Phase1/2 First in Human Study to evaluate mRNA-3210 in PKU Patients
Research type
Research Study
Full 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
IRAS ID
1008839
Contact name
Mariana Faria-Urbina
Contact email
Sponsor organisation
Moderna Therapeutics (ModernaTx), Inc.
Clinicaltrials.gov Identifier
Research summary
ModernaTX, Inc. is sponsoring this trial to learn more about a possible treatment for phenylketonuria (PKU). PKU is a condition where people are unable to breakdown an amino acid (the “building blocks” of protein) called phenylalanine (Phe). Because of PKU, Phe builds up in the blood to harmful levels that can cause serious health problems. Currently, medicines that help lower blood Phe levels are limited, and dietary treatment is difficult to follow. Therefore, there is an unmet medical need for people with PKU.
The experimental treatment being studied in this trial is called mRNA-3210. The main goal of this study is to learn about the safety of different doses of mRNA-3210. To do this, researchers will check whether participants develop any medical problems whilst being treated with mRNA-3210.
This trial will include adults 18-70 years old with PKU. Participants will receive, through a needle in a vein, 12 doses of mRNA 3210 at a certain dose level over a period of 3-9 months. In this trial, researchers will test a low dose of mRNA-3210 in a group of participants first before gradually increasing the dose level for the next groups. Participants will know the dose level they are getting.
This trial will be conducted at 25 sites in 7 countries, including the United Kingdom. Participants will be in the trial for about 16-22 months and have about 60 visits. During study visits, participants will answer questions about their health and have different tests, such as blood and urine tests, done at a study site or at home so study doctors can check their health and mRNA 3210’s effects.
mRNA-3210 has not been tested in humans before, so benefits or side effects are not yet known. Participants in this trial may help doctors learn more about PKU and a possible treatment for it.REC name
London - Surrey Borders Research Ethics Committee
REC reference
23/LO/0861
Date of REC Opinion
4 Mar 2024
REC opinion
Further Information Favourable Opinion