An open-label, Phase I/II, non-comparative, clinical study to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of MTL-CEBPA in paediatric participants with mucopolysaccharidosis type 1H (MPS1H, Hurler Syndrome) (SMART in MPS1H)

MiNA Therapeutics Ltd0 个研究点目标入组 12 人开始时间: 2023年2月24日最近更新: 2023年5月1日

试验速览

暂无简介。

•1. Verbal assent will be obtained from the participant as well as written informed consent from the parent/legal guardian prior to any study specific procedure
•2. Male or female participants aged =12-16 years (Cohort 1), or =4-11 years (Cohort 2) with a biochemical and genetic diagnosis of MPS-1H
•3. Minimum 6 months post HSCT
•4. Off immunosuppression, including corticosteroids for at least 4 weeks prior to study
•5. Stable myeloid chimerism (>80% donor cells as determined by short tandem repeats [STR] in )
•6. Anticipated life expectancy in excess of the study period
•7. Not enrolled in any other interventional study
•8. Acceptable laboratory parameters , consistent with participant age and MPS-1H diagnosis
•9. Negative blood pregnancy test for females of childbearing potential within 10 days prior to the first drug administration
•10. For female participants of child-bearing potential, agreement to be abstinent or use highly effective contraception (defined as method(s) that result in a failure rate of <1% per year) in females of childbearing potential during the entire study and defined post-study period
•11. Willingness and ability to comply with all protocol requirements including scheduled visits, treatment plans, laboratory tests and other study procedures

•1. Participants who received investigational drug(s) within the last 30 days (or 5 half-lives if longer) prior to study treatment initiation
•2. Participants receiving laronidase at commencement of study (minimum 6 weeks wash out)
•3. Major surgery within the last 30 days prior to study treatment initiation, or planned within the study period
•4. Pregnant or lactating women
•5. Known hypersensitivity to the active substance (MTL-CEBPA) or to any of the excipients, not managed by conventional approaches. Excipients include: 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphocholine (POPC); 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE); Cholesteryl hemisuccinate (CHEMS); and Cholesteryl-4-[[2-(4-morpholinyl)ethyl]amino]-4-oxobutanoate (MOCHOL)
•6. Any other condition (e.g., known or suspected poor compliance, etc.) that, in the judgment of the investigator, may affect the participant’s ability to follow the protocol specific procedures

发起方

MiNA Therapeutics Ltd