Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
- Registration Number
- NCT05506254
- Lead Sponsor
- LogicBio Therapeutics, Inc
- Brief Summary
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
- Detailed Description
Patients will be followed for a period of 15 years which includes the duration of time that the patient was in the treatment study.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 4
Inclusion Criteria
- Participant has previously participated in a LogicBio clinical trial and received hLB-001.
Exclusion Criteria
- None
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Patients who have previously received hLB-001 hLB-001 -
- Primary Outcome Measures
Name Time Method Incidence of treatment emergent adverse events 15 years
- Secondary Outcome Measures
Name Time Method
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
What molecular mechanisms does hLB-001 utilize to correct MMUT mutations in methylmalonic acidemia patients?
How do the long-term outcomes of hLB-001 gene therapy compare to standard-of-care for MMUT-related MMA?
Which biomarkers correlate with therapeutic response to hLB-001 in MMA patients with MMUT deficiency?
What are the long-term safety profiles of AAV-based gene therapy hLB-001 in MMA patients with MMUT mutations?
Are there other gene therapies or combination approaches targeting MMA caused by MMUT mutations besides hLB-001?
Trial Locations
- Locations (1)
Clinical Trial Site
🇺🇸Seattle, Washington, United States