Phase 1 Inborn Errors of Metabolism Clinical Trials
2 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–2 of 2 trials
Recruiting
Phase 1Phase 2
Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT
LeukemiaInborn Errors of MetabolismHLH+11 more
Children's Hospital of Philadelphia100 enrolled1 locationNCT06839456
Not Yet Recruiting
Phase 1
A study to assess the safety of increasing single doses of AP003 in healthy adults.
Liver-associated inborn errors of metabolismArg-TGA premature termination codon mutationsLiver stop codon disease+1 more
Alltrna, Incorporated30 enrolled1 locationACTRN12626000194369