A Dose-finding Study to Evaluate mRNA-3210 in Participant... | NCT06147856

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

NCT06147856

Sponsor

ModernaTX, Inc.

Status

Withdrawn

Last Update Posted

Oct 22, 2024Actual

Enrollment

0Actual

Phase

Phase 1Phase 2

Conditions

Phenylketonuria

Interventions

mRNA-3210

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
2023-506963-32-00	Other Identifier	EU-CT Number

Status Module

Record Verification Date

Oct 2024

Overall Recruitment Status or Expanded Access Status

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

The Sponsor no longer intends to recruit to this study.

Expanded Access Info

Start Date

Mar 29, 2024Estimated

Primary Completion Date

Aug 10, 2026Estimated

Completion Date

Aug 5, 2027Estimated

First Submitted Date

Nov 18, 2023

First Submission Date that Met QC Criteria

Nov 18, 2023

First Posted Date

Nov 28, 2023Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Oct 20, 2024

Last Update Posted Date

Oct 22, 2024Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

0Actual

Label	Type	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
mRNA-3210	Drug	IV infusion	mRNA-3210

Measure	Description	Time Frame
Number of Participants with Treatment Emergent Adverse Events (TEAEs)		Day 1 up to 52 weeks after EOT (up to 91 weeks)

Measure	Description	Time Frame
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)

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

70 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D010661	Phenylketonurias
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D001927	Brain Diseases
D008659	Metabolic Diseases

Ancestor Terms

ID	Term
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D000592	Amino Acid Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors

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