At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- mRNA-3927 (biological)
- How long the study runs
- Study runs about 76 months (dates as stated)
- About the drug or intervention
- mRNA-3927 — biological: mRNA-3927 dispersion for IV infusion
- Patient visit burden
- Not specified by the sponsor
In plain English
This is an open-label study of a medicine called mRNA-3927 in people with propionic acidemia (PA), a rare inherited condition affecting how the body breaks down certain fats and proteins. 'Open-label' means everyone who takes part knows they are receiving the study medicine. The study is run by ModernaTX, Inc.
Who can take part
- People aged 1 year or older with propionic acidemia confirmed by genetic testing showing PCCA and/or PCCB changes.
- The first 2 people joining Part 1 must be at least 8 years old when they agree to take part.
- For Part 2 only: at least one documented metabolic decompensation event (a serious metabolic crisis) in the 12 months before agreeing to take part.
- Babies under 1 year may join if PA is picked up by newborn screening, or suspected because of metabolic symptoms plus a sibling with PA. They can start screening while waiting for genetic test results, but can only be enrolled once PA is confirmed.
- Babies in neonatal intensive care (NICU) must have been born after at least 37 weeks of pregnancy, with no other conditions that could affect the study results.
- Babies must weigh at least 3 kilograms at screening.
- Babies must have had at least one PA-related event before screening, such as signs of metabolic deterioration, a metabolic decompensation event, or certain abnormal lab results (metabolic acidosis with high anion gap, high ammonia in the blood, or low white cells or platelets).
Who may not be able to
- Lab results that are clearly abnormal and judged by the study doctor or sponsor to interfere with taking part or understanding the results.
- An estimated glomerular filtration rate (a measure of kidney function) below 30 mL/min/1.73m², or being on long-term dialysis.
- Having had, or planning, an organ transplant during the study.
- A corrected QT heart rhythm measurement above 480 milliseconds.
- Grade 3 or 4 heart failure.
- Being pregnant or breastfeeding.
- Any other condition that, in the study doctor's opinion, could affect safety, interfere with the results, or limit taking part.
What taking part involves
- • Taking the study medicine mRNA-3927 — how it is given is not stated — ask the trial team.
- • Attending screening visits to check eligibility, which for babies includes checks on weight and lab results.
- • Part 2 participants will have had their metabolic events recorded before joining.
Time commitment: Not stated — ask the trial team.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- Not specified
- Who
- All
- Number of participants
- 77
- Started
- 2021-04-15
- Last checked
- 2026-01
Plain English Summary
What is this study?
- • Testing a new treatment for propionic acidemia
- • Phase1/Phase2 - 77 participants
- • This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA)
Who can take part?
- • Adults
- • Diagnosed with propionic acidemia
Where?
- • Birmingham - University Hospital Birmingham NHS Foundation Trust
- • Birmingham - Birmingham Children's Hospital
- • London - Great Ormond Street Hospital for Children NHS Foundation Trust
- • Manchester - Willink Biochemical Genetics Unit - PPDS
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (\<1 year of age).
More detail
During the Dose Optimization Stage, after each dose cohort is fully enrolled (≥1 year of age), and the dose-limiting toxicity (DLT) observation window of at least 14 days is complete for the final participant in that cohort, the Sponsor will review the totality of available safety data in conjunction with all available PK/PD data. Based on this review, the Sponsor will recommend a revised dose and/or dosing interval. The Sponsor will abide by predefined constraints as to the maximum percentage change in dose and dose interval. A maximum of 9 cohorts will be enrolled in Part 1 (Dose Optimization). Upon establishment of a dose with an acceptable safety and PD activity in Part 1 (participants ≥1 year of age), additional participants will be enrolled into the study in Part 2 (participants ≥1 year of age) to allow for determination of the safety, efficacy, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response in infants (\<1 year of age). Participants in all the phases will participate in a predosing observational period, followed by a treatment period, and then a follow-up period after withdrawal of treatment.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: Not specified
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Participants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply:
- ✓≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants in Part 1.
- ✓≥1 year of age at the time of consent/assent if enrolled after the first 2 participants in Part 1.
- ✓Confirmed diagnosis of PA based on diagnosis by molecular genetic testing via central laboratory (PCCA and/or PCCB...
- ✓Part 2 only: At least one documented MDE in the 12-month period before consent.
Who cannot take part
- ✗Participants of all ages are excluded from the study if during Screening any of the following criteria apply:
- ✗Estimated glomerular filtration rate (eGFR) \<30 milliliters (mL)/minute/1.73 square meter (m\^2) for participants...
- ✗History of organ transplantation or planned organ transplantation during the period of study participation.
- ✗Corrected QT interval (QTc) \>480 milliseconds (ms) using Bazett's correction.
- ✗Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York...
See the full criteria
Where Is This Study? (4 UK sites)
University Hospital Birmingham NHS Foundation Trust
Birmingham B15 2TH, United Kingdom
Birmingham Children's Hospital
Birmingham B4 6NH, United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London WC1N 3JH, United Kingdom
Willink Biochemical Genetics Unit - PPDS
Manchester M13 9WL, United Kingdom
How to Get in Touch
Moderna WeCare Team
Sponsor contactCONTACT
