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PAH Deficiency clinical trials

View clinical trials related to PAH Deficiency.

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NCT ID: NCT05222178 Terminated - Phenylketonuria Clinical Trials

Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

Start date: June 3, 2022
Phase: Phase 1
Study type: Interventional

This is an open-label, sequential ascending dose-escalation, Phase 1 study to evaluate the safety and efficacy of a single intravenous (I.V.) administration of HMI-103, a gene editing development candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe restricted dietary management.

NCT ID: NCT03952156 Terminated - Phenylketonurias Clinical Trials

Gene Therapy Clinical Study in Adult PKU

pheNIX
Start date: June 10, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of HMI-102 and will be followed for safety and efficacy for 1 year.