Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT06147856




Registration number
NCT06147856
Ethics application status
Date submitted
18/11/2023
Date registered
28/11/2023
Date last updated
14/05/2024

Titles & IDs
Public title
A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria
Scientific 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
Secondary ID [1] 0 0
2023-506963-32-00
Secondary ID [2] 0 0
mRNA-3210-P101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Phenylketonuria 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - mRNA-3210

Experimental: 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.


Treatment: Drugs: mRNA-3210
IV infusion
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
Day 1 up to 52 weeks after EOT (up to 91 weeks)
Secondary outcome [1] 0 0
Change from Baseline in Blood Phenylalanine Levels
Timepoint [1] 0 0
Day 1 up to 52 weeks after EOT (up to 91 weeks)
Secondary outcome [2] 0 0
Maximum Observed Effect (Emax)
Timepoint [2] 0 0
Day 1 up to 52 weeks after EOT (up to 91 weeks)
Secondary outcome [3] 0 0
Area Under the Effect Versus Time Curve (AUEC)
Timepoint [3] 0 0
Day 1 up to 52 weeks after EOT (up to 91 weeks)
Secondary outcome [4] 0 0
Maximum Observed Concentration (Cmax)
Timepoint [4] 0 0
Day 1 through Day 15 for Dose 1 and Dose 12
Secondary outcome [5] 0 0
Area Under the Plasma Concentration-Time Curve (AUC)
Timepoint [5] 0 0
Day 1 through Day 15 for Dose 1 and Dose 12
Secondary outcome [6] 0 0
Number of Participants with Anti-Polyethylene Glycol Antibodies
Timepoint [6] 0 0
Day 1 up to 52 weeks after EOT (up to 91 weeks)

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1/Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/03/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
5/08/2027
Actual
Sample size
Target
54
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment postcode(s) [1] 0 0
5006 - Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Texas

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
ModernaTX, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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 website
https://clinicaltrials.gov/ct2/show/NCT06147856
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Moderna Clinical Trials Support Center
Address 0 0
Country 0 0
Phone 0 0
1-877-777-7187
Fax 0 0
Email 0 0
clinicaltrials@modernatx.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT06147856