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

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Homology Medicines, Inc
Study ID
NCT04348708
Status
Enrolling By Invitation

Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - 55 Years
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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

Key Dates

Start date
Aug 19, 2020
Status verified
Jun 2022
Primary completion
Jan 30, 2026
Completion
Dec 1, 2026

Study Design

Enrollment
21 participants (estimated)

Arms

  • Arm: Dose Level Cohort 1
    Dose Level 1 of HMI-102 delivered intravenously one time
  • Arm: Dose Level Cohort 2
    Dose Level 2 of HMI-102 delivered intravenously one time
  • Arm: Dose Level Cohort 3
    Dose Level 3 of HMI-102 delivered intravenously one time

Primary Outcome Measure

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 ]

Locations (2)

FacilityCityStateZIPSite coordinators
Boston Children's HospitalBostonMassachusetts02115-
Icahn School of Medicine at Mount SinaiNew YorkNew York10029-

Find similar trials in Boston, MA

By research site
Boston Children's Hospital· Boston, MAIcahn School of Medicine at Mount Sinai· New York, NY

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