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

Phenylketonuria Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04348708
• Other study ID #: HMI-102-102
• Status: Enrolling by invitation
• Start date: August 19, 2020
• Est. completion date: December 1, 2026

Study information

• Verified date: June 2022
• Source: Homology Medicines, Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 21
• Est. completion date: December 1, 2026
• Est. primary completion date: January 30, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

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.

Related Conditions & MeSH terms

• PAH Deficiency
• Phenylketonuria
• Phenylketonurias

Intervention

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

Locations

Country	Name	City	State
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Icahn School of Medicine at Mount Sinai	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Homology Medicines, Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence and severity of adverse events of special interest (AESIs) and serious adverse events (SAEs) related to HMI 102	Subjects with at least one AESI or SAE	Baseline to year 5
Secondary	Plasma Phe Concentration	Plasma phenylalanine (Phe) concentration at each time point during the study	Baseline to year 5
Secondary	Phe-restricted diet	Incidence of subjects with a Phe-restricted diet	Baseline to year 5
Secondary	Phenylketonuria Quality of Life Questionnaire (PKU-QOL	Quality of life (QOL), as assessed using the Phenylketonuria-QOL (PKU-QOL) questionnaire	Baseline to year 5
Secondary	Protein intake	Protein intake relative to Phe concentration at each time point during the study	Baseline to year 5