Long-Term Follow Up Study of Subjects Previously Administered HMI 102
An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects With PAH Deficiency Previously Administered HMI 102
1 other identifier
observational
21
1 country
2
Brief Summary
An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects with PAH Deficiency Previously Administered HMI 102
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Aug 2020
Longer than P75 for all trials
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 13, 2020
CompletedFirst Posted
Study publicly available on registry
April 16, 2020
CompletedStudy Start
First participant enrolled
August 19, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 30, 2026
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2026
ExpectedJune 30, 2022
June 1, 2022
5.5 years
April 13, 2020
June 28, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Incidence and severity of adverse events of special interest (AESIs) and serious adverse events (SAEs) related to HMI 102
Subjects with at least one AESI or SAE
Baseline to year 5
Secondary Outcomes (4)
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
Protein intake
Baseline to year 5
Study Arms (3)
Dose Level Cohort 1
Dose Level 1 of HMI-102 delivered intravenously one time
Dose Level Cohort 2
Dose Level 2 of HMI-102 delivered intravenously one time
Dose Level Cohort 3
Dose Level 3 of HMI-102 delivered intravenously one time
Interventions
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene
Eligibility Criteria
Subjects with PAH Deficiency who were previously administered HMI-102
You 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 may not qualify if:
- Participation in the study is not in the subject's best interest, in the opinion of the Investigator.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (2)
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
Biospecimen
Neutralizing AAV antibody, AAV antibody (IgG), Anti-PAH antibody, Vector DNA in blood, Vector DNA in body fluids, Serum Chemistry, Hematology
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 13, 2020
First Posted
April 16, 2020
Study Start
August 19, 2020
Primary Completion
January 30, 2026
Study Completion (Estimated)
December 1, 2026
Last Updated
June 30, 2022
Record last verified: 2022-06