Long-Term Follow Up Study of Subjects Previously Administered HMI 102
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: HMI-102.
- Who it may be relevant to
- Registry conditions: Phenylketonuria, PAH Deficiency. Basic parameters: 18 years — 55 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects With PAH Deficiency Previously Administered HMI 102
Overview
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
Interventions
- Genetic HMI-102
HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene
Primary outcome measures
- 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]
Secondary outcome measures (4)
- Plasma Phe Concentration [Time frame: Baseline to year 5]
- Phe-restricted diet [Time frame: Baseline to year 5]
- Phenylketonuria Quality of Life Questionnaire (PKU-QOL [Time frame: Baseline to year 5]
- Protein intake [Time frame: Baseline to year 5]
Eligibility criteria
Inclusion criteria
- 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.
Exclusion criteria
- Participation in the study is not in the subject's best interest, in the opinion of the Investigator.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 2 centers
- Boston Children's Hospital — Boston
- Icahn School of Medicine at Mount Sinai — New York
Identifiers
NCT: NCT04348708 · HMI-102-102