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Clinical Trials/NCT04348708
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:
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Conditions

Trial Snapshot

Phase
Not Applicable
Status
Enrolling By Invitation
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 OverviewStudy DesignEligibility CriteriaOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

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

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (2)

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