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Gene Therapy Clinical Study in Adult PKU

NCT03952156 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2023-08-29

No results posted yet for this study

Summary

This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of HMI-102 and will be followed for safety and efficacy for 1 year.

Conditions

  • Phenylketonurias
  • PAH Deficiency

Interventions

GENETIC

HMI-102

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

GENETIC

HMI-102

Control subjects will generally have the same assessments as treated subjects. Control subjects will undergo pre-baseline procedures to confirm that they are eligible to receive treatment with HMI-102. Once eligible control subjects are dosed with HMI-102, they will initiate the same post-dose procedures as subjects who received HMI-102.

Sponsors & Collaborators

  • Homology Medicines, Inc

    lead INDUSTRY

Principal Investigators

  • Olaf A Bodamer, M.D. · Boston Children's Hospital

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-06-10
Primary Completion
2023-01-10
Completion
2023-08-01
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03952156 on ClinicalTrials.gov