Status:
ENROLLING_BY_INVITATION
Long-Term Follow Up Study of Subjects Previously Administered HMI 102
Lead Sponsor:
Homology Medicines, Inc
Conditions:
Phenylketonuria
PAH Deficiency
Eligibility:
All Genders
18-55 years
Brief Summary
An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects with PAH Deficiency Previously Administered HMI 102
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 a...
Eligibility Criteria
Inclusion
- 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
- Participation in the study is not in the subject's best interest, in the opinion of the Investigator.
Key Trial Info
Start Date :
August 19 2020
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 1 2026
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT04348708
Start Date
August 19 2020
End Date
December 1 2026
Last Update
June 30 2022
Active Locations (2)
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1
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
2
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029