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

June 28, 2022 updated by: Homology Medicines, Inc

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

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

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

Study Type

Observational

Enrollment (Anticipated)

21

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • New York
      • New York, New York, United States, 10029
        • ICAHN School of Medicine at Mount Sinai

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 55 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Subjects with PAH Deficiency who were previously administered HMI-102

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Dose Level Cohort 1
Dose Level 1 of HMI-102 delivered intravenously one time
Genetic: HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene
Dose Level Cohort 2
Dose Level 2 of HMI-102 delivered intravenously one time
Genetic: HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene
Dose Level Cohort 3
Dose Level 3 of HMI-102 delivered intravenously one time
Genetic: HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Homology Medicines, Inc

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 19, 2020

Primary Completion (Anticipated)

January 30, 2026

Study Completion (Anticipated)

December 1, 2026

Study Registration Dates

First Submitted

April 13, 2020

First Submitted That Met QC Criteria

April 13, 2020

First Posted (Actual)

April 16, 2020

Study Record Updates

Last Update Posted (Actual)

June 30, 2022

Last Update Submitted That Met QC Criteria

June 28, 2022

Last Verified

June 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HMI-102-102

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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