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Search / Trial NCT04348708

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

Launched by HOMOLOGY MEDICINES, INC · Apr 13, 2020

Trial Information

Current as of July 21, 2025

Enrolling by invitation

Keywords

Pku Phenylketonuria Pah Deficiency Phenylalanine Adeno Associated Virus Aavhsc15

ClinConnect Summary

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

Gender

ALL

Eligibility criteria

  • 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.

About Homology Medicines, Inc

Homology Medicines, Inc. is a clinical-stage gene editing company focused on developing transformative genetic therapies for patients with rare diseases. Leveraging its proprietary gene editing platform, the company aims to provide innovative and durable treatment options by precisely correcting genetic mutations at the DNA level. With a robust pipeline of product candidates targeting conditions such as phenylketonuria (PKU) and other genetic disorders, Homology Medicines is committed to advancing the field of genetic medicine through rigorous clinical research and a patient-centric approach. The company is dedicated to improving patient outcomes and quality of life by addressing the underlying causes of genetic diseases.

Locations

New York, New York, United States

Boston, Massachusetts, United States

Patients applied

0 patients applied

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

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