NCT04348708Enrolling By InvitationNot Applicable
An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects With PAH Deficiency Previously Administered HMI 102
Homology Medicines, Inc2 sites in 1 country21 target enrollmentStarted: August 19, 2020Last updated:
Conditions
Trial Snapshot
- Phase
- Not Applicable
- Status
- Enrolling By Invitation
- Sponsor
- Homology Medicines, Inc
- Enrollment
- 21
- Locations
- 2
- Primary Endpoint
- Incidence and severity of adverse events of special interest (AESIs) and serious adverse events (SAEs) related to HMI 102
Study Overview
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 administered intravenously
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 18 Years to 55 Years (Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Subject was previously administered HMI
- •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.
Outcomes
Primary Outcomes
Incidence and severity of adverse events of special interest (AESIs) and serious adverse events (SAEs) related to HMI 102
Time Frame: Baseline to year 5
Subjects with at least one AESI or SAE
Secondary Outcomes
- Protein intake(Baseline to year 5)
- Plasma Phe Concentration(Baseline to year 5)
- Phe-restricted diet(Baseline to year 5)
- Phenylketonuria Quality of Life Questionnaire (PKU-QOL(Baseline to year 5)
Investigators
Study Sites (2)
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