Phenylketonuria
Conditions
Keywords
mRNA-3210, Autosomal recessive genetic disorder, Central Nervous System Diseases, Nervous System Diseases, Brain Diseases, Metabolic Diseases, Phenylketonuria, In-born errors of metabolism, Phenylalanine, Rare metabolic disease
Brief summary
The main goal of this study is to assess the safety, and tolerability of multiple doses of mRNA-3210 in participants with phenylketonuria (PKU).
Interventions
DRUGmRNA-3210
IV infusion
Sponsors
ModernaTX, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
* Confirmed diagnosis of PKU due to phenylalanine hydroxylase (PAH) deficiency by molecular genetic testing from a central lab. * At least 3 blood phenylalanine levels ≥600 micromole(μmol)/Litre (L) regardless of diet: 2 obtained during the screening period (at least 72 hours apart) and at least one historical value 6 to 24 months prior to start of screening. * Have received documented approval from a study dietitian confirming that participant is willing and able to maintain dietary protein intake consistent with baseline intake during study participation. * If applicable, maintained stable dose of neuropsychiatric medication (that is, for attention deficit hyperactivity disorder (ADHD), depression, anxiety, or other psychiatric disorders) prior to enrollment and willing to maintain stable dose throughout study participation unless, per investigator assessment, a change is clinically indicated.
Exclusion criteria
* Receipt of sapropterin or large-neutral amino acids within 14 days or 5 half-lives (whichever is longer) of the start of screening. * Receipt of pegvaliase within 2 months of start of screening. * For participants previously on pegvaliase: use or planned use of any injectable drugs containing polyethylene glycol (PEG), including medroxyprogesterone injection, within 3 months prior to the start of screening and during study participation with the exception of COVID-19 vaccinations. * Receipt of any investigational drug within 30 days or 5-half-lives (whichever is longer) of screening. * History of hypersensitivity to any component/excipient used in this study. * Any other clinically significant medical condition that, in the Investigator's opinion, could interfere with the interpretation of study results or limit the participant's participation in the study Note: Other protocol-defined inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants with Treatment Emergent Adverse Events (TEAEs) | Day 1 up to 52 weeks after EOT (up to 91 weeks) |
Secondary
| Measure | Time frame |
|---|---|
| Maximum Observed Effect (Emax) | Day 1 up to 52 weeks after EOT (up to 91 weeks) |
| Area Under the Effect Versus Time Curve (AUEC) | Day 1 up to 52 weeks after EOT (up to 91 weeks) |
| Change from Baseline in Blood Phenylalanine Levels | Day 1 up to 52 weeks after EOT (up to 91 weeks) |
| Area Under the Plasma Concentration-Time Curve (AUC) | Day 1 through Day 15 for Dose 1 and Dose 12 |
| Number of Participants with Anti-Polyethylene Glycol Antibodies | Day 1 up to 52 weeks after EOT (up to 91 weeks) |
| Maximum Observed Concentration (Cmax) | Day 1 through Day 15 for Dose 1 and Dose 12 |
Outcome results
None listed