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Homology Medicines Inc. has discontinued the development of this program.
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This is an open-label, sequential ascending dose-escalation, Phase 1 study to evaluate the safety and efficacy of a single intravenous (I.V.) administration of HMI-103, a gene editing development candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe restricted dietary management.
This is an open-label, sequential ascending dose-escalation, Phase 1 study to evaluate the safety and efficacy of a single intravenous administration of HMI-103, a gene editing development candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe-restricted dietary management. Up to 3 dose levels of HMI-103 may be investigated. At a given dose level, 3 participants are planned to be enrolled and dosed. Participant dosing will be staggered.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Low Dose Cohort | Experimental | HMI-103 delivered IV one time |
|
| Intermediate Dose Cohort | Experimental | HMI-103 delivered IV one time |
|
| High Dose Cohort | Experimental | HMI-103 delivered IV one time |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| HMI-103 | Drug | HMI-103 is an AAVHSC15 capsid containing a functional copy of the human PAH gene |
|
| Measure | Description | Time Frame |
|---|---|---|
| To measure incidence and severity of Treatment Emergent Adverse Events (TEAEs) and adverse events of special interest (AESIs) of a single administration of HMI-103 | Baseline to Week 104 | |
| To evaluate the efficacy of HMI-103 on reduction of plasma Phe concentration at each dose level | Mean percent change from baseline at Weeks 24-32 in plasma Phe concentration within each dose cohort post-administration of HMI-103 | Baseline to Weeks 24-32 |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the effect of HMI-103 on plasma Phe concentration relative to treatment guidelines for PKU | Incidence of plasma Phe of ≤ 360 μmol/L within each dose cohort at each timepoint post-administration of HMI-103 | Baseline to Week 104 |
| To assess durability of response |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Community Health Clinic | Topeka | Indiana | 46571 | United States | ||
| Clinic for Special Children |
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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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Dose Escalation
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Incidence of plasma Phe ≤ 360 μmol/L during Weeks 48-52 post-administration of HMI-103 |
| Weeks 48-52 |
| To assess the changes in dietary protein intake | Change from baseline in natural and total protein intake (g/day) at each timepoint post-administration of HMI-103 | Baseline to Week 104 |
| Lancaster |
| Pennsylvania |
| 17579 |
| 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 |