Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
LogicBio Therapeutics, Inc
With
Alexion Pharmaceuticals, Inc.
Age
0–12
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
At the time of dosing, participants must be 6 months to 12 years of age
Males and females with diagnosis of severe MMA meeting all the following;
1. Isolated MMA with genetically confirmed, pathogenic mutations in the MMUT gene
2. Screening serum/plasma methylmalonic acid level of \>100 µmol/L
3. One or more of the following considered by the PI to be MMA-related: (i) An unscheduled ER visit, hospitalization or requirement for sick day diet in the year prior to screening visit (ii) Developmental delay, movement disorder, optic neuropathy or feeding disorder with tube feeding requirement
4. Medically stable for the 2 months prior to the start of screening
You may not be if
Participants with organic acidemias other than isolated MMA, or with any other causes of hyperammonemia
Having received MMA-targeted gene therapy or nucleic acid therapy
Participants on insulin or high dose hydroxocobalamin (\> 1 mg/day OHB12 parenteral)
Kidney or liver transplant, including hepatocyte cell therapy
Estimated glomerular filtration rate (eGFR) of \< 60 mL/min/1.73 m2 based on age appropriate equations, or ongoing dialysis for renal disease
Participant tests positive for anti-rAAV-LK03-neutralizing antibodies
Clareo Health | Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia
