An observational study in Methylmalonic Acidemia, sponsored by Alexion Pharmaceuticals, Inc.. Active, not recruiting at 4 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-02-05.
Sponsored by Alexion Pharmaceuticals, Inc. · Observational
This is a non-interventional long-term follow-up study of patients who have participated in LogicBio studies for the treatment of MMA and received hLB-001
Patients will be followed for a period of 15 years which includes the duration of time that the patient was in the treatment study.
Alexion Pharmaceuticals, Inc. is the lead sponsor of 249 studies on the registry; 25 are open to participants now.
Of its 98 completed or terminated interventional studies of FDA-regulated products, 70 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
This is a non-interventional long-term follow-up study of participants who have participated in LogicBio studies for the treatment of MMA and received hLB-001.
Exclusion Criteria:
Drug: hLB-001
hLB-001 gene therapy
Incidence of treatment emergent adverse events
Time frame: 15 years
Plan to share: Undecided
No publications or documents are linked to this record.
This study is active, not recruiting, as verified in Feb 2026. You cannot join it, but the record below documents what was studied.
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Methylmalonic acidemia
Alexion Pharmaceuticals, Inc.