This study is accepting new patients by invitation only
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents with Achondroplasia
Summary
- Eligibility
- for people ages 3-17 (full criteria)
- Location
- at San Francisco, California and other locations
- Dates
- study startedcompletion around
- Principal Investigator
- by Paul Harmatz
Description
Summary
Official Title
Keywords
Eligibility
Locations
Lead Scientist at UCSF
- Paul Harmatz
Dr. Paul Harmatz is a gastroenterologist who specializes in mucopolysaccharidoses (MPS) and other lysosomal storage diseases (genetic disorders in which a lack of certain enzymes results in progressive damage to cells and organ systems). He leads a team of specialists who diagnose and care for patients with these rare diseases, offering therapies such as weekly enzyme infusion.
Details
- Status
- accepting new patients by invitation only
- Start Date
- Completion Date
- (estimated)
- Sponsor
- QED Therapeutics, Inc.
- ID
- NCT06164951
- Phase
- Phase 3 Achondroplasia Research Study
- Study Type
- Interventional
- Participants
- Expecting 110 study participants
- Last Updated