Observational Study for Dwarfism
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to observe and understand the growth patterns and health challenges in children with achondroplasia, a genetic condition affecting bone growth. Researchers seek to learn more about the overall quality of life, body pain, and functional abilities of these children without administering any medication. Children aged 2.5 to less than 17 years with achondroplasia, who have not recently undergone treatments like growth hormone therapy, might be suitable candidates.
As an unphased trial, this study provides a unique opportunity to contribute to understanding achondroplasia and improving future care.
Do I need to stop my current medications to join the trial?
The trial does not specify if you need to stop taking your current medications. However, if you have been treated with growth hormone, IGF 1, or anabolic steroids in the past 6 months, or have had long-term treatment with oral corticosteroids, you may not be eligible to participate.
Why are researchers excited about this trial?
Researchers are excited about this trial because it focuses on understanding achondroplasia, a genetic condition that affects bone growth in children, through an observational study. Unlike treatments that aim to modify growth or symptoms directly, this study seeks to gather comprehensive data on the natural progression of achondroplasia. By analyzing this information, scientists hope to better understand the condition and potentially improve future treatment strategies. This approach could lead to more personalized and effective interventions for children with achondroplasia.
Who Is on the Research Team?
QED Therapeutics, Inc. VP, Clinical Development
Principal Investigator
QED Therapeutics
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Observational
Evaluation of growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants
Follow-up
Participants are monitored for safety and effectiveness after the observational period
What Are the Treatments Tested in This Trial?
Interventions
- Observational Study
Find a Clinic Near You
Who Is Running the Clinical Trial?
QED Therapeutics, Inc., a Bridgebio company
Lead Sponsor
QED Therapeutics, a BridgeBio company
Lead Sponsor
QED Therapeutics, Inc.
Lead Sponsor