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NCT04348708 Clinical Trial

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
Secondary ID
Status Enrolling by invitation
Phase
First received
Last updated
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
Health authority
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.

Study Design

Related Conditions & MeSH terms

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
See also
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Completed NCT00925054 - Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU Phase 2
Completed NCT00778206 - PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
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