A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria

Phenylketonuria Clinical Trial

Official title:

A Phase 1/2, First-in-Human, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of mRNA 3210 in Participants With Phenylketonuria

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06147856
• Other study ID #: mRNA-3210-P101
• Secondary ID: 2023-506963-32-0
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: March 29, 2024
• Est. completion date: August 5, 2027

Study information

• Verified date: April 2024
• Source: ModernaTX, Inc.
• Contact: Moderna Clinical Trials Support Center
• Phone: 1-877-777-7187
• Email: clinicaltrials[@]modernatx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 54
• Est. completion date: August 5, 2027
• Est. primary completion date: August 10, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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.

Related Conditions & MeSH terms

• Phenylketonuria
• Phenylketonurias

Intervention

Drug:
• mRNA-3210
IV infusion

Locations

Country	Name	City	State
Australia	Royal Adelaide Hospital	Adelaide	South Australia
United States	University of Texas Southwestern Medical Center	Dallas	Texas

Sponsors (1)

Lead Sponsor	Collaborator
ModernaTX, Inc.

Countries where clinical trial is conducted

United States,  Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants with Treatment Emergent Adverse Events (TEAEs)		Day 1 up to 52 weeks after EOT (up to 91 weeks)
Secondary	Change from Baseline in Blood Phenylalanine Levels		Day 1 up to 52 weeks after EOT (up to 91 weeks)
Secondary	Maximum Observed Effect (Emax)		Day 1 up to 52 weeks after EOT (up to 91 weeks)
Secondary	Area Under the Effect Versus Time Curve (AUEC)		Day 1 up to 52 weeks after EOT (up to 91 weeks)
Secondary	Maximum Observed Concentration (Cmax)		Day 1 through Day 15 for Dose 1 and Dose 12
Secondary	Area Under the Plasma Concentration-Time Curve (AUC)		Day 1 through Day 15 for Dose 1 and Dose 12
Secondary	Number of Participants with Anti-Polyethylene Glycol Antibodies		Day 1 up to 52 weeks after EOT (up to 91 weeks)