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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-000446-17 | EudraCT Number | ||
| 2022-501997-20-00 | Registry Identifier | CTIS (EU) |
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The primary objective of this study is to evaluate the long-term safety and clinical activity of mRNA-3705 administered to participants with isolated methylmalonic acidemia (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency who have previously participated in other clinical studies of mRNA-3705.
Participants with isolated MMA due to MUT deficiency who were previously enrolled in other clinical studies of mRNA-3705 will have the option to enroll into this extension study provided all eligibility criteria have been met. The study will include 2 periods: 1) Treatment Period and 2) Follow-up Period (up to 6 months after the last dose of study drug).
Treatment Period will continue unless one of the following occurs: mRNA-3705 receives marketing approval and reimbursement in the country of origin of the participant (following market approval and access being in place, all participants who wish to continue treatment will be offered mRNA-3705 through market access, with the intention of preventing treatment interruption. Other appropriate access program is offered as allowed by local regulation to continue treatment with mRNA-3705. Safety monitoring will be performed for all participants under treatment per market access requirements), the participant discontinues study drug, the participant is no longer receiving clinical benefit in the opinion of the Investigator, or Sponsor discontinues the development of mRNA-3705.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| mRNA-3705 | Experimental | Participants will receive mRNA-3705 at the same dose levels at the same dosing interval (every 2 weeks [Q2W], or every 3 weeks [Q3W]) last received in the clinical study of mRNA-3705 in which they initially participated, unless the Sponsor recommends modification. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| mRNA-3705 | Drug | A sterile liquid for injection |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Drug-Related TEAEs, Unrelated TEAEs, Adverse Events of Special Interests (AESIs), Serious Adverse Events (SAEs) and TEAEs Leading to Discontinuation | Baseline up to follow-up period (up to 6 years + 6 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change in Plasma Methylmalonic Acid and 2-Methylcitric Acid (2-MC) Levels (Primary Biomarkers) From Baseline up to 18 Months | Baseline, Year 6 (Treatment Period) + Month 6 (Follow up) | |
| Pre- and Postdose Human Methylmalonyl-Coenzyme A Mutase (hMUT) mRNA and SM-86 Levels |
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Inclusion Criteria:
Exclusion Criteria:
NOTE: Other inclusion and exclusion criteria may apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Moderna WeCare Team | Contact | 1-866-663-3762 | WeCareClinicalTrials@modernatx.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCLA Medical Center | Recruiting | Los Angeles | California | 90095 | United States |
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| Baseline up to 6 years |
| Change From Baseline in Pretreatment and Post-treatment Annualized Metabolic Decompensation Events (MDEs) Rate | Baseline, Year 6 (Treatment Period) + Month 6 (Follow up) |
| Number of Healthcare Resource Utilization Visits | Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up) |
| Number of Annualized MMA-related Hospitalizations | Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up) |
| Number of Annualized MMA-related Healthcare Visits | Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up) |
| Number of Anti-Polyethylene Glycol (PEG) and Anti-hMUT Antibodies | Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up) |
| Change in Health-Related Quality of Life (HRQoL) as Measured Using the Pediatric Quality of Life Inventory (PedsQL™) at Month 3 up to 18 Months | Month 3, Year 6 (Treatment Period) + Month 6 (Follow up) |
| Change in Investigator Global Assessment of Improvement (IGA-I) Score | Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up) |
| Change in Investigator Global Assessment of Severity (IGA-S) Score | Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up) |
| Change in EuroQoL 5-Dimensions 5-level/Youth Questionnaire (EQ-5D-5L/Y) Score | Baseline up to Year 6 (Treatment Period) + Month 6 (Follow up) |
| Lucile Packard Children's Hospital at Stanford | Recruiting | Palo Alto | California | 94304 | United States |
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| Altman Clinical and Translational Research Institution | Recruiting | San Diego | California | 92037 | United States |
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| Stollery Children's Hospital University of Alberta | Recruiting | Edmonton | Alberta | T6G 2R7 | Canada |
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| Hospital For Sick Children | Recruiting | Toronto | Ontario | M5G 1X8 | Canada |
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| Hôpital Necker - Enfants Malades APHP | Recruiting | Paris | 75015 | France |
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| Erasmus MC | Recruiting | Rotterdam | 3015 AA | Netherlands |
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| Universitair Medisch Centrum Utrecht | Recruiting | Utrecht | 3584 CX | Netherlands |
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| Hospital Universitario Cruces | Recruiting | Barakaldo | Vizcaya | 48903 | Spain |
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| Hospital Universitario 12 de Octubre | Recruiting | Madrid | 28041 | Spain |
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| Birmingham Children's Hospital NHS Foundation Trust | Terminated | Birmingham | B4 6NH | United Kingdom |
| Royal Manchester Childrens Hospital | Recruiting | Manchester | M13 9WL | United Kingdom |
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| ID | Term |
|---|---|
| C537358 | Methylmalonic acidemia |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| ID | Term |
|---|---|
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| D008765 | Methylmalonyl-CoA Mutase |
| ID | Term |
|---|---|
| D019751 | Intramolecular Transferases |
| D007535 | Isomerases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
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