Gene Therapy Clinical Study in Adult PKU
Stopped early · Phase 1/Phase 2
Conditions studied: Phenylketonurias, PAH Deficiency
In brief
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.
Key facts
- Study ID
- NCT03952156
- Run by
- Homology Medicines, Inc
- People needed
- 10
- Starts
- 2019-06-10
- Expected to finish
- 2023-08-01
- Last updated by the study team
- 2023-08-29
Who can join
Age: 18 and older, up to 55. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Kaiser Permanente Los Angeles Medical Center — Los Angeles, California, United States
- Children's Hospital of Orange County — Orange, California, United States
- University of South Florida — Tampa, Florida, United States
- Emory University Hospital — Atlanta, Georgia, United States
- Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- Boston Children's Hospital — Boston, Massachusetts, United States
- Icahn School of Medicine at Mount Sinai — New York, New York, United States
- The University of North Carolina At Chapel Hill — Chapel Hill, North Carolina, United States
- Nationwide Children's Hospital — Columbus, Ohio, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- University of Texas Southwestern Medical Center — Dallas, Texas, United States
- University of Utah — Salt Lake City, Utah, United States
Full record on ClinicalTrials.gov
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