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An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects with PAH Deficiency Previously Administered HMI 102
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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| HMI-102 | Genetic | HMI-102 is an AAVHSC15 vector containing a functional copy of the human PAH gene |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma Phe Concentration | Plasma phenylalanine (Phe) concentration at each time point during the study | Baseline to year 5 |
| Phe-restricted diet | Incidence of subjects with a Phe-restricted diet |
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Inclusion Criteria:
Exclusion Criteria:
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Subjects with PAH Deficiency who were previously administered HMI-102
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States | ||
| Icahn School of Medicine at Mount Sinai |
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| ID | Term |
|---|---|
| D010661 | Phenylketonurias |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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Neutralizing AAV antibody, AAV antibody (IgG), Anti-PAH antibody, Vector DNA in blood, Vector DNA in body fluids, Serum Chemistry, Hematology
| Baseline to year 5 |
| Phenylketonuria Quality of Life Questionnaire (PKU-QOL | Quality of life (QOL), as assessed using the Phenylketonuria-QOL (PKU-QOL) questionnaire | Baseline to year 5 |
| Protein intake | Protein intake relative to Phe concentration at each time point during the study | Baseline to year 5 |
| New York |
| New York |
| 10029 |
| United States |
| D009422 | Nervous System Diseases |
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |