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

NCT05222178 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2023-10-10

No results posted yet for this study

Summary

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.

Conditions

Interventions

DRUG

HMI-103

HMI-103 is an AAVHSC15 capsid containing a functional copy of the human PAH gene

Sponsors & Collaborators

  • Homology Medicines, Inc

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-06-03
Primary Completion
2023-09-14
Completion
2023-09-14
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05222178 on ClinicalTrials.gov