NCT06147856

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).

Trial Health

45
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Timeline
15mo left

Started Mar 2024

Typical duration for phase_1

Status
withdrawn

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress63%
Mar 2024Aug 2027

First Submitted

Initial submission to the registry

November 18, 2023

Completed
10 days until next milestone

First Posted

Study publicly available on registry

November 28, 2023

Completed
4 months until next milestone

Study Start

First participant enrolled

March 29, 2024

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 10, 2026

Expected
12 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 5, 2027

Last Updated

October 22, 2024

Status Verified

October 1, 2024

Enrollment Period

2.4 years

First QC Date

November 18, 2023

Last Update Submit

October 20, 2024

Conditions

Phenylketonuria

Keywords

mRNA-3210Autosomal recessive genetic disorderCentral Nervous System DiseasesNervous System DiseasesBrain DiseasesMetabolic DiseasesPhenylketonuriaIn-born errors of metabolismPhenylalanineRare metabolic disease

Outcome Measures

Primary Outcomes (1)

  • Number of Participants with Treatment Emergent Adverse Events (TEAEs)

    Day 1 up to 52 weeks after EOT (up to 91 weeks)

Secondary Outcomes (6)

  • 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)

  • Area Under the Effect Versus Time Curve (AUEC)

    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

  • Area Under the Plasma Concentration-Time Curve (AUC)

    Day 1 through Day 15 for Dose 1 and Dose 12

  • +1 more secondary outcomes

Study Arms (1)

mRNA-3210

EXPERIMENTAL

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.

Drug: mRNA-3210

Interventions

mRNA-3210DRUG

IV infusion

mRNA-3210

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

PhenylketonuriasCentral Nervous System DiseasesNervous System DiseasesBrain DiseasesMetabolic Diseases

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNutritional and Metabolic Diseases
0

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 18, 2023

First Posted

November 28, 2023

Study Start

March 29, 2024

Primary Completion (Estimated)

August 10, 2026

Study Completion (Estimated)

August 5, 2027

Last Updated

October 22, 2024

Record last verified: 2024-10