Overview
Sponsor-declared trial summary
Phase
Phase I and Phase II (Integrated) - Other
Status
Authorised, recruitment pending
Participants planned
38
Countries
3
Sites
6
Propionic Acidemia
Evaluate the long-term safety of mRNA-3927 administered to participants with PA who have previously participated in Study mRNA-3927-P101
Key facts
- Sponsor
- Moderna Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Decision date (initial)
- 2026-02-23
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Moderna TX, Inc.
External identifiers
- EU CT number
- 2022-502911-12-00
- ClinicalTrials.gov
- NCT05130437
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy, Pharmacodynamic, Safety, Efficacy
Evaluate the long-term safety of mRNA-3927 administered to participants with PA who have previously participated in Study mRNA-3927-P101
Secondary objectives 1
- Evaluate the long-term frequency of PA-related events.
Conditions and MedDRA coding
Propionic Acidemia
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 26.0 | LLT | 10080615 | Propionic acidemia | 10010331 |
Study design 2 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Treatment Period Participants will continue receiving mRNA-3927 at the same dose and at the same dosing interval last received in the mRNA-3927-P101 study, unless the Sponsor recommends modification to the dose regimen based on individual participant circumstance. Participants will receive a single dose of mRNA-3927 IV infusion on each dosing day as indicated in the SoA. Participants will receive premedication with acetaminophen/paracetamol or ibuprofen and H1/H2-receptor blockers approximately 60±10 minutes before the infusion of mRNA-3927. Safety will be monitored throughout the study based on vital signs and physical examinations, clinical laboratory analyses, ADAs, 12-lead ECGs, and reporting of AEs and SAEs.
|
Not Applicable | None | ||
| 2 | Follow-up Period Follow-up Period (up to 90 days after the last dose of mRNA-3927). Participants who transition to receiving mRNA-3927 by another mechanism (eg, another access program or commercially available mRNA-3927) will not enter the Follow-up Period but will complete the study after completing the EOT visit.
|
Not Applicable | None |
Regulatory references
- Scientific advice from competent authorities
- Food And Drug Administration, Spanish Agency Of Medicines And Medical Devices, Pharmaceuticals And Medical Devices Agency, European Medicines Agency, Medicines And Healthcare Products Regulatory Agency
- EMA paediatric investigation plan (PIP)
- EMEA-003419-PIP01-23
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2022-502910-10-00 | A Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia | Moderna Therapeutics Inc. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Participated in Study mRNA-3927-P101.
- Completed the EOT/ ET Visit in Study mRNA-3927-P101 and enroll in this study such that the first dose in this study is planned to be within 14±3 days of the last dose of mRNA-3927 in mRNA-3927-P101 study.
- Participant and/or legally authorized representative is willing and able to provide informed consent and/or assent as mandated by local regulations.
- Participant’s caregiver (and, if able to provide consent/assent, the participant) is willing and able to comply with study-related assessments.
Exclusion criteria 5
- Not expected to receive clinical benefit from continued mRNA-3927 administration, in the opinion of the Investigator.
- Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study.
- History of liver and/or kidney transplant.
- Pregnant or breastfeeding.
- Sexually active and does not agree to use a highly effective method of contraception during the Treatment Period and for 3 months after the last dose of the mRNA-3927.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Incidence and severity of AEs (including mRNA-3927 -related and not related AEs), SAEs, and AEs leading to treatment discontinuation.
Secondary endpoints 4
- Annualized frequency of Investigator-reported MDEs.
- Annualized frequency of Investigator-reported MDE-related hospitalizations.
- Annualized frequency of Investigator-reported PA-related hospitalizations.
- Annualized frequency of Investigator-reported PA-related urgent healthcare encounters.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD10256168 · Product
- Active substance
- Modified Messenger Ribonucleic Acid Encoding Human Propionyl-Coenzyme a Carboxylase Alpha and Beta Subunits Encapsulated Into Lipid Nanoparticles
- Pharmaceutical form
- DISPERSION FOR INJECTION
- Route of administration
- INTRAVENOUS
- Authorisation status
- Not Authorised
- MA holder
- MODERNATX, INC.
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/19/2156
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Moderna Inc.
- Sponsor organisation
- Moderna Inc.
- Address
- 325 Binney Street
- City
- Cambridge
- Postcode
- 02142-1038
- Country
- United States
Scientific contact point
- Organisation
- Moderna Therapeutics Inc.
- Contact name
- Moderna Clinical Trials
Public contact point
- Organisation
- Moderna Therapeutics Inc.
- Contact name
- Moderna Clinical Trials
Third parties 14
| Organisation | City, country | Duties |
|---|---|---|
| Charles River Laboratories International Inc. ORG-100041066
|
Mattawan, United States | Other |
| Fisher Clinical Services Inc. ORG-100014726
|
Allentown, United States | Other |
| Ppd Inc. ORG-100018960
|
Middleton, United States | Other |
| Pharmaceutical Product Development LLC ORG-100016999
|
Highland Heights, United States | Code 10, Other |
| PPD Development LP ORG-100011560
|
Richmond, United States | Other |
| Pharmaceutical Product Development LLC ORG-100016999
|
Wilmington, United States | Other, Code 8 |
| PPD Global Central Labs ORG-100046496
|
Zaventem, Belgium | Other |
| Fisher Clinical Services GmbH ORG-100017323
|
Weil Am Rhein, Germany | Other |
| Pharmaceutical Product Development LLC ORG-100016999
|
Wilmington, United States | Code 14 |
| PPD Development LP ORG-100011560
|
Wilmington, United States | On site monitoring, Code 11, Code 2, Code 5, Data management |
| Praxis Communications LLC ORG-100045170
|
Buffalo, United States | Other |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | E-data capture |
| Gray Consulting Inc. ORG-100044159
|
Philadelphia, United States | Other |
| PPD Development Ireland Limited ORG-100007309
|
Athlone, Ireland | Other |
Locations
3 EU/EEA countries · 6 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Authorised, recruitment pending | 6 | 2 |
| Netherlands | Authorised, recruitment pending | 4 | 2 |
| Spain | Authorised, recruitment pending | 3 | 2 |
| Rest of world
Japan, United Kingdom, United States, Saudi Arabia, Canada
|
— | 25 | — |
Investigational sites
Centre Hospitalier Regional De Marseille
Service Neurometabolisme pédiatrique, 264 Rue Saint Pierre, 13005, Marseille
Assistance Publique Hopitaux De Paris
CIC 1419 Service Maladies Métaboliques Pédiatriques, 149 Rue De Sevres, 75015, Paris
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 33 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Moderna Inc_mRNA-3927-P101-EXT_Protocol_2022-502911-12-00_Public | 5-EU-1 |
| Protocol (for publication) | D1_Moderna Inc_mRNA-3927-P101-EXT_Protocol_2022-502911-12-00_TC_NotPublic | 5-EU-1 |
| Recruitment arrangements (for publication) | K1_mRNA-3927-P101-EXT_Recruitment_Informed_Consent_Procedure_FRA_French_Public | 1.0 |
| Recruitment arrangements (for publication) | K1_mRNA-3927-P101-EXT_Recruitment-Arrangements_ESP_Public | 1.0 |
| Recruitment arrangements (for publication) | K1_mRNA-3927-P101-EXT_Recruitment-arrangements_NLD_ENG | N/A |
| Recruitment arrangements (for publication) | K2_mRNA-3927-P101_EXT_Justification-Inclusion-Vulnerable-Subjects_FRA_English_Public | n/a |
| Subject information and informed consent form (for publication) | L1_mRNA 3927-P101-EXT_Assent ICF_Ages 06 to 10_FR_FRA_Public | 7.0 |
| Subject information and informed consent form (for publication) | L1_mRNA 3927-P101-EXT_Assent ICF_Ages 11 to 14_FR_FRA_Public | 7.0 |
| Subject information and informed consent form (for publication) | L1_mRNA 3927-P101-EXT_Assent ICF_Ages 15 to 17_FR_FRA_Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_mRNA 3927-P101-EXT_Consent-12-to-17_ESP_SPA_Public | 7.0 |
| Subject information and informed consent form (for publication) | L1_mRNA-3927-P101 EXT_SIS-and-ICF_12-16 years_NLD_NLD_Public | 7.0 |
| Subject information and informed consent form (for publication) | L1_mRNA-3927-P101 EXT_SIS-and-ICF_Adults_ ICF_NLD_NLD_Public | 8.0 |
| Subject information and informed consent form (for publication) | L1_mRNA-3927-P101 EXT_SIS-and-ICF_below 12y_NLD_NLD_Public | 7.0 |
| Subject information and informed consent form (for publication) | L1_mRNA-3927-P101 EXT_SIS-and-ICF_Parent ICF_NLD_NLD_Pubic | 8.0 |
| Subject information and informed consent form (for publication) | L1_mRNA-3927-P101 EXT_SIS-and-ICF-pregnant-partner_NLD_NLD_Public | 2.3 |
| Subject information and informed consent form (for publication) | L1_mRNA-3927-P101-EXT_Adult-Parental_ICF_ESP_SPA_Public | 8.0 |
| Subject information and informed consent form (for publication) | L1_mRNA-3927-P101-EXT_Adults_ ICF_FR_FRA_Public | 8.0 |
| Subject information and informed consent form (for publication) | L1_mRNA-3927-P101-EXT_Clincierge-ICF_ESP_SPA_Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_mRNA-3927-P101-EXT_Parents_ ICF_FR_FRA_Public | 8.0 |
| Subject information and informed consent form (for publication) | L1_mRNA-3927-P101-EXT_Pregnancy and Newborn_Adult_FR_FRA_ICF_Public | 2.3 |
| Subject information and informed consent form (for publication) | L1_mRNA-3927-P101-EXT_Pregnancy and Newborn_Assent_FR_FRA_ICF_Public | 2.3 |
| Subject information and informed consent form (for publication) | L1_mRNA-3927-P101-EXT_Pregnant-Partner ICF_ESP_SPA_Public | 1.0 |
| Subject information and informed consent form (for publication) | L2_mRNA-3927-P101 EXT_Comfort Item_Memo_FR_FRA_Public | N/A |
| Subject information and informed consent form (for publication) | L2_mRNA-3927-P101-EXT_Childcare Reimbursement Receipt Template_FR_FRA_Public | 1 |
| Subject information and informed consent form (for publication) | L2_mRNA-3927-P101-EXT_Clincierge_Participant_Welcome_Letter_FR_FRA_Public | 1.0 |
| Subject information and informed consent form (for publication) | L2_mRNA-3927-P101-EXT_Clincierge-Data-Protection-Notice_FRA_French_Public | 2.0 |
| Subject information and informed consent form (for publication) | L2_mRNA-3927-P101-EXT_Clincierge-DPN_NLD_NLD_Public | 1.0 |
| Subject information and informed consent form (for publication) | L2_mRNA-3927-P101-EXT_Clincierge-Travel-Policy_FRA_French_Public | 1.0 |
| Subject information and informed consent form (for publication) | L2_mRNA-3927-P101-EXT_Patient-Card_FRA_French_Public | 3.0.0 |
| Synopsis of the protocol (for publication) | D1_Moderna Inc_mRNA-3927-P101-EXT_Protocol LL Summary_2022-502911-12-00_ENG_Public | 5-EU-1 |
| Synopsis of the protocol (for publication) | D1_Moderna Inc_mRNA-3927-P101-EXT_Protocol LL Summary_2022-502911-12-00_ES_SPA_Public | 5-EU-1 |
| Synopsis of the protocol (for publication) | D1_Moderna Inc_mRNA-3927-P101-EXT_Protocol LL Summary_2022-502911-12-00_FR_FRA_Public | 5-EU-1 |
| Synopsis of the protocol (for publication) | D1_Moderna Inc_mRNA-3927-P101-EXT_Protocol LL Summary_2022-502911-12-00_NL_DUT_Public | 5-EU-1 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-10-21 | France | Acceptable with conditions 2026-02-20
|
2026-02-23 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2026-04-09 | France | Acceptable with conditions 2026-02-20
|
2026-04-09 |