PhenylketonuriaGeneticsRare diseaseGenetic
What this study is
An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects with PAH Deficiency Previously Administered HMI 102
What's being tested
- HMI-102
Intervention · GENETIC
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene
Study details
- Official title
- An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects With PAH Deficiency Previously Administered HMI 102
- Sponsor
- Homology Medicines, Inc
- Conditions
- Phenylketonuria · PAH Deficiency
- Enrolling
- 21 people
- Starts
- 2020-08-19
- Est. completion
- 2026-12-01
- Record
- View on ClinicalTrials.gov ↗
Who can join
Age 18 Years – 55 YearsSex ALLHealthy volunteers Not acceptedYou may qualify if
- 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.
You can't join if
- Participation in the study is not in the subject's best interest, in the opinion of the Investigator.
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (2)
2 openBoston Children's Hospital
Boston, Massachusetts, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Study team
Sponsor
Homology Medicines, Inc
Study ID HMI-102-102
Have questions? The record lists no central contact. Use Consider me and the site team can reach you, or find them through ClinicalTrials.gov ↗.
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.