Long-Term Safety and Clinical Activity of mRNA-3705 in Methylmalonic acidemia (MMA) Patients
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Description
This extension study will determine the long-term safety and effectiveness of mRNA-3705, a treatment for Methylmalonic acidemia (MMA), in patients who previously enrolled in and completed a prior mRNA-3705 study.
Eligibility and criteria
IRB Number:
22-020354
Eligible age range:
Clinical trial phase:
Phase I
Phase II
Official title:
What to expect
This study will include the following procedures:
- Review of Medical Records and contraception/a pregnancy test (if applicable)
- Interviews
- Questionnaires
- Physical exams and assessment of vital signs
- ECGs, ECHOs
- Nutritional assessments
- Blood and Urine collection in excess of Standard of Care
- Neurocognitive and Motor assessments
- Administration of study drug (mRNA-3705) via an intravenous infusion, pre-dose H2 blocker (such as famotidine), H1 blocker (cetirizine), and acetaminophen or ibuprofen
Related specialties
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