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)

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 03 Aug 2023

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At a glance

Drugs MTL CEBPA (Primary)
Indications Mucopolysaccharidosis I
Focus Adverse reactions
Acronyms SMART in MPS-IH
Sponsors MiNA Therapeutics

Most Recent Events

18 Apr 2023 Planned initiation date changed from 1 Sep 2022 to 1 Aug 2023.
18 Apr 2023 Status changed from recruiting to not yet recruiting.
01 Mar 2023 Status changed from not yet recruiting to recruiting.

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