The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 22
- 试验地点
- 10
- 主要终点
- Number of Treatment Emergent Adverse Events (TEAEs)
研究概览
简要总结
The purpose of this study (Study INZ701-304 [ADAPT]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
详细描述
The investigational product INZ-701 is being developed as a therapeutic protein for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency and adenosine triphosphate (ATP)-binding cassette subfamily C member 6 (ABCC6) Deficiency. INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin (Ig) G1 antibody.
The ADAPT Study (INZ701-304) is an open-label study to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.
The study will consist of a 30-day Screening Period, followed by an open-label Treatment Period during which all participants will receive once-weekly subcutaneous (SC) doses of INZ-701 and continue with treatment until INZ-701 is commercially available in the country/region of the participant's residence or until Inozyme chooses to discontinue development of INZ-701. Participants will complete an End of Study (EOS) safety follow-up visit approximately 30 days after their last designated study visit assigned by the Investigator and/or Sponsor.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Individuals eligible to participate must meet all of the following inclusion criteria:
- •Provide written or electronic informed consent after the nature of the study has been explained, and prior to any research-related procedures, per International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP)
- •Provide assent in accordance with local regulations, if <18 years of age
- •Male or female, greater than 1 year of age
- •Must have completed the protocol-required safety and PK/PD and/or efficacy period(s) of a previous INZ-701 clinical study in ENPP1 or ABCC6 Deficiency, as confirmed by the Sponsor
- •Female participants of childbearing potential who are sexually active must be using or agree to use 1 highly effective form of contraception (per CTFG 2020) 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); participants must agree to not donate ova from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701
- •Male participants who are sexually active must agree to use condoms from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701; participants must agree to not donate sperm from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701
- •In the opinion of the Investigator, able to complete all aspects of the study
- •Individuals who meet any of the following
排除标准
- •will not be eligible to participate:
- •In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency or ABCC6 Deficiency, that will preclude study participation and/or may confound interpretation of study results
- •Known intolerance to INZ-701 or any of its excipients
- •Concurrent participation in another interventional clinical study and/or has received an investigational drug other than INZ-701 within 5 half-lives or within 4 weeks prior to the first dose of INZ-701 in this study, whichever is longer, or use of an investigational device
- •Pregnant, trying to become pregnant, or breastfeeding
- •Male participants trying to father a child
研究组 & 干预措施
INZ-701
INZ-701 will be administered by subcutaneous injection on a once-weekly basis as follows:
- Study participants from 1 year to <13 years of age will receive a dose of 2.4 mg/kg
- Study participants ≥13 years will either receive a 1.8 mg/kg dose or a 150 mg flat dose of INZ-701
干预措施: INZ-701 (Drug)
结局指标
主要结局
Number of Treatment Emergent Adverse Events (TEAEs)
时间窗: 6 years (long term safety assessment)
Treatment-emergent AEs are defined as any AE occurring from the first dose of INZ-701 through 30 days after the last dose of INZ-701.
Incidence of Anti-Drug Antibodies (ADA)
时间窗: 6 years (long term safety assessment)
For each subject, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.
次要结局
- Mean Change from Baseline in Plasma PPi Concentration(6 years (long term safety assessment))
- Time to maximum serum concentration (Tmax)(6 years (long term safety assessment))
- Maximum Plasma Concentration (Cmax) of INZ-701(6 years (long term safety assessment))