplements within 36 hours prior to Study Day 1 if randomized to the INZ-701 arm, Planned orthopedic surgery that may confound the interpretation of study results during the 52-week Randomized Treatment Period, Known intolerance to INZ-701 or any of its excipients, A positive COVID-19 test within 5 days prior to Randomization, only if required as per local regulations or institutional policy, Previous treatment with INZ-701, Concurrent participation in another interventional clinical study and/or has received an investigational drug within 5 half-lives of the last dose or within 4 weeks prior to Randomization, whichever is longer, or use of an investigational device
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