Phase 1 MPS I Clinical Trials

3 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 1–3 of 3 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

Terminated

Phase 1

A trial to evaluate the effect of FLX-787 ODT on Motor Neuron Disease.

Spasticity & cramps in patients with Motor Neuron Disease

Flex Pharma, Inc50 enrolled3 locationsACTRN12616001060437

Completed

Phase 1

A Randomised, Cross-over Study to Evaluate Efficacy and Tolerability of FLX-787 in Patients with Multiple Sclerosis spasticity and spasms/cramps

Spasticity & cramps in patients with MS

Flex Pharma, Inc80 enrolled8 locationsACTRN12616000714482