A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria
- Registration Number
- NCT06147856
- Lead Sponsor
- ModernaTX, Inc.
- 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- WITHDRAWN
- Sex
- All
- Target Recruitment
- 54
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/exclusion criteria apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description mRNA-3210 mRNA-3210 Participants will receive single dose of mRNA-3210 by intravenous (IV) infusion every 3 weeks (Q3W), every 2 weeks (Q2W), or every 1 week (Q1W) for up to 12 doses.
- Primary Outcome Measures
Name Time Method Number of Participants with Treatment Emergent Adverse Events (TEAEs) Day 1 up to 52 weeks after EOT (up to 91 weeks)
- Secondary Outcome Measures
Name Time Method Area Under the Effect Versus Time Curve (AUEC) 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 Change from Baseline in Blood Phenylalanine Levels Day 1 up to 52 weeks after EOT (up to 91 weeks) Maximum Observed Effect (Emax) 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 Number of Participants with Anti-Polyethylene Glycol Antibodies Day 1 up to 52 weeks after EOT (up to 91 weeks)
Trial Locations
- Locations (2)
University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Royal Adelaide Hospital
🇦🇺Adelaide, South Australia, Australia