A Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjec… (NCT05778877) | Clinical Trial Compass
WithdrawnPhase 1/2
A Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA
Stopped: Decided by sponsor
United States0Started 2022-12-19
Plain-language summary
This Phase 1/2 study will evaluate the safety and pharmacodynamics (PD) of SEL-302, which consists of the gene transfer vector MMA-101 following administration of an immunomodulatory SEL-110 agent in pediatric subjects with Methylmalonyl-CoA Mutase (MMUT) MMA.
Who can participate
Age range3 Years – 17 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Age 3 to \<18 years at time of consent (assent where possible)
✓. Confirmed diagnosis of MMUT type methylmalonic acidemia by molecular genetic testing
✓. Clinical and biochemical diagnosis of severe MMA as defined by:
✓. sMMA level between 100 to 3,000 μmol/L
✓. A clinical history consistent with severe MMA
✓. Subjects must have fully recovered from any hospitalization for metabolic ketoacidosis or surgery at least 4 weeks prior to the start of the screening period.
✓. Parent or legal guardian are willing and able to provide informed consent. Written assent will be obtained from minors older than age seven whenever possible.
✓. Subject and caregiver must be willing to comply with study-related assessments and adhere to lifestyle considerations throughout study duration.
Exclusion criteria
✕. History of any organ transplantation.
✕. High MMUT liver enzymatic activity in the range seen in healthy subjects or MMA patients after corrective liver transplant, as demonstrated by POBT levels.
✕. Presence of Nab against AAV8 or polyethylene glycol (PEG)