Long-Term Follow Up Study of Subjects Previously Administered HMI 102

An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects With PAH Deficiency Previously Administered HMI 102

Status

Enrolling by invitation

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04348708

Enrollment

Registered

2020-04-16

Start date

2020-08-19

Completion date

2026-12-01

Last updated

2022-06-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

PAH Deficiency, Phenylketonuria

Keywords

PKU, Phenylketonuria, PAH Deficiency, Phenylalanine, Adeno Associated Virus, AAVHSC15

Brief summary

An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects with PAH Deficiency Previously Administered HMI 102

Detailed description

This Long-Term Follow Up Study will evaluate the safety and efficacy of PKU Subjects with PAH Deficiency Previously Administered HMI 102. Subjects will have already received a single dose of HMI-102 administered intravenously

Interventions

GENETICHMI-102

HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to 55 Years

Healthy volunteers

Inclusion criteria

* Subject was previously administered HMI 102. * Subject is able to understand the purpose and risks of the study and is willing to provide informed consent. * Subject is able to comply with all study procedures and long-term follow-up.

Exclusion criteria

* Participation in the study is not in the subject's best interest, in the opinion of the Investigator.

Design outcomes

Primary

Measure	Time frame	Description
Incidence and severity of adverse events of special interest (AESIs) and serious adverse events (SAEs) related to HMI 102	Baseline to year 5	Subjects with at least one AESI or SAE

Secondary

Measure	Time frame	Description
Plasma Phe Concentration	Baseline to year 5	Plasma phenylalanine (Phe) concentration at each time point during the study
Phe-restricted diet	Baseline to year 5	Incidence of subjects with a Phe-restricted diet
Phenylketonuria Quality of Life Questionnaire (PKU-QOL	Baseline to year 5	Quality of life (QOL), as assessed using the Phenylketonuria-QOL (PKU-QOL) questionnaire
Protein intake	Baseline to year 5	Protein intake relative to Phe concentration at each time point during the study

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026