Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt) — StellaPhase 1 — Early testing in a small group of people (usually 20–80) to evaluate safety, determine safe dosage, and identify side effects.
Trial locations(1 site)
United States
Children's Medical Center Dallas, Dallas, Texas Age range
4 Months – 10 Years
Last updated October 2024