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Gene Therapy
Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia (SUNRISE Trial)
Phase 1 & 2
Waitlist Available
Research Sponsored by LogicBio Therapeutics, Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 52
Awards & highlights
No Placebo-Only Group
Summary
This trial is a study to see if a new treatment is safe and effective for children with a rare disease. The disease is called methylmalonyl-CoA mutase gene (MMUT) mutations and the new treatment is called hLB-001.
Eligible Conditions
- Methylmalonic Acidemia
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline, week 52
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 52
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Number of Participants With Infusional Toxicities
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Secondary study objectives
Change From Baseline in Serum Albumin-2A Level at Week 52
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
5Treatment groups
Experimental Treatment
Group I: Dose Level 2 Part BExperimental Treatment1 Intervention
6 month to 2 year-olds
Group II: Dose Level 2 Part AExperimental Treatment1 Intervention
3 year-olds to 12 year-olds
Group III: Dose Level 1 Part CExperimental Treatment1 Intervention
6 month to 12 year-olds
Group IV: Dose Level 1 Part BExperimental Treatment1 Intervention
6 month to 2 year-olds
Group V: Dose Level 1 Part AExperimental Treatment1 Intervention
3 year-olds to 12 year-olds
Find a Location
Who is running the clinical trial?
AlexionIndustry Sponsor
246 Previous Clinical Trials
38,514 Total Patients Enrolled
Alexion Pharmaceuticals, Inc.Industry Sponsor
263 Previous Clinical Trials
140,587 Total Patients Enrolled
LogicBio Therapeutics, IncLead Sponsor
1 Previous Clinical Trials
4 Total Patients Enrolled
LogicBio Therapeutics, Inc.Lead Sponsor
1 Previous Clinical Trials
4 Total Patients Enrolled
Daniel Gruskin, MDStudy DirectorLogicBio Therapeutics
1 Previous Clinical Trials
4 Total Patients Enrolled