PhenylketonuriaPhase 1/2NCT06147856
A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria
A Phase 1/2 study testing mRNA-3210 in people with Phenylketonuria, run by ModernaTX, Inc..
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). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Phenylketonuria
- What is being tested?
- mRNA-3210
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 29 Mar 2024to 5 Aug 2027
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
About this study
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
The main goal of this study is to assess the safety, and tolerability of multiple doses of mRNA-3210 in participants with phenylketonuria (PKU).
- Conditions
- Phenylketonuria
- Being tested
- mRNA-3210
- Keywords
- mRNA-3210, Autosomal recessive genetic disorder, Central Nervous System Diseases, Nervous System Diseases, Brain Diseases, Metabolic Diseases, Phenylketonuria, In-born errors of metabolism, Phenylalanine, Rare metabolic disease
- Medical subject headings
- Phenylketonurias, Central Nervous System Diseases, Nervous System Diseases, Brain Diseases, Metabolic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mRNA-3210Drug
IV infusion
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: mRNA-3210 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Measured: Day 1 up to 52 weeks after EOT (up to 91 weeks)
Secondary outcomes (6)
Change from Baseline in Blood Phenylalanine Levels
Measured: Day 1 up to 52 weeks after EOT (up to 91 weeks)
Maximum Observed Effect (Emax)
Measured: Day 1 up to 52 weeks after EOT (up to 91 weeks)
Area Under the Effect Versus Time Curve (AUEC)
Measured: Day 1 up to 52 weeks after EOT (up to 91 weeks)
Maximum Observed Concentration (Cmax)
Measured: Day 1 through Day 15 for Dose 1 and Dose 12
Area Under the Plasma Concentration-Time Curve (AUC)
Measured: Day 1 through Day 15 for Dose 1 and Dose 12
Number of Participants with Anti-Polyethylene Glycol Antibodies
Measured: Day 1 up to 52 weeks after EOT (up to 91 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Nov 2023
- Study start29 Mar 2024 (estimated)
- Primary completion10 Aug 2026 (estimated)
- Study completion5 Aug 2027 (estimated)
Why it stopped: The Sponsor no longer intends to recruit to this study.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriaStudies of the same condition, open and under way.
- More trials run by ModernaTX, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- ModernaTX, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06147856
- Sponsor's protocol ID
- mRNA-3210-P101
- Other IDs
- 2023-506963-32-00, 2023
- Status last verified
- Oct 2024
- First submitted
- 18 Nov 2023
- Last updated
- 22 Oct 2024
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06147856
- Acronym / sponsor ID
- mRNA-3210-P101
- Conditions
- Phenylketonuria
- Keywords
- mRNA-3210, Autosomal recessive genetic disorder, Central Nervous System Diseases, Nervous System Diseases, Brain Diseases, Metabolic Diseases, Phenylketonuria, In-born errors of metabolism
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The Sponsor no longer intends to recruit to this study.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- ModernaTX, Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Mar 2024 (estimated)
- Primary completion
- 10 Aug 2026 (estimated)
- Study completion
- 5 Aug 2027 (estimated)
- First posted
- 28 Nov 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main goal of this study is to assess the safety, and tolerability of multiple doses of mRNA-3210 in participants with phenylketonuria (PKU).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| mRNA-3210 | Experimental | Drug: mRNA-3210Participants 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. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| mRNA-3210IV infusion | Drug | mRNA-3210 |
Primary outcome
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Time frame: Day 1 up to 52 weeks after EOT (up to 91 weeks)
6 secondary outcomes
Change from Baseline in Blood Phenylalanine Levels
Time frame: Day 1 up to 52 weeks after EOT (up to 91 weeks)
Maximum Observed Effect (Emax)
Time frame: Day 1 up to 52 weeks after EOT (up to 91 weeks)
Area Under the Effect Versus Time Curve (AUEC)
Time frame: Day 1 up to 52 weeks after EOT (up to 91 weeks)
Maximum Observed Concentration (Cmax)
Time frame: Day 1 through Day 15 for Dose 1 and Dose 12
Area Under the Plasma Concentration-Time Curve (AUC)
Time frame: Day 1 through Day 15 for Dose 1 and Dose 12
Number of Participants with Anti-Polyethylene Glycol Antibodies
Time frame: Day 1 up to 52 weeks after EOT (up to 91 weeks)
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing mRNA-3210 in people with Phenylketonuria, run by ModernaTX, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 22 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Treatment Emergent Adverse Events (TEAEs) (time frame: Day 1 up to 52 weeks after EOT (up to 91 weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Oct 2024.