Phase 1 LMA Clinical Trials
4 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 1–4 of 4 trials
Recruiting
Phase 1
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Pompe Disease (Infantile-Onset)MPS VIIMPS I+6 more
University of California, San Francisco10 enrolled1 locationNCT04532047
Recruiting
Phase 1Phase 2
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
Angelman Syndrome
MavriX Bio, LLC12 enrolled3 locationsNCT07181837
Recruiting
Phase 1Phase 2
An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705
Methylmalonic Acidemia
ModernaTX, Inc.56 enrolled12 locationsNCT05295433
Recruiting
Phase 1Phase 2
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
Angelman Syndrome
Ionis Pharmaceuticals, Inc.70 enrolled11 locationsNCT05127226